2022 FDA Approvals
I would like to start a thread on FDA approval in 2022 with the focus on small to medium market cap companies. I will start with February as I will play the run-up and not the actual approval because of the associated risks.
Feb
17 Feb 2022 – (AGIO) Agios Pharmaceuticals, Inc.
23 Feb 2022 – (IMCR) Immunocore Holdings Limited
25 Feb 2022 – (RETA) Reata Pharmaceuticals, Inc.
28 Feb 2022 – (LEGN) Legend Biotech Corp
28 Feb 2022 – (AMYT) Amryt Pharma plc
28 Feb 2022 – (CTIC) CTI BioPharma Corp.
March
16 Mar 2022 – (RYTM) Rhythm Pharmaceuticals, Inc.
20 Mar 2022 – (MRNS) MARINUS PHARMACEUTICALS INC
21 Mar 2022 – (TXMD) THERAPEUTICSMD, INC.
25 Mar 2022 – (TGTX) TG Therapeutics, Inc.
25 Mar 2022 – (ZGNX) ZOGENIX, INC.
09 Mar 2022 – (AKBA) Akebia Therapeutics, Inc.
29 Mar 2022 – (FGEN) FIBROGEN INC
April
05 Apr 2022 – (BTAI) BioXcel Therapeutics
14 Apr 2022 – (ALNY) ALNYLAM PHARMACEUTICALS, INC.
18 Apr 2022 – (INCY) INCYTE
17 Feb 2022 – (AGIO) Agios Pharmaceuticals, Inc.
- Pipeline: Pipeline - Agios
- Approved drug(s): Idhifa (enasidenib) and Tibsovo (ivosidenib)
- SEC Filing for the Mitapivat Approval : https://www.sec.gov/Archives/edgar/data/0001439222/000119312521317685/d199842dex991.htm
- Received FDA Priority Review for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency; PDUFA Date Set for Feb. 17, 2022
- I believe FDA priority review was provided given the lack of approved therapies for this genetic disease, which leads to red blood cell destruction and anaemia
- NDA (new drug application) submission is based on results from two pivotal studies, ACTIVATE and ACTIVATE-T (https://investor.agios.com/news-releases/news-release-details/agios-announces-updated-data-activate-and-activate-t-phase-3)
- The trial found that 40% of patients who received mitapivat experienced a change in hemoglobin levels, which was sustained at several observation points following dosing. None of the patients receiving the placebo experienced a change in hemoglobin.
- Given this tiny population, the FDA and EMA “were comfortable” with the size and design of Activate-T, he added, particularly as a supplement to the randomised, placebo-controlled Activate trial in non-transfusion-dependent PKD patients, which read out positively in December
- No new safety concerns or adverse events that led to discontinuing treatment were observed in the trial.
- Long-term extension data demonstrate that previously reported effects of mitapivat on hemoglobin and transfusion burden were maintained over time (https://investor.agios.com/news-releases/news-release-details/agios-presents-mitapivat-long-term-extension-data-demonstrating)
- PKD is a small indication, just 15-20% of those with PKD, a disease that itself only affects 3,000-8,000 in the US and EU
- The firm expects peak sales of $444 million in 2034 for mitapivat in pyruvate kinase deficiency.
- Analysts Just Made A Major Revision To Their Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Revenue Forecasts (https://www.nasdaq.com/articles/analysts-just-made-a-major-revision-to-their-agios-pharmaceuticals-inc.-nasdaq%3Aagio)
- I do not expect a run up given the small patient group and the revenue forecasts
23 Feb 2022 – (IMCR) Immunocore Holdings Limited
- Pipeline: Pipeline :: Immunocore
- News release for tebentafusp in metastatic uveal melanoma: Immunocore Reports Third Quarter 2021 Financial Results and
- Biologics License Application (BLA) and Marketing Authorisation Application (MAA) submissions accepted for tebentafusp in metastatic uveal melanoma; FDA set a PDUFA date of February 23, 2022
- The submissions are based on findings from the phase 3 IMCgp100-202 trial (NCT03070392) , which evaluated tebentafusp in a population of patients with previously untreated metastatic uveal melanoma.2
- Data from the study, which were presented at the 2021 American Association for Cancer Research Annual Meeting , indicated that after a median follow up of 14.1 months, patients treated with the experimental agent experienced a median overall survival (OS) of 21.7 months (95% CI, 18.6-23.6) compared with investigator’s choice of pembrolizumab (Keytruda), ipilimumab (Yervoy), or dacarbazine at 16.0 months (95% CI, 9.7-19.4; stratified HR, 0.51; 95% CI, 0.37-0.71; P <.0001). Moreover, the 1-year OS rate was 73.2% in the tebentafusp arm vs 58.5% in the control arm.
