Category Archives: Molecular Medicine

Launching today: eLearning programme to support advanced therapy training in the NHS, UK universities and government bodies

UK, 19th July 2021 The Advanced Therapy Treatment Centre (ATTC) network, London Advanced Therapies (LAT) and the Cell and Gene Therapy Catapult (CGTC), in partnership with Health Education England elearning for healthcare, have developed a new eLearning programme targeted at healthcare and academic professionals to support their learning on both the fundamentals and clinical adoption of advanced therapies.

This series of eLearning sessions is designed to give the learner a core understanding of advanced therapies, how they function in the body, and the steps involved in delivering these medicines. The modules take the learner from the basics of cell and gene therapy, through to a more in-depth look at products currently being delivered through both commissioned treatments and clinical trials.

Advanced therapy medicinal products (ATMPs) are medicines for human use, which use genes, tissues or cells to offer ground-breaking new opportunities for the treatment of disease and injury. These therapies are potentially curative and can offer the promise of treating and altering the course of diseases which cannot be addressed adequately by existing pharmaceuticals, offering a lifeline to some patients who may have exhausted all other treatment options.

Learners will also be introduced to the unique challenges of bringing these pioneering advanced therapy treatments to patients, including the often nuanced logistical and handling requirements that can present unique challenges within usual standard of care.

The sessions, which can be mixed and matched according to the learners needs, comprise the following topics:

For more information and to access the sessions, visit the programme page https://www.e-lfh.org.uk/programmes/advanced-therapy-medicinal-products/

Professor Uta Griesenbach, Professor of Molecular Medicine, Imperial College London and Chair of the pan-UK ATMP Training Group commented:

As more ATMPs enter late-stage clinical trials, we identified a training need for healthcare professionals to ensure fast development of ATMPs and smooth transition into the NHS. This is a fantastic example of collaboration between NHS, academia and industry, coordinated by CGTC to produce valuable training for UK personnel and future-proof the healthcare system. Access to these sessions sets the UK apart from other countries by making high quality, standardised training available to all staff involved in the delivery of these life-changing medicines.

Professor Fiona Thistlethwaite, iMATCH Director, Medical Oncology Consultant and Christie NHIR CRF Director commented:

Providing comprehensive training for staff to understand the complex requirements of these new therapies is essential as we scale up our delivery of ATMPs across multiple patient groups. With input from recognised ATMP experts from the ATTC network and NHS partners across the UK, staff can now use the information provided to support their continued professional development. This tailored online learning approach also allows our staff to build their knowledge in a way that fits around their schedules.

About Health Education England elearning for healthcare

Health Education England elearning for healthcare (HEE elfh) works in partnership with the NHS, third sector and professional bodies to support patient care by developing eLearning resources to educate and train the health and care workforce. The eLearning programmes cover content from anaesthesia to dentistry, end of life care to mental health, and population wellbeing to sepsis. Users can access statutory and mandatory training, obtain certificates and complete eLearning sessions relevant to their role. For more information about elfh visit http://www.e-lfh.org.uk

About the Advanced Therapy Treatment Centre (ATTC) network

The Advanced Therapy Treatment Centre network was set up to address barriers to the clinical adoption of advanced therapies by increasing the capacity and capability of the NHS to efficiently deliver the growing number of these new medicines. The network is coordinated by the Cell and Gene Therapy Catapult and comprises partners in industry, academia and healthcare providers and three regional UK centres: Innovate Manchester Advanced Therapy Centre Hub (iMATCH); the Midlands-Wales Advanced Therapy Treatment Centre (MW-ATTC, comprising Birmingham, Bristol, Cambridge, Cardiff, Leicester, Nottingham and Swansea); the Northern Alliance Advanced Therapies Treatment Centre (NA-ATTC, comprising Edinburgh, Glasgow, Leeds and Newcastle). The network was established through funding from the Industrial Strategy Challenge Fund, and the fund is delivered by UK Research and Innovation. For more information please visit theattcnetwork.co.uk.

About London Advanced Therapies

London Advanced Therapies (LAT) brings together the London scientific community working in the field of Cell and gene based therapies. Funded by research England and led by Kings College London, Imperial College London and University College London, LAT aims to catalyse Londons capabilities and outputs in the area of advanced therapies, through fostering collaborative work, facilitating commercial partnerships and creating a microclimate for innovation. Established in 2019, LAT is rapidly expanding, working with colleagues throughout the UK to establish a nationwide Network of Networks.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 350 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the Industrial Strategy Challenge Fund

This project has been funded by the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The fund is delivered by UK Research and Innovation.

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Launching today: eLearning programme to support advanced therapy training in the NHS, UK universities and government bodies - PharmiWeb.com

DetailsCategory: AntibodiesPublished on Tuesday, 20 July 2021 18:19Hits: 299

Abelacimab achieved a ~80% reduction in venous thromboembolism versus a standard of care comparator in gold standard proof-of-concept efficacy study, indicating its potential in a range of thromboembolic disorders

CAMBRIDGE, MA, USA I July 19, 2021 I Anthos Therapeutics, a clinical-stage biopharma company developing innovative therapies for cardiovascular and metabolic diseases, today announced final results from the Phase 2 ANT-005 study with its novel investigational anticoagulant abelacimab. Published today in the New England Journal of Medicine,1 and simultaneously presented as a late breaker at the International Society of Thrombosis and Haemostasis (ISTH) 2021 Congress, the data showed that a single postoperative dose of abelacimab reduced the rate of venous thromboembolism (VTE) by ~80% compared to enoxaparin (a commonly used low molecular weight heparin), following elective total knee arthroplasty, the gold standard setting for potential new anticoagulants to demonstrate efficacy.

