Today, we will see why Fate Therapeutics (FATE) is an attractive pick in March 2020.
Fate Therapeutics is a clinical-stage biopharmaceutical company focused on the development of next-generation cellular immunotherapies for cancer and immune disorders. The company has pioneered proprietary iPSC (induced pluripotent stem cell) platform technology to develop off-the-shelf cell-based cancer immunotherapy products. Current patient-derived autologous and allogeneic cell therapies suffer from drawbacks such as high costs, manufacturing complexity, product heterogeneity, and high turnaround time. These methods, including patient and donor-derived approaches to cell therapy, also require batch-to-batch sourcing and engineering of millions of primary cells.
Fate Therapeutics aims to be the game-changer in cell-based cancer immunotherapy space by enabling the development of off-the-shelf cell products derived from master cell lines. The company aims to develop less costly, homogenous, and multi-dose or multi-cycle cell therapies with small turnaround time. The resultant cell therapy products are expected to be well-defined and uniform in the composition and can be mass-produced at a significant scale in a cost-effective manner and can be delivered off-the-shelf for broad patient accessibility.
The company's cell therapy pipeline comprises immune-oncology programs including off-the-shelf NK- and T-cell product candidates derived from master iPSC lines, and immuno-regulatory programs, including product candidates to prevent life-threatening complications in patients undergoing hematopoietic cell transplantation and to promote immune tolerance in patients with autoimmune disease.
Human-induced Pluripotent Stem cells are generated by reprogramming adult somatic cells to a pluripotent state. Fibroblasts are the most commonly used primary somatic cell type for the generation of induced pluripotent stem cells. They are reprogrammed using retroviruses. Pluripotent cells are capable of differentiating in all cell types that make up the body.
A single human iPSC can potentially differentiate into more than 200 cell types and provides a renewable source for making cells.
NK (natural killer) cells are the body's first line of defense against tumors and various pathogens. Fate Therapeutics is leveraging its iPSC platform to produce off-the-shelf NK cell therapy products.
FT500 is Fate Therapeutics' first off-the-shelf iPSC-derived NK-cell product candidate. The FT500 study is an open-label, multi-dose Phase 1 clinical trial designed to evaluate FT500 for the treatment of advanced solid tumors.
The dose-escalation stage of the study was originally designed to assess the safety and tolerability of three once-weekly doses of FT500, without IL-2 cytokine support, as a monotherapy and in combination with one of three FDA-approved ICI (immune checkpoint inhibitor) therapies in patients that have failed prior ICI therapy.
Data for the first 12 patients in the Phase 1 study has demonstrated clean safety for the iPSC platform. The cutoff date considered was November 28, 2019. It was seen that there were no reported dose-limiting toxicities, no FT500 related Grade 3 or greater adverse events or serious adverse events, and no incidents of cytokine release syndrome, neurotoxicity, or graft-versus-host disease.
Further, the trial also involved the evaluation of a multi-dose treatment course consisting of outpatient lympho-conditioning followed by three once-weekly doses of FT500 over up to two 30-day treatment cycles. Here, based on patients' T-cell and antibody repertoire, no anti-product immune responses against FT500 were evident over the multi-dose treatment course.
A total of 62 doses of FT500 were administered to these 12 patients in a safe and well-tolerated manner. Initial clinical data thus provides strong evidence that multiple doses of iPSC-derived NK-cells can be delivered off-the-shelf without patient matching.
In December 2019, the company disclosed plans to amend the trial protocol by including IL-2 cytokine support with each dose of FT500 after completion of 300 million cells per dose cohort in the ICI combination arm. The company has commenced dose-expansion part of Phase 1 trial with 300 million cells per dose and is focusing on enrolling NSCLC patients who are refractory to or have relapsed following CBT. This tumor type is highly susceptible to NK-cell recognition and killing. The study is enrolling at three clinical sites in the U.S. Fate Therapeutics expects expansion data readout from the trial in the second half of 2020.
Fate Therapeutics is studying the second product candidate from iPSC product platform and off-the-shelf NK-cell cancer immunotherapy, FT516, in an open-label, multi-dose Phase 1 trial. This product has been engineered to augment antibody-dependent cellular cytotoxicity.