- Priority review designation for the novel T-cell receptor bispecific immunotherapy agent could shorten the review period from a standard 10 months to 6 months from the filing acceptance. As such, the anticipated Prescription Drug User Fee Act date is February 23, 2022
- Previously, the FDA had granted tebentafusp a breakthrough therapy designation as a treatment for adult patients with HLA-A*02:01–positive unresectable or metastatic uveal melanoma.
- Uveal melanoma is a rare and aggressive form of melanoma, which affects the eye.
- approximately 8,000 new patients diagnosed globally each year (1,600-2,000 cases per year in the United States). Up to 50% of people with uveal melanoma will eventually develop metastatic disease. When the cancer spreads beyond the eye, only approximately half of patients will survive for one year.
- I believe this is long-term investment and a potential buy out candidate
25 Feb 2022 – (RETA) Reata Pharmaceuticals, Inc.
- Pipeline: Reata Pharmaceuticals | Biogen
- SEC Filing: https://www.sec.gov/Archives/edgar/data/0001358762/000156459021055047/reta-10q_20210930.htm
- In April 2021, the FDA accepted for filing the NDA for bardoxolone for the treatment of patients with CKD caused by Alport syndrome, which is currently under review by the FDA.
- We also completed a mid-cycle communication meeting with the FDA and were advised that an Advisory Committee meeting is scheduled for December 8, 2021.
- Reata Pharmaceuticals Stock Nosedives On FDA Panel’s Rejection Of Kidney Drug: https://www.investors.com/news/technology/reta-stock-nosedives-on-fda-panels-rejection-of-kidney-drug/
- FDA’s Cardiovascular and Renal Drugs Advisory Committee delivered a complete shutout. None of the 13 experts said the evidence shows bardoxolone slows the progression of chronic kidney disease in those patients.
- The FDA has until Feb. 25 to make a decision on bardoxolone. Although the agency can reject the advisory committee’s vote, it often takes the recommendation into consideration
- I believe it is highly unlikely that this will be approved, share price plummeted already heavily so it is not even a bearish play.
28 Feb 2022 – (LEGN) Legend Biotech Corp
- Pipeline: Pipeline - Legend Biotech
- SEC Filing: https://www.sec.gov/Archives/edgar/data/0001801198/000119312521361120/d270866d424b5.htm
- As part of the BLA acceptance, the FDA originally granted cilta-cel priority review and set the PDUFA target action date for November 29, 2021
- In the U.S., cilta-cell has been granted the FDA’s breakthrough therapy and orphan drug designations
- based on data from a Phase 1b/2 clinical trial called CARTITUDE-1 (NCT03548207).
- CARTITUDE-1 included patients who had received at least three prior lines of treatment, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and a CD38 inhibitor, or who failed to respond to treatment with a PI and an IMiD. Among the 97 participants in the study, the median number of prior therapies was six, and most were refractory to three classes of treatment.
- The PDUFA date was subsequently extended by the FDA to February 28, 2022 to allow sufficient time for the FDA to review information submitted pertaining to an updated analytical method following an FDA information request.
- A concern for Legend is that larger, better-funded companies such as Bristol Myers Squibb, Amgen, and Janssen, a subsidiary of Johnson & Johnson, are also targeting BCMA to treat multiple myeloma.
- strategic collaboration with Janssen Biotech, Inc., or Janssen, to develop and commercialize our lead product candidate, ciltacabtagene autoleucel, or cilta-cel, an investigational BCMA-targeted CAR-T cell therapy for patients living with multiple myeloma
- Interim data from the trial showed high response rates after a median of 12.4 months, or just over one year. These early results showed that 97% of patients experienced a reduction in cancer burden, and 67% had no detectable cancer.