In this parallel group study, 412 participants were randomly assigned to one of three single postoperative intravenous doses of abelacimab (150mg, 75mg or 30mg) in a blinded fashion or open-label standard of care enoxaparin given subcutaneously 40mg once daily for approximately 10 days after surgery. The primary composite efficacy outcome which included deep vein thrombosis detected by venography of the operated leg and documented symptomatic VTE events occurred in4%, 5% and 13% of patients in the 150mg, 75mg and 30mg abelacimab groups respectively, compared with 22% of patients in the enoxaparin group. The 75mg and 150mg abelacimab regimens were both statistically superiorto enoxaparin(p<0.001) while the 30mg dose was non-inferior. Abelacimab was well tolerated with no safety signals and bleeding was insignificant in both study arms.

"The results of this study provide exciting new evidence that inhibition of Factor XI appears an effective way to reduce the risk of pathological thrombosis in this case, with a single post-operative dose of abelacimab. The data highlight the potential of this new approach in other clinical settings where the unmet clinical need is high," observed JeffreyI. Weitz, MD, Professor at McMaster University, Hamilton, Ontario, and one of the study authors.

Abelacimab is a highly selective, fully human monoclonal antibody with novel dual activity against both Factor XI and its activated form, Factor XIa, achieving profound Factor XI suppression for up to 30 days following a single intravenous or subcutaneous dose.1,2

Beyond the compelling efficacy data shown in this study, Anthos' vision in developing abelacimab is to achieve 'hemostasis-sparing' anticoagulation: effective protection from thromboembolic events with a reduced risk of clinically significant bleeding. According to a recently described model of the coagulation cascade,3 Factor XI plays an important role in the development of pathological thrombosis but is hypothesized to play only a minimal role in physiological hemostasis. Factor XI inhibition thus provides a potentially significant opportunity to pharmacologically 'uncouple' the two pathways.

Dan Bloomfield, MD, Chief Medical Officer at Anthos Therapeutics, explained: "All current anticoagulants including direct oral anticoagulants (DOACs) impact physiological hemostasis as well as pathological thrombosis due to their action on the 'common pathway' of the coagulation cascade, thus carrying a well-documented bleeding risk which may be even greater in the real world than in clinical trials.4,5 Fear of bleeding commonly deters prescribers and patients from pursuing optimal anticoagulation6-8soour aim in developing abelacimab is to address this major unmet need." The ongoing Phase 2 ANT-006 study (AZALEA-TIMI 71), investigating long-term once-monthly subcutaneous administration of abelacimab for stroke prevention in patients with atrial fibrillation, is expected to provide insight on the bleeding risk with abelacimab compared to a commonly used DOAC.

Anthos Therapeutics' Chief Executive Officer, John Glasspool, commented: "More than 1 in 4 people worldwide continue to die from thromboembolic events and yet 40-50% of high-risk individuals fail to receive optimal anticoagulation, mainly due to the prevailing fear of bleeding. Factor XI inhibition may provide a paradigm shift towards safer anticoagulation that inspires greater confidence among prescribers and patients." Mr. Glasspool added: "I am proud of the progress we have made to address unmet needs in high-risk cardiovascular and metabolic conditions since we launched two years ago with investment from Blackstone Life Sciences. The efficacy findings announced today represent the first major milestone in our development plans for abelacimab."

ENDS

1. Verhamme P et al. New Engl J Med 2021, in press2. Yi BA et al. ISTH 2021 poster PB00773. Hsu C et al. J Am Coll Cardiol 2021, in press4. Buderi R et al. ISTH 2021 poster PB00475. Fox, KAA et al. BMJ Open 2017; Dec 21;7(12):e017157 6. Hsu JC et al. JAMA Cardiol. 2016; 1: 5562.7. Piccini JP et al. Circulation. 2019; 139:14971506.8. Cohen AT et al. Lancet 2008; 2;371(9610):387-94

ABOUT ANTHOS THERAPEUTICSAnthos Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of genetically and pharmacologically validated innovative therapies to advance care for people living with cardiovascular and metabolic (CVM) diseases. Anthos Therapeutics aims to combine the agility of a biotech with the rigor of a large pharmaceutical company.Anthos Therapeutics was launched by Blackstone Life Sciences in 2019.

For more information: https://www.anthostherapeutics.com/

About Blackstone Life SciencesBlackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines that improve patients' lives. More information is provided athttps://www.blackstone.com/our-businesses/life-sciences/.

SOURCE: Anthos Therapeutics

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Anthos Therapeutics' novel Factor XI inhibitor abelacimab significantly outperforms standard of care enoxaparin in prospective Phase 2 efficacy...

Louis Fourie

ALTHOUGH the pharmaceutical industry is constantly evolving and new therapeutic approaches are being developed, one aspect has not changed: the use of small, synthetic molecules, which still account for 90 percent of the therapeutics on the pharmaceutical market. Some of the top-selling drugs are small-molecule drugs.

Small-molecule drugs

A small-molecule drug is an organic compound of low molecular weight (less than 900 Daltons or 1,49449-21 grams) and the size of about one nanometre capable of modulating biochemical processes to diagnose, treat or prevent diseases.

Because of their low molecular weight, small-molecule drugs have some definite advantages as therapeutics, because most can be administered orally and can pass through cell membranes to reach intracellular targets. Once inside the cells, they can be designed to engage biological targets, such as proteins, by various modes of action. Their distribution can also be customised, for example, to allow for systemic exposure or to destroy cancer cells. Therefore, many targeted therapies today are small-molecule drugs made from synthetic chemical reactions. However, they are also used in pesticides and in many other roles.