In December 2019, the company announced results for two patients dosed with FT516. FT516 was administered as a monotherapy to the first patient who was suffering from relapsed/refractory AML (acute myeloid leukemia). The company dosed FT516 in combination with rituximab to the second patient who was suffering from high-risk DLBCL (diffuse large B-cell lymphoma) and had relapsed after multiple rituximab combination regimens, autologous hematopoietic stem cell transplant, and CAR (chimeric antigen receptor) T-cell therapy. The patients had received a first treatment cycle consisting of outpatient lympho-conditioning, three once-weekly doses of FT516 and IL-2 to better promote NK-cell activity.
Initial clinical data based on bone marrow biopsy at day 42 demonstrated no morphologic evidence of leukemia. There was even evidence of hematopoietic recovery following the completion of the first FT516 treatment cycle in the AML patient. There was also no circulating leukemia cells in the patient's peripheral blood. The patient even reported the recovery of neutrophils without growth factor support. The data did not demonstrate dose-limiting toxicities, although serious adverse events were seen. Initial dose escalation data may be read out in the second half of 2020.
This initial clinical evidence highlights the high probability of engineered iPSC-derived NK-cells demonstrating anti-tumor activity in AML indication. Besides, there is a body of data that has demonstrated clinical proof-of-concept for donor-derived NK-cell therapy in relapsed refractory AML and relapsed refractory DLBCL.
In December 2019, FDA accepted FT516's second IND application for studying the product in combination with PDL1, PD1, EGFR and HER2-targeting monoclonal antibody therapies in solid tumor indications. Initially, the company plans to prioritize the combination of FT516 and avelumab in patients with advanced solid tumors who are refractory to or have relapsed following, at least one line of anti-PDL1 monoclonal antibody therapy. The company plans to initiate enrollment in a clinical trial for FT516 and avelumab in mid-2020.
Fate Therapeutics is studying off-the-shelf multi-antigen targeted CAR NK-cell product candidate, FT596, in solid tumor indications.
In December 2019, Fate Therapeutics reported favorable in vivo preclinical data for FT596.
Here, in humanized mouse models of lymphoma and leukemia, FT596's efficacy was comparable to that of primary CAR T-cells in promoting tumor clearance and extending survival. FT596 combined with rituximab also showed the enhanced killing of lymphoma cells in vivo as compared to rituximab alone. FT596 can thus emerge to be best-in-class off-the-shelf treatment in B-cell malignancies. Fate Therapeutics has started enrolling patients in the open-label Phase I study. Initial dose escalation data readout on FT596 is expected in the second half of 2020.
Fate Therapeutics has high hopes for FT596, considering that initial clinical data from a donor-derived CAR19 NK-cell program at MD Anderson, demonstrated a 73% overall response rate in patients with relapsed refractory non-Hodgkin's lymphoma and chronic lymphocytic leukemia with no major toxicities. Hence, while the efficacy seemed similar to CAR T therapy, the safety profile was differentiated in favor of CAR NK-cell therapies.
Although early, this data has highlighted CAR NK-cells' capacity to confer a high level of efficacy without the CAR-T cell therapy-related toxicities. Fate Therapeutics expects FT596 to effectively replace patient-specific and allogeneic CAR19 T-cell immunotherapies. The latter single-antigen specific and hence pose a risk of disease relapse due to antigen escape as well as cause significant toxicities due to off-target activity. FT596, on the other hand, has been engineered with three active anti-tumoral functional components.
Fate Therapeutics aims to be the first company to introduce off-the-shelf iPSC-derived CAR T-cell therapy to patients, FT819, by submitting IND in the second quarter of 2020. The company expects to file an IND application for off-the-shelf CRISPR-edited, iPSC-derived NK-cell product candidate, FT538, by early May 2020. The company has also planned IND submission for FT576 in the second half of 2020.
Although Fate Therapeutics is pioneering a revolutionary approach for mass production of off-shelf cell therapy products, its pipeline is very early stage. There has not been sufficient data from its clinical programs to make an informed estimate about the success probability of these programs. In this backdrop, the company is exposed to significant R&D failure risks. In case data readouts from FT500 and FT596 clinical programs do not match expectations, the company may witness increased share price volatility.