- More recent data, with a median follow-up time of 18 months, showed a similarly high response rate (98%), with 80% of participants having no detectable cancer. That suggests a deepening of clinical response over time, according to the researchers.
- Legend Biotech (LEGN) Announces Proposed $300M Share Offering: Legend Biotech (LEGN) Announces Proposed $300M Share Offering
- $500 m Sales in 2026
- CAR-T requires a complex ecosystem in terms of manufacturing and the overall supply chain, so the collaboration with Janssen was needed to establish and fund this ecosystem. Wondering if the collaboration will evolve into a acquisition. Could be a good candidate to play the run-up.
28 Feb 2022 – (AMYT) Amryt Pharma plc
- Pipeline: Pipeline - Amryt Pharma
- Three approved products: metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); and lomitapide (Juxtapid®/ Lojuxta®).
- SEC Filing: https://www.sec.gov/Archives/edgar/data/0001783010/000117184321008160/f424b3_112321.htm
- In June 2021, Amryt received confirmation from the FDA that its NDA for Oleogel-S10 had been accepted and granted priority review
- Oleogel-S10 has received Fast Track Designation, and has been granted Rare Pediatric Disease Designation by FDA
- The NDA is supported by positive results from Amryt’s pivotal global Phase 3 trial in EB (“EASE”) reported in September, 2020. EASE was the largest ever global Phase 3 study conducted in patients with EB and is the first Phase 3 trial ever to demonstrate positive results in EB.
- EASE is the first Phase 3 trial to demonstrate a statistically significant increase in speed of wound healing in Epidermolysis Bullosa
- The primary endpoint of the trial was met (p-value = 0.013). EASE is the largest Phase 3 trial ever conducted in EB.
- The FDA extended the Prescription Drug User Fee Act (“PDUFA”) goal date to allow time to review additional analyses of data previously submitted by Amryt. The submission of this additional information has been determined by the FDA to constitute a Major Amendment to the NDA, resulting in an extension of the PDUFA goal date by three months to February 28, 2022.
- Epidermolysis Bullosa (EB) is a rare and devastating group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development, which does not currently have any approved treatment.
- Global market opportunity for EB is estimated by the Company to be in excess of $1.0 billion.
28 Feb 2022 – (CTIC) CTI BioPharma Corp .
- Pipeline: CTI BioPharma | Novel Blood Cancer Therapy | Clinical Trials
- News Release: CTI BioPharma Announces Extension of FDA Review Period for Pacritinib in Myelofibrosis with Severe Thrombocytopenia
- FDA granted priority review for CTI’s NDA for patients with myelofibrosis with a PDUFA date of November 30, 2021.
- FDA has extended the review period for the New Drug Application (NDA) for pacritinib for the treatment of adult patients with intermediate or high-risk primary or secondary myelofibrosis (MF) with a baseline platelet count of <50 × 109/L. The Prescription Drug User Fee Act (PDUFA) action date has been extended by three months to February 28, 2022.
- in the course of product labeling discussions, the FDA requested additional clinical data, which was submitted to the agency on November 24, 2021
- The delay effectively caused them to lose their priority review status
- Pacritinib is a novel oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R, without inhibiting JAK1.
- NDA was accepted based on the data from the Phase 3 PERSIST-2 and PERSIST-1 and the Phase 2 PAC203 clinical trials
- In the PERSIST-2 study, in patients with severe thrombocytopenia who were treated with pacritinib 200 mg twice a day, 29% of patients had a reduction in spleen volume of at least 35%, compared to 3% of patients receiving the best available therapy, which included ruxolitinib; 23% of patients had a reduction in total symptom scores of at least 50%, compared to 13% of patients receiving the best available therapy.
- CTI BioPharma: Long And Troubled History, Finally Approaching Approval: CTI BioPharma: Long And Troubled History, Finally Approaching Approval (NASDAQ:CTIC) | Seeking Alpha
- Myelofibrosis is bone marrow cancer that results in formation of fibrous scar tissue and can lead to thrombocytopenia and anemia, weakness, fatigue and an enlarged spleen and liver.
- Within the U.S. there are approximately 21,000 patients with myelofibrosis, 7,000 of which have severe thrombocytopenia
- Current price is close to the price before the delay was announced (6 days before PDFUA), not sure how much more this will run.
… to be continued