One well-known small-molecule drug is aspirin, which has been with us since 1899. Currently, the world consumes about 40000 tons of aspirin every year for a range of indications, such as cardiovascular health, Alzheimers disease, cancer treatment, pulmonary diseases, and everyday aches and pains. Another classic example is the drug penicillin, which successfully reduced the death rate caused by bacterial-related pneumonia to less than 1 percent during World War II.

Although small-molecule drugs have dominated the pharmaceutical industry since the beginning of modern medicine, it seems as though major developments are now happening in biologics, despite their excessive cost. RNA interference and CRISPR-Cas9, for example, are exciting new gene-editing tools.

But it is not the end of small-molecule medicine. Recent discoveries of small molecules that can modulate protein-protein interactions have created renewed interest in and utilities for small-molecule drugs for many diseases.Furthermore, the ability to design small molecules that can interact with and modulate RNA could create new opportunities for targeting challenging disease pathways.

An anti-ageing drug

Although with us for many years, the rapid advancement of biopharmaceutical research and technology opens up many opportunities for inventive and ingenious approaches to developing small-molecule drugs. We have therefore in recent years seen that significant advancements in structure-based design and imaging, together with automation, artificial intelligence and machine learning, have become important enablers to expedite research and enhance the success rate of small-molecule-led optimisation.

One of these innovative small-molecule drugs that could delay ageing is being tested by the US Militarys Special Operations Command (Socom), the organisation that controls the USs Special Operations forces. The anti-ageing pill comprises a human performance small molecule in the form of a nutraceutical with the aim of improving performance characteristics such as endurance and faster recovery from injuries, which typically declines with age.

Socom is working with the private biotech laboratory Metro International Biotech (MetroBiotech) on the development of an anti-ageing pill based on a human performance small molecule.

MetroBiotech is an offshoot of David Sinclairs Harvard University Medical School laboratory. The first-in-class small molecule on which they are working is Nicotinamide Mononucleotide (NMN), which MetroBiotech describes on their website as an enhancer that leverages the nicotinamide adenine dinucleotide (NAD+) cycle that is critically important to the function of all living cells and also to treat rare mitochondrial diseases and other medical conditions. These rare mitochondrial diseases often have serious effects on skeletal and cardiac muscle, as well as the central nervous system.

Increased NAD+ levels have been shown to induce mitochondrial biogenesis and enhance natural pathways (for example, sirtuins a family of dormant proteins found in all living beings) that are key to improving mitochondrial health. Mitochondria are cell parts (organelles) that produce energy for the cell in the form of a chemical called adenosine triphosphate (ATP). Cells need ATP to function properly, and NAD+ is a cofactor required for the enzymatic processes that generate energy within the cell through the continuous production of ATP inside the mitochondria.

Research has demonstrated the broad therapeutic potential of increasing NAD+ levels to preserve health and normal metabolism. It increases mitochondrial health and longevity, rejuvenates stem cells and provides neuroprotection. In general, it improves the health of most organs such as the brain (neurodegeneration), heart (inflammation, cardio protection), liver fatty acid oxidation, gluconeogenesis or regeneration), pancreas (insulin secretion), and skeletal muscles (insulin sensitivity, fatty acid oxidation) and white adipose tissue or white fat (lipogenesis).

Ageing and certain diseases, such as mitochondrial dysfunction, inflammation and other associated diseases, cause a decline in the NAD+ levels in humans, with serious consequences with regard to energy, performance and endurance. Treating people with NAD+ could thus slow the degenerative effects of ageing, prevent the onset of injury and thus allow people, according to MetroBiotech, to lead longer and healthier lives.

Several studies have been published in respected journals indicating how supplementation with a NAD precursor delays motor neuron degeneration, decreases markers of neuroinflammation in the spinal cord, improves blood flow, heart pathology, and musculoskeletal endurance, slows Alzheimers, improves energy metabolism, insulin sensitivity and plasma lipid profile, reverses retinal degeneration, mitochondrial biogenesis in skeletal muscle, and prevents noise-induced hearing loss and spiral ganglion neuron degeneration after noise exposure.

Since the small-molecule drug is a nutraceutical, a dietary supplement or food containing health-giving additive with medicinal benefit, it is not regulated by the US Food and Drug Administration and is therefore exempt from the rigorous standards regulating prescription drugs. It seems that MetroBiotech is following the nutraceutical route and not that of a prescription drug, because ageing is not yet itself a diagnosable disease to be treated by a prescription. Still, Socom have finished pre-clinical safety and dosing studies, and will soon start performance testing of the anti-ageing pill in clinical trials.

The thinking behind the anti-ageing pill is to address the cause of mitochondrial diseases and cure them all at once instead of the current repetitious and often futile one-by-one approach. According to MetroBiotech, its technology also supports key organ functions and could slow neurodegeneration, decrease inflammation in the body, increase cardio protection and improve sleep. NAD+ can apparently also reduce the functional effects of ageing on the human body, such as speed and reaction time.

A pipedream or a breakthrough?

For as long as humans have existed, they have been on a quest for a magic substance that would extend life or even bestow immortality. Over the years, the medieval alchemists and many others pursued the elixir of life. The slowing or prevention of ageing has therefore long been the Holy Grail of medicine, but has largely evaded us until now, except for a few studies in their infancy, such as Israeli oxygen therapy to increase telomere length and decrease the number of senescent cells (geriatric cells that can no longer divide).