At the end of 2019, the company had cash worth $261 million on its balance sheet. The company spent cash worth $83.2 million on operating activities in 2019. This is a proxy for the 2019 cash burn rate. We assume that the annual cash burn rate in 2020 will be around $120 million, considering that three assets have entered in-human trials. Hence, the company seems to have cash that can sustain operations until the end of 2021. However, if cash is needed at a faster pace, the company may land up requiring more funds. This can lead to equity dilution.
According to finviz, the 12-month consensus target price of Fate Therapeutics is $37.94. On March 4, Citi analyst Yigal Nochomovitz reiterated the "Buy" rating and increased target price from $26 to $41. On March 4, Barclays analyst Peter Lawson also initiated coverage of Fate Therapeutics with an Overweight rating and $40 price target.
On March 3, BMO Capital analyst Do Kim raised the firm's price target on Fate Therapeutics to $28 from $22 and reiterated the "Market Perform" rating. On March 3, Guggenheim analyst Michael Schmidt reiterated the "Buy" rating and increased target price from $25 to $41. On March 3, Roth Capital analyst Tony Butler reiterated the "Neutral" rating but increased the target price from $20 to $30. On March 3, BTIG analyst Amanda Murphy reiterated the "Buy" rating and increased target price from $27 to $42. The analyst has also raised the estimated value of the company's iPSC platform from $740 million to $2.0 billion.
On March 3, Oppenheimer analyst Matthew Biegler reiterated the "Outperform" rating and increased the target price from $27 to $36. Piper Sandler analyst, Edward Tenthoff also reiterated the "Overweight" rating and raised the target price from $28 to $57.
In September 2019, Fate Therapeutics launched in-house GMP (Good Manufacturing Practices) manufacturing facility at headquarters in San Diego, California. This is custom designed to use clonal master iPSC lines as a renewable cell source for the consistent and scaled manufacture of off-the-shelf NK-cell and CAR T-cell products. The company has already produced hundreds of cryopreserved, infusion-ready doses of FT500, FT516, and FT596 at a low cost per dose. Currently stored in inventory, these doses are immediately available for use in the clinical settings.
The full control of cGMP production and the technical expertise to genetically engineer iPSCs and create qualified clonal master lines for clinical use implies that the company has operational expertise and redundancies required for the consistent cost-effective manufacturing and clinical supply of off-the-shelf cell products.
I believe that the 12-month target price of $30 fairly reflects the growth potential as well as risks associated with early-stage Fate Therapeutics. I consider this company to be a good pick for aggressive biotech investors with an investment horizon of at least one year.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
Read this article:
Fate Therapeutics: Potential Catalysts Ahead - Seeking Alpha
- Stem Cell Therapy for Treatment of Ocular Disorders - Hindawi - December 27th, 2022
- Researchers find out why some stress is good for you - November 6th, 2022
- Shinya Yamanaka - Wikipedia - October 29th, 2022
- Implanting a Patient's Own Reprogrammed Stem Cells Shows Early Positive ... - October 29th, 2022
- World's first stem cell treatment for spina bifida delivered during fetal surgery - UC Davis Health - October 13th, 2022
- JDRF Announces the Appointment of Qizhi Tang, Ph.D., as Co-Director of The JDRF Center of Excellence in Northern California - PR Newswire - October 13th, 2022
- California court creates regulatory uncertainty over the FDA regulation of stem cell therapies - BioEdge - October 4th, 2022
- Scientists have created a mechanical womb that can grow life in the lab - Inverse - October 4th, 2022
- ISCT: California stem cell ruling is flawed and has inserted regulatory uncertainty into the CGT market - BioPharma-Reporter.com - September 16th, 2022