The anti-ageing pill of MetroBiotech that slows down ageing and keeps you young may therefore sound like a sci-fi story. We will thus have to wait for the clinical trials to prove that NAD+ can indeed do what MetroBiotech is claiming it can do. However, the US military has spent serious money on this nutraceutical about R41 million since 2018. It must thus indeed be a promising drug that could become a game-changer in slowing the effects of ageing and preventing the onset of injuries. Despite being a controversial figure and often criticised, Sinclair may indeed be on the verge of a public health breakthrough that we have never seen before.

See you at our 150-year party!

Professor Louis CH Fourie is a technology strategist.

*The views expressed here are not necessarily those of IOL or of title sites.

BUSINESS REPORT ONLINE

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Tech news: Soon you will be able to stay young forever By Louis Fourie Jul 19 - IOL

Variations on a common tablet design, which can be distinguished by both colour and shape. Photo by Ragesoss (CC BY-SA 2.0)

Insulin costs around $6,000 annually for an insured individual in the U.S., a consequence of the countrys for-profit healthcare system. To get around this aspect of the government not enacting a fair and equitable healthcare system, alternative ideas are being considered. In relation to insulin, one such idea is the collective ownership by diabetics of the therapy.

It has been more than 40 years since drug development has changed from systems (heart, liver, spleen) to molecular medicine (targeted therapies). However, the pharmaceutical business model has not significantly shifted.

In relation to insulin specifically, the most commonly used forms of insulin is estimated to cost ten time more in the U.S. than in any other high-income country. The Big Three pharmaceutical companies that produce 90 percent of the insulin in the U.S. are Eli Lilly, Novo Nordisk, and Sanofi Aventis.

The situation for many diabetics is precarious. For example: You dont know if you will have enough of a freaking liquid that your whole life depends on. You dont know if you have enough life. Thats what being not sure if you can afford your insulin means said Marina Tsaplina in a Business Insider article.

Yet there are alternative models, provided that the pharmaceutical world is prepared to innovate. This requires the need to avoid duplication and internal conflicts across multi-billion-dollar organizations discourage initiative and create an unsupportable level of overhead.

Increasing innovation will require a complete restructuring of the industry and other technology-driven industries have undergone multiple generations of change. All industries and business models follow the law of diminishing returns, and many industries have come and gone through history. In fact, the pharma industry itself sprouted out from the terminal decline of the chemicals and dye industry as it was slowly commoditized.

A new breakthrough with insulin treatment could be one funded by diabetics who believe in it and stand to benefit the most from it.

The idea is for a new type of organization, and such an organization has been formed. This is a DAO (Decentralized Autonomous Organization) that exists on a blockchain. Such an organization is decentralized and therefore does not have a central point of failure.

VitaDAO is the first organization to tackle biomedical research. Thorough the use a token, the process it provides patients with a way to get governance over the intellectual property in the medicines they need. With this DAO, those working on new therapies and anyone who provides valuable work or resources can be financially empowered through tokens, becoming an active participant.

The idea is that by decentralizing intellectual property ownership the investment and capital injections can happen at an earlier stage and new open commercialization models can incentivize their development.

The VitaDAO model enables fundraising through alternative routes. Here, the public can get mobilized towards a new approach to medicine, thats not designed to keep them buying drugs to just manage the symptoms of the chronic diseases that are associated with age, but rather prevent cellular degeneration, which is the major risk factor for most chronic diseases. Cell and gene therapies, regenerative medicine approaches have the potential to completely change the way the world provides medical care.

The aim is for the experimental architecture to solve some of the problems presented, and in the long run perhaps all of them by creating entirely new, open, intellectual property business models.

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Disrupting the pharma development process with blockchain - Digital Journal

CAMBRIDGE, Mass. & NEW YORK--(BUSINESS WIRE)--Oncology practices will now be able to order and track Foundation Medicine, Inc.s comprehensive genomic profiling (CGP) tests for their patients without leaving Flatiron Healths OncoEMR platform, the two companies announced today.

This integration, the first of a series planned by Flatiron, will support more efficient clinical decision making by allowing electronic ordering, order tracking and receipt of Foundation Medicines CGP test results all within the OncoEMR platform. Almost all oncology practices use an electronic medical record (EMR) system to input, view and manage the full patient medical record in a single location, replacing a traditional paper chart with a digital one.1 EMR platforms also support clinical teams by enabling them to more efficiently order and track tests, view results, communicate treatment plans to patients and enable the completion of charting, documentation, and billing.

With the number of targeted treatments growing exponentially, the opportunity for cancer care transformation has never been greater. Clinicians increasingly rely on genomic insights to guide clinical decision-making, and Foundation Medicine is committed to implementing new solutions that enable widespread access to CGP, said Kathleen Kaa, Interim Chief Commercial Officer at Foundation Medicine. The integration of Foundation Medicine tests into OncoEMR, and other leading EMR systems to follow, is just one way were improving our offerings to fuel precision medicine for cancer patients. The integrations will create efficiencies for oncology healthcare teams to deliver precision treatment plans based on individual genomic insights to their patients.

"We are excited to welcome Foundation Medicine in the first of our planned CGP integrations with OncoEMR, said James Hamrick, MD, MPH, Vice President, Clinical Oncology at Flatiron Health. This kind of integration marks an important milestone in advancing precision medicine, helping oncologists have access to the information they need to select therapies.

The two companies are planning similar integrations with other CGP platforms and EMRs, respectively, in the oncology space, with the goal of helping every patient to realize the benefit of precision cancer care. These workflow-streamlining integrations are being designed by clinical and product experts in partnership with oncology practices.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine is a registered trademark of Foundation Medicine, Inc.

About Flatiron Health

Flatiron Health is a healthtech company dedicated to helping cancer centers thrive and deliver better care for patients today and tomorrow. Through clinical and data science, we translate patient experiences into real-world evidence to improve treatment, inform policy, and advance research. Cancer is smart. Together, we can be smarter. Flatiron.com @FlatironHealth

OncoEMR is a registered trademark of Flatiron Health.

1 2019 Genentech Oncology Trend Report. 11th ed. San Francisco, CA: Genentech; 2019: 16. Available at: https:/www.genentech-forum.com/content/dam/gene/genentech-forum/pdfs/genentech-oncology-trend-report-2019.pdf. Accessed June 22, 2021.

Source: Foundation Medicine

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Foundation Medicine and Flatiron Health Announce First-of-its-Kind Integration of Genomic Profiling Into OncoEMR - Business Wire

Magic mushroom, computer-enhanced composite image.

Psychedelic agents are experiencing a veritable renaissance. And this time not as illicit mind-expanding drugs that helped give shape to the 1960s counterculture. In recent years a slew of psychedelic agents have filled the drug development pipeline. These therapeutics are being investigated for treating conditions, such as major depressive disorder, severe anxiety, and substance abuse. Psychedelic therapeutics have moved from the fringes of medicine to the mainstream.

In May, the journal Nature Medicine published findings from a study on MDMA - Methylenedioxymethamphetamine - commonly known as Ecstasy or Molly. The first Phase 3 clinical trial conducted with psychedelic-assisted therapy found that MDMA combined with psychological counseling yielded marked relief to patients with severe post-traumatic stress disorder.

In April, a study published in the New England Journal of Medicine highlighted the benefits of treating depression with psilocybin, the psychoactive ingredient in magic mushrooms, have excited scientists, psychotherapists and entrepreneurs in the rapidly expanding field of psychedelic medicine. Other studies suggest substantial rapid and enduring antidepressant effects of psilocybin-assisted therapy among patients with major depressive disorder. The Food and Drug Administration (FDA) granted psilocybin breakthrough therapy designation.

And, esketamine was approved by the FDA on March 5th, 2019, for treatment-resistant depression. It is sold under the trade name, Spravato. Esketamine became the first FDA-approved psychedelic treatment for a psychiatric disorder. In August of last year, the FDA extended its approval for esketamine to adults with major depressive disorder with acute suicidal ideation or behavior.

In a disease area such as mental health, with a significant amount of unmet need, any increase in promising treatment options is welcome. Major depressive disorder affects approximately 17 million Americans, many of whom currently suffer from a lack of adequate treatment alternatives. However, there are possible pitfalls associated with each of the aforementioned drugs where the precautionary principle may apply. In each instance, risks of abuse and diversion must be considered.

There is the potential for abuse and possible long-term negative effects related to MDMA, an amphetamine derivative. Research hasnt definitively answered whether MDMA is addictive, although data suggest that regular MDMA use yields adaptations in the serotonin and dopamine pathways in the brain and central nervous system that may be connected to substance use disorder as well as increased impulsivity. Clearly this is an important factor to consider as medical uses for MDMA are being pursued.

In the case of psilocybin, in the span of a couple of years, the drug has gone from being a completely prohibited Schedule I drug, defined by the Drug Enforcement Administration as a controlled substance having no currently accepted medical use and a high potential for abuse, to a what some researchers recommend should be a Schedule IV controlled substance drug with a relatively low potential for abuse.

Yet, psilocybin is considered to have abuse potential. While advocates assert that psilocybin is not addictive, chronic abuse and misuse can lead to hallucinogen use disorder.

The FDA is the focal point for abuse potential assessment, and works with sponsors of agents with possible abuse potential to determine the studies required to establish approval endpoints, scheduling recommendations, and all aspects of labeling. Psilocybin has not yet been examined in an abuse potential study that would meet the criteria recommended by the FDA in its 2017 Guidance: Assessment of the Abuse Potential of Drugs.

Furthermore, abuse and misuse are not the only problems that require investigating. However rare the risks appear to be from initial reports, possible adverse events must be looked into thoroughly, particularly since clinical trial and real-world settings are vastly different. Persistent use of psilocybin may lead to long-term psychosis, alter a persons personality and perception of reality, and produce hallucinations.

Dr. Bogenschutz, a professor of psychiatry at New York University, said that until now the majority of clinical studies on psilocybin have been conducted with relatively small numbers of individuals in clinical settings designed to exclude those with schizophrenia and other serious mental problems. It is precisely these subgroups that could be predisposed to psychotic episodes, exacerbated by possibly psychosis-inducing psilocybin.

And then theres the case of esketamine, which is the S-enantiomer of ketamine, a similar (in molecular structure) but more potent agent than ketamine.The FDA label for esketamine includes a black box warning of the potential for misuse.

The experience with off-label use of ketamine for clinical depression is a sobering reminder of the importance of close monitoring for the purpose of preventing abuse and misuse from occurring. As noted in several clinical studies, ketamine drug-seeking behavior has appeared as a clinical issue, with some patients shopping infusion clinics to obtain repeated injections for mood elevation. In 2017, the American Psychiatric Association issued a consensus statement on ketamine for mood disorders: Considering the known potential for abuse of ketamine and recent reports of abuse of prescribed ketamine for the treatment of depression, clinicians should be vigilant about assessing the potential for patients to develop ketamine use disorder.