- Scientists Use Stem Cells to Create Synthetic Mouse Embryos - September 16th, 2022
- UK Equine Research Hall of Fame Inductees Announced - Equi Management - September 16th, 2022
- Cultured meat startup Prolific Machines unveils its Henry Ford approach to cell growth - TechCrunch - September 16th, 2022
- New Stem Cell Therapy Protects Brain Against the Neurological Disorder - Nature World News - September 8th, 2022
- Scientists Are Working on New and Improved Hair Loss Treatments - Gizmodo - September 8th, 2022
- What happens to the brain on prescription steroids? - Medical News Today - September 8th, 2022
- How this scientist uses Legos to explain the power of stem cells - August 30th, 2022
- CIRM Bridges intern researches stem cells to grow kidneys - August 30th, 2022
- Alzheimer's: Could controlling the brain's own clean-up crew help? - Medical News Today - August 22nd, 2022
- Trial to Compare Safety and Efficacy of Novel Cell Therapy to Standard of Care in Patients With Rare Blood Cancers - Curetoday.com - August 5th, 2022
- Medpoint Global was awarded the Best Revolutionary Hair Transplant Center of the Year and continues to follow developments in its field and offer more... - August 5th, 2022
- Cell Culture Media Market Worth $14.64 Billion by 2029 - Exclusive Report by Meticulous Research - GlobeNewswire - August 5th, 2022
- Haifan Lin takes on new role as President of the ISSCR - EurekAlert - July 3rd, 2022
- UCI-led team discovers signaling molecule that potently stimulates hair growth - UCI News - July 3rd, 2022
- Calidi Biotherapeutics Announces Appointment of W.K. Alfred Yung, M.D., to its Medical Advisory Board - Business Wire - July 3rd, 2022
- Organoids reveal similarities between myotonic dystrophy type 1 and Rett syndrome - EurekAlert - July 3rd, 2022
- Our View: Roe reversal hard hit to science - The Durango Herald - July 3rd, 2022
- Connection Map Reveals Changes in the Injured Brain - Technology Networks - June 22nd, 2022
- Scientists Used CRISPR to Trace Every Human Gene to Its Function - Singularity Hub - June 22nd, 2022
- Maike Sander named to lead the Max Delbrck Center - EurekAlert - June 22nd, 2022
- CRISPR-Cas12a Editing Rates Improve with Better Directions to the Nucleus - Genetic Engineering & Biotechnology News - June 22nd, 2022
- UB-led study presents critical step forward in understanding Parkinson's disease and how to treat it - University at Buffalo - June 13th, 2022
- Stem Cells: A Case for the Use of Human Embryos in Scientific Research - June 4th, 2022
- Top Beverly Hills Plastic Surgeon, Dr. John Anastasatos, Explores Cell-Assisted Lipotransfer (CAL) with Breast Augmentation in New Publication -... - June 4th, 2022
- Regenerative Medicine Market To Grow At A CAGR Of 11.27% By 2027, Due To Advancements In Cell Biology, Genomics Research, And Gene-Editing Technology... - June 4th, 2022
- Google Reverses Ban on Ads for All Stem Cell Therapies, Will Allow FDA-Approved Ones - Gizmodo Australia - May 15th, 2022
- Head-To-Head Survey: Jasper Therapeutics (NASDAQ:JSPR) & CytRx (OTCMKTS:CYTR) - Defense World - May 15th, 2022
- Primary Cell Culture Market Mechanical Separation Segment Is Expected To Witness A Lucrative CAGR Of 11.7% Till 2028, Owing To Advancements In... - May 15th, 2022
- Ray Therapeutics Receives $4M in Funding From the California Institute for Regenerative Medicine (CIRM) - BioSpace - May 2nd, 2022
- Predicting the Future of Food - Bon Appetit - May 2nd, 2022
- FDA Grants Direct Biologics Regenerative Medicine Advanced Therapy (RMAT) Designation for the use of ExoFlo in COVID-19 Related ARDS USA - English -... - April 19th, 2022
- Chronic Diseases Is Expected To Have Positive Impact On Stem Cell Characterization Kits Market Demand Bloomingprairieonline - Bloomingprairieonline - April 19th, 2022
- Cleveland Cord Blood Center and Deverra Therapeutics Announce Agreement - Yahoo Finance - April 6th, 2022
- A model that mimics malformation associated with severe epilepsy paves way to novel therapies - EurekAlert - March 25th, 2022
- Stem cell agency: Following the money - and its performance - Capitol Weekly - December 24th, 2021