Classified as a Schedule III substance, esketamines safety profile based on real-world data includes possible dissociation, sedation, and suicidal ideation. Mark Horowitz of University College London, asserted that what the sponsor demonstrated very clearly in the trials that theyve done is that esketamine gets you a bit high for a few hours and has little effect on depression scores at 4 weeks. Horowitz maintains that esketamine is an ineffective medication. On top of that, its also a reasonably dangerous medication.

Ignoring or downplaying possible downsides or risk factors isnt going to make these issues go away. The experience with prescription opioids and other drugs, such as benzodiazepines, should give pause. Despite the benzodiazepine clonazepam being the most commonly diverted pharmaceutical in the U.S., it remains the drug most prescribed by psychiatrists to Medicare beneficiaries.

Regulators are proactively establishing a strict set of protocols for psychedelic medications. Several regulatory barriers have already been erected for the approved drug esketamine. Presumably, a similar set of restrictions would be put in place for MDMA and psilocybin. With respect to psilocybin, it appears that only licensed therapists and manufacturers will be allowed to grow the mushrooms or extract psilocybin from them, or to synthetically produce the drug, set up a psilocybin therapy center or provide therapy. Importantly, individuals being treated with the drug may only ingest it at a licensed facility with a certified therapist present.

These safeguards will help, along with systematically implemented post-marketing surveillance plans. Still, regulators and treatment providers will need to work out safe ways of administering these powerful substances in the real world, which will be different from the highly circumscribed and controlled conditions of clinical trials.

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Psychedelic Drugs Are Moving From The Fringes Of Medicine To The Mainstream - Forbes

In cases of breast cancer, bone metastasis when cancer cells spread to new sites in the bone causes the most breast cancer-related harm and is often incurable in advanced disease. A new study by University of Arizona Health Sciences researchers found that cancer cells become more aggressive when exposed to tissue stiffening and that these changes persist over time.

Tumor stiffening, which develops as diseased breast tissue becomes fibrotic, plays a major role in how breast cancer cells spread throughout the body. The paper, Breast tumor stiffness instructs bone metastasis via maintenance of mechanical conditioning, published today in the journal Cell Reports, found that the stiffness of the breast tumor microenvironment can cause changes to cancer cells that make them more aggressively spread to the bone. The resulting changes are maintained as mechanical memory, which instructs the cancer cells to send signals that lead to the breakdown of bone. Once this happens, patients often suffer debilitating complications like spontaneous fractures.

Unfortunately, bone metastasis is normally not identified until an advanced state when its not reversible, said senior author Ghassan Mouneimne, PhD, associate professor of cellular and molecular medicine and cancer biology in the UArizona College of Medicine Tucson. Whats really exciting is one day being able to take a sample from the patients primary tumor and predict who is at high risk for bone metastasis. Then we could intervene with a prevention strategy that we are now validating in the lab.

The study, which is the first to demonstrate the concept of mechanical memory during cancer metastasis, developed a novel mechanical conditioning, or MeCo, score, to quantify the cellular changes. Eventually, researchers hope the MeCo score can be used to help identify breast cancer patients who might benefit from repurposed antifibrotic treatments to prevent bone metastasis.

The higher the patients breast tumor MeCo score, the higher the likelihood they would go on to have bone metastasis and poorer outcomes, said Casey Romanoski, PhD, assistant professor of cellular and molecular medicine and a member of the BIO5 Institute and UArizona Cancer Center. This stiffness signature could have incredible clinical utility.

To further explore the clinical application, Dr. Mouneimne and Adam Watson, PhD, a former graduate student and postdoctoral fellow at the UArizona Cancer Center, worked with Tech Launch Arizona, the office of the university that commercializes inventions stemming from research, to launch a startup, MeCo Diagnostics, LLC. The company is working toward maturing the technology and bringing it to the marketplace where it can impact the lives of breast cancer patients everywhere.

It was previously known that tumor stiffness induces cellular changes that lead to a more aggressive cancer, but according to Dr. Watson, lead author on the paper, the concept of stiffness was misleading.

Most early-stage breast tumors are stiffer than surrounding tissue, yet most dont spread to bone, he said. Its not about tumor stiffness but rather stiffness responsiveness of the cancer cells, which we call mechanical conditioning.

To study this phenomenon, the team created a laboratory environment that mimicked the stiff or soft tumor environments encountered in the body and assessed how breast cancer cells responded. They found that cells grown in a stiff environment had a mechanoresponse characterized by cell spreading, invasion and turning on genes linked with both bone development and disease. And these gene changes endured even after the cells were moved to a soft environment.

Next, researchers looked at what genes were turned on and off in breast cancer cells in response to the stiff environments. Based on these gene expression changes, they developed the MeCo score, which was validated and refined using data from thousands of patients with breast cancer.

This is the culmination of a lot of work by researchers from many different fields, Dr. Mouneimne said. It highlights the environment we have at the University of Arizona Health Sciences, and how working together can make progress in this challenging area of breast cancer metastasis.

Future investigations could focus on how cancer cells maintain the gene expression changes that drive metastasis, based on additional findings that identified a transcription factor called RUNX2 that was activated by fibrotic-like stiffness. RUNX2 stays attached to the DNA as the cell divides and bookmarks which genes remain turned on, which includes the genes that drive bone metastasis and the breakdown of bone.

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Study Finds Breast Cancer's Response to Tumor Stiffness May Predict Bone Metastasis | UArizona Health Sciences - The University of Arizona Health...

Radiopharmaceuticals inadvertently injected into tissue rather than veins can harm patients and should fall under required national reporting guidelines, researchers urged Monday in Frontiers in Medicine. But the call has drawn criticism from some imaging advocates.

A 1980 policy from the Nuclear Regulatory Commission, which oversees radioactive isotope use in the U.S., currently exempts providers from reporting these medical events, known as extravasations.

But the NRC is currently reviewing a petition filed last year by Lucerno Dynamicsa Cary, North Carolina, company that sells a device used to monitor injection qualitythat would require quantification and reporting of certain extravasations.

In this context, authors of the current study reviewed adverse event databases on the topic, pointing to 38 examples listing diagnostic radiopharmaceutical extravasation as a factor in such events, according to Dustin Osborne, with the Radiology Department at the University of Tennessee Graduate School of Medicine, and colleagues.

While some questions remain, they claim their research points to new hypotheses related to inadvertent injections.

Our findings suggest that significant extravasations can or have caused patient harm and can irradiate patients' tissue with doses that exceed medical event reporting limits and deterministic effect thresholds, they added June 28. Therefore, diagnostic radiopharmaceutical injections should be monitored, and dosimetry of extravasated tissue should be performed in certain cases where thresholds are thought to have been exceeded.

Importantly, four of the seven authors are employed by Lucerno Dynamics.

Following last years petition, the NRC opened a comment period garnering nearly 400 responses. Many, including the NRC's Advisory Committee on the Medical Uses of Isotopes, supported the claim that there is no clinical data suggesting radiopharmaceutical extravasation is a patient safety issue.

A number of medical societies and organizations hold a similar sentiment, including the American College of Radiology.

In a statement sent to Health Imaging on Tuesday, the ACR said NRCs medical event requirements are a serious regulatory mechanism, adding reporting isnt meant to gather inconsequential data.

Therefore, the ACR is on recordalongside hundreds of medical and scientific stakeholdersas opposed to the companys (Lucerno Dynamics)petition, the college said over email. We support the NRCs medical advisory committee recommendations that infiltrations reported to NRC as medical events be limited to rare, outlier extravasations that result in actual harm.

The Society of Nuclear Medicine and Molecular Imaging offered its own comment Tuesday.

SNMMI firmly believes that extravasation of diagnostic radiopharmaceuticals is not a patient safety issue, past President Alan Packard, PhD, told Health Imaging over email. On those rare occasions when a significant extravasation occurs, it is managed under existing procedures under the direction of the authorized user.

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As regulators weigh revising nuclear medicine reporting rules, imaging advocates and others take sides - Health Imaging

EXTON, Pa.--(BUSINESS WIRE)--Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company that utilizes its human memory B cell discovery engine platform to discover and develop first-in-class antibody therapeutics, today announced the appointment of Andrew D. Badley, M.D., to the companys COVID-19 Advisory Board. Dr. Badley currently serves as the Chair of the Mayo COVID-19 Research Taskforce and is a leading voice in the fight against COVID-19.

Dr. Badley is a thought leader in infectious disease, and specifically COVID-19. I am delighted to announce his appointment to Immunomes COVID-19 Advisory Board, said Purnanand Sarma, Ph.D., President and CEO of Immunome. Andrews experience will be invaluable as we develop novel therapeutics to combat the evolving COVID-19 virus and other novel outbreaks.

Dr. Badley is a well-known physician and researcher who focuses on virus-host interactions, particularly on how viral proteins modify the host-immune response and on cell survival. Dr. Badley has spent 19 years with the Mayo Clinic in various roles. Currently, Dr. Badley is a Professor of Infectious Disease, serves as the Chair of the Mayo Clinic COVID-19 Research Taskforce, and is Professor and Chair of the Department of Molecular Medicine. Dr. Badley began his career at the University of Ottawa, where he served as an Associate Professor and Infectious Disease Physician. Dr. Badley received his bachelors and medical degrees from Dalhousie University. After Dalhousie, Dr. Badley completed his residency in internal medicine and his fellowship in infectious disease at Mayo School of Graduate Medical Education.

About Immunome

Immunome is a biopharmaceutical company that utilizes its proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics that are designed to change the way diseases are treated. The companys initial focus is on developing therapeutics to treat oncology and infectious diseases, including COVID-19. Immunomes proprietary discovery engine identifies novel therapeutic antibodies and their targets by leveraging the highly educated components of the immune system, memory B cells, from patients whose bodies have learned to fight off their disease. For more information, please visit http://www.immunome.com.

Forward-Looking Statements

This press release includes certain disclosures that contain forward-looking statements intended to qualify for the safe harbor from liability established by the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Immunomes beliefs and expectations regarding the advancement of its oncology and COVID-19 therapeutic antibody programs, execution of its clinical and strategic plans, anticipated upcoming milestones for IMM-BCP-01 and IMMONC01, including expectations regarding therapeutic potential and benefits thereof, and IND filings. Forward-looking statements may be identified by the words anticipate, believe, estimate, expect, intend, plan, project, may, will, could, should, seek, potential and similar expressions. Forward-looking statements are based on Immunomes current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, those risks and uncertainties associated with: the impact of the COVID-19 pandemic on Immunomes business, operations, strategy, goals and anticipated milestones; Immunomes ability to execute on its strategy including with respect to the timing of its R&D efforts, IND filings, initiation of clinical studies and other anticipated milestones; the timing and effectiveness of any antibody therapeutics which may be developed by Immunome; Immunomes ability to fund operations; and the additional risks and uncertainties set forth more fully under the caption Risk Factors in Immunomes Annual Report on Form 10-K filed with the United States Securities and Exchange Commission (SEC) on March 25, 2021, and elsewhere in Immunomes filings and reports with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Immunome undertakes no duty to publicly update or revise any forward looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable law.