- Epithelial Cell Culture Media Market to hit US$ 303040.33 thousand by 2028, at 11.4% CAGR: The Insight Partners - Digital Journal - December 24th, 2021
- What is Biohacking? Understanding the Silicon Valley Wellness Trend Youll Hear About in 2022 - SheKnows - December 24th, 2021
- Second Woman Spontaneously Clears HIV: 'We Think More Are Out - Medscape - November 22nd, 2021
- Business Partnership for Development and Commercialization of Medical Device Implant Utilizing MSC2 for the Regeneration of Esophageal Tissue in... - November 22nd, 2021
- Misinformation on stem cell treatments for COVID-19 linked to overhyped science, researchers argue - EurekAlert - October 16th, 2021
- Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in... - October 16th, 2021
- StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WSAW - October 5th, 2021
- BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa - WWNY - October 5th, 2021
- UC Davis and the School of Medicine set new records in research funding - UC Davis Health - August 31st, 2021
- FDA gives speedy review to Bayer's Parkinson's stem cell therapy - - pharmaphorum - July 21st, 2021
- Stem cell model reveals how COVID-19 can infect the brain - Study Finds - July 21st, 2021
- Introducing the 3D bioprinted neural tissues with the potential to 'cure' human paralysis - 3D Printing Industry - July 21st, 2021
- Insception Lifebank and Cells for Life Join the Generate Life Sciences Family to Create a Global Platform - Canada NewsWire - June 6th, 2021
- Innovative Regenerative Medicine Therapies Safety Comes First - FDA.gov - June 6th, 2021
- ViaCyte to Present Late-Breaking Data at the American Diabetes Association's 81st Scientific Sessions - PRNewswire - June 6th, 2021
- Viral vector unlikely to be cause of leukemia in gene therapy patient - Science Magazine - March 11th, 2021
- Astrocytes Derived from Patients with Bipolar Disorder Malfunction - UC San Diego Health - March 11th, 2021
- Peer Recognition | The UCSB Current - The UCSB Current - March 11th, 2021
- Stem cells on the ballot - Science Magazine - January 20th, 2021
- Controlling the immune system with 'invisible stem' cells - BioNews - January 20th, 2021
- How a UCSF team is giving Cronutt the sea lion a second chance with neuroscience - University of California - October 20th, 2020
- What Proposition 14 Tells Us About California - The New York Times - October 20th, 2020
- California Proposition 14: Funding medical research that uses stem cells - KTLA - October 20th, 2020
- Technology to natures rescue - The Hindu - October 20th, 2020
- What You Need to Know About Prop 14, The Stem Cell Research Bond (Transcript) - KQED - October 5th, 2020
- UC Davis Engineers Lead $36M Effort to Improve Recovery From Spinal Cord Injuries - UC Davis - October 5th, 2020
- Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT - October 5th, 2020
- Stem Cells Offer Hope of Treatment for COVID-19 Patients with Acute Respiratory Distress Syndrome (ARDS) - HospiMedica - September 15th, 2020
- David Shavelle, MD, Named Medical Director of Adult Cardiology for the MemorialCare Heart & Vascular Institute at Long Beach Medical Center -... - September 15th, 2020
- Seeing the eye like never before | Newsroom - UW Medicine Newsroom - September 15th, 2020
- Why The FDA's Recent Approval Of New Vaccine Is A Gigantic Win In The War On Cancer - Innovation & Tech Today - September 7th, 2020
- Gore partners with San Diego company on cell delivery system with potential to treat Type 1 diabetes - delawarebusinessnow.com - August 12th, 2020
- Celularity announces the activation of first California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19 - PRNewswire - August 12th, 2020
- Meet The 12 Next-Gen Food Techs Transforming The Future Of Protein - Green Queen Media - August 12th, 2020
- BeyondSpring Initiates Expanded Access Program with Plinabulin for Patients Suffering from CIN in the US - BioSpace - August 12th, 2020
- BCLI: Phase 3 ALS Data Expected by the End of November 2020 - Yahoo Finance - August 12th, 2020