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Immunome Appoints Andrew Badley, M.D., to COVID-19 Advisory Board - Business Wire

SAN RAFAEL, Calif., July 2, 2021 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced three oral presentations and nine poster presentations related to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress being held July 17-21, 2021. Notably, these presentations will include highlights from the Phase 3 GENEr8-1 trial, the largest gene therapy trial in Hemophilia A, and five years of clinical follow-up from the Phase 1/2 study, both of which continue to demonstrate prolonged hemostatic efficacy without the need for other treatment for hemophilia A.

"We are proud of the consistent and dramatic bleed control results to date, based on both long-term extension studies of at least five years, and the largest and most definitive gene therapy study in Hemophilia A. We look forward to the scientific presentations of the growing body of evidence for valoctocogene roxaparvovec and ensuing discussions at this important meeting," saidHank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.

BioMarin's presentations at ISTH include:

Platform Presentations

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results from the Phase 3 GENEr8-1 TrialProfessor Margareth C. Ozelo, Hematology and Transfusion Medicine,Internal Medicine Department - School of Medical Sciences of UNICAMP,University of Campinas-UNICAMPMonday, July 19, 2021, 10-11 AM EDT

Hemostatic Response is Maintained for up to 5 Years Following Treatment with Valoctocogene Roxaparvovec, an AAV5-hFVIII-SQ Gene Therapy for Severe Hemophilia AProfessor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia CentreWednesday, July 21, 2021, 10-11 AM EDT

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia ModelDr. Paul Batty, Department of Pathology and Molecular Medicine, Queen's UniversityWednesday, July 21, 2021, 1-2 PM EDT

Poster Presentations

Poster #

Title and Authors

LPB0022

Global seroprevalence of pre-existing immunity against various AAV serotypes in people with haemophilia A

Klamroth R, Hayes G, Andreeva T, Suzuki T, Hardesty B, Shima M, Pollock T, Slev P, Oldenburg J, Ozelo M, Castet S, Mahlangu J, Peyvandi F, Kazmi R, Leavitt A, Callaghan M, Pan-Petesch B, Quon D, Li M, Wong WY.

PB0663

A savvy approach in clinical trial recruitment for the SAAVY (Seroprevalence of AAV AntibodY) study in the era of COVID-19: Designing for a prospective, observational study in the United States during a global pandemic

Valentino L, Vaghela M, Lauw M, Dela Cerda G, Jones M, Hinds D, Newman V, Leal-Padinas F, Rotellini D, Schafer K, Pipe S.

PB0488

Exploring the level of congruence between patient- and physician-reported anxiety and depression in persons with haemophilia A

Burke T, Shaikh A, Pedra G, Hawes C, Camp C, O'Hara J.

PB0468

Examination and validation of a patient-centric joint metric: "PROBLEM JOINT"; empirical evidence from the CHESS Paediatrics dataset

Burke T, Rodriguez-Santana I, O'Hara J, Chowdary P, Curtis R, Khair K, McLlaughlin P, Noone D, O'Mahoney B, Pasi J, Skinner M.

PB0452

Real-world clinical and patient-centric outcomes in people with haemophilia A in France: Combined findings from the CHESS and CHESS II studies

Shaikh A, Burke T, Hawes C, Duport G, O'Hara J, Camp C.

PB0487

Real-world clinical and patient-centric outcomes in people with haemophilia A in Germany: Combined findings from the CHESS and CHESS II studies

Shaikh A, Burke T, Hawes C,Becker T, Brandt S, O'Hara J, Camp C.

PB0464

Real-world clinical and patient-centric outcomes in people with haemophilia A in Italy: Combined findings from the CHESS and CHESS II studies

Shaikh A, Burke T, Hawes C, Lupi A, O'Hara J, Camp C.

PB0456

Real-world clinical and patient-centric outcomes in people with haemophilia A in Spain: Combined findings from the CHESS and CHESS II studies

Shaikh A, Burke T, Hawes C, O'Hara J, Camp C.

PB0479

Real-world clinical and patient-centric outcomes in people with haemophilia A in the United Kingdom: Combined findings from the CHESS and CHESS II studies

Shaikh A, Burke T, Hawes C, McKeown W, Morgan D, O'Hara J, Camp C.

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. The ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October.

Regulatory Status

BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) on June 25, 2021. In May 2021, the EMA granted the Company's request for accelerated assessment. Accelerated assessment potentially reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is anticipated in the first half of 2022.

The MAA submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study.

In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec inMarch 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A.The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A. The Company is also running a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 45 to 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for adults with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visitwww.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about: (i) the development of BioMarin's valoctocogene roxaparvovec program generally, (ii) the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, (iii) the anticipated timing of a CHMP opinion in the first half of 2022, (iv) our plans in the U.S. to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study in response to FDA's request for these data to support their benefit-risk assessment of valoctocogene roxaparvovec, (v) our target Biologics License Application (BLA) submission date in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review procedure by the FDA, and (vi) the potential approval and commercialization of valoctocogene roxaparvovec for the treatment of severe hemophilia A, including timing of such approval decisions.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the above interim data; any potential adverse events observed in the continuing monitoring of the patients in the Phase 1/2 trial; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the content and timing of decisions by local and central ethics committees regarding the clinical trials; BioMarin's ability to successfully manufacture valoctocogene roxaparvovec; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter endedMarch 31, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

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SOURCE BioMarin Pharmaceutical Inc.

http://www.biomarin.com

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BioMarin Announces 12 Presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress - PRNewswire