Biogen's 2nd Alzheimer's Antibody, BeiGene's Cancer Drug Label Expansion, Adcom Test For Regeneron And More: January PDUFA Catalysts For Biotech Investors

Zinger Key Points
  • PDUFA date is a key binary catalyst for biotech stocks, having the potential to create wild swings.
  • It is the date by which the FDA is required to announce its verdict regarding the approvability/non-approvability of a drug.

Biopharma stocks were not immune to the broader market downturn seen for much of 2022. The iShares Biotechnology ETF IBB, which is heavily weighted with large-cap biotech companies, has lost 13.2% compared to a steeper 27.8% pullback by the SPDR S&P Biotech XBI.

What Happened: This clearly suggests that the sell-off is more acute in the small-cap space. More importantly, the declines of the year came on top of sharp losses experienced by the sector in 2021.

See Also: Best Biotech Socks Right Now

The year was a forgettable one from the fundamental perspective too. Drug innovation dwindled, as evident from the drop in new molecular entity approvals from 50 in 2021 to 34 in 2022. The alleviation of the COVID-19 situation did not bode well for vaccine manufacturers and their stocks pulled back in line with the reduced forecast for vaccine sales.

December proved to be a mixed month for FDA approvals. Mirati Therapeutics Inc.’s MRTX adagrasib, a KRAS G12C inhibitor, received the nod for treating a certain type of lung cancer. Gilead Sciences Inc. GILD also succeeded in pushing its long-acting AIDS drug past the finish line.

On the flip side, Coherus Biosciences Inc.’s CHRS toripalimab approval has been delayed and Cytokinetics Inc. CYTK was hit by a negative FDA panel verdict with respect to its heart failure drug.

Here are the major PDUFA dates for the upcoming month:

Will MediWound’s Burn Treatment Emerge Unscathed On Second Try?

Company: MediWound Ltd. MDWD/Vericel Corp. VCEL
Type of Application: biologic license application
Candidate: NexoBrid
Indication: thermal burns
Date: Jan. 1

Israel-based biotech MediWound’s NexoBrid resubmission was accepted for review in early August for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company had out-licensed the North American commercial rights for the investigational therapy exclusively to Vericel.

MediWound’s original application was handed down a complete response letter in late-June 2021, citing issues with chemistry, manufacturing and controls.

NexoBrid, a concentrate of proteolytic enzymes enriched in Bromelain, is a topically administered biological product that enzymatically removes non-viable burn tissue, or eschar, in patients with deep partial and full-thickness thermal burns within four hours of application without harming viable tissue.

NexoBrid is approved in the European Union and other international markets.

Biogen’s Second AD Therapy Awaits FDA Clearance

Company: Biogen Inc. BIIB/Eisai Co. Ltd. ESALY
Type of Application: BLA
Candidate: lecanemab
Indication: mild Alzheimer’s disease
Date: Jan. 6

Lecanemab is Biogen/Eisai’s investigational anti-amyloid beta protofibril antibody being evaluated for mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease. This investigational asset assumes importance because of a lack of success with the combo’s first-approved Alzheimer's treatment Aduhelm.

A draft report released by the Institute for Clinical and Economic Review showed that the treatment candidate will likely be priced between $8,500 and $20,600 annually, making it cheaper than its predecessor therapy Aduhelm.

Detailed data released from a late-stage study of lecanemab in late November showed that it succeeded in slowing cognitive decline but might carry the risk of side effects for certain patients. Two deaths have been reported in a trial extension study.

Can Phathom's Vonoprazan Snag Nod For A Second Indication?

Company: Phathom Pharmaceuticals, Inc. PHAT
Type of Application: NDA
Candidate: vonoprazan
Indication: esophagitis and heartburn
Date: Jan.11

Phathom’s vonoprazan application was accepted for review in late-May 2022 as a treatment option for adults for the healing of erosive esophagitis (EE), or inflammation of the esophagus, and relief of heartburn, as well as maintenance of healing of EE and heartburn relief.

Vonoprazan is an orally administered investigational potassium-competitive acid blocker. It was approved for treating Helicobacter pylori infection in May.

EE, according to Phathom, is a major type of gastroesophageal disease affecting about 20 million people in the U.S. If not adequately treated, EE may progress to cancer.

Seagan Knocks FDA Door For Colorectal Cancer Drug Nod

Company: Seagen Inc. SGEN
Type of Application: supplemental NDA
Candidate: Tukysa in combination with trastuzumab
Indication: colorectal cancer
Date: Jan.19

The Tukysa-trastuzumab combo therapy is being evaluated for adult patients with HER2-positive colorectal cancer, who have received at least one prior treatment regimen. The application was made based on the results of a Phase 2 study dubbed MOUNTAINEER. There are no currently no-FDA approved therapies for metastatic colorectal cancer that specifically target HER2.

BeiGene Seeks Label Expansion For Blood Cancer Drug

Company: BeiGene Ltd. BGNE
Type of Application: sNDA
Candidate: Brukinsa
Indication: blood cancer
Date: Jan.20

BeiGene’s Brukinsa is currently under review for chronic lymphocytic leukemia and small lymphocytic lymphoma. Incidentally, the company communicated a three-month extension to the review period in mid-June, citing the additional time FDA required to review the application for expanding the Brukinsa label, following which a new PDUFA date was issued.

Brukinsa is a small-molecule compound discovered by BeiGene and is currently being evaluated globally in clinical programs, as monotherapy and in combination with other therapies, to treat B-cell malignancies. It has currently been approved for treating mantle cell lymphoma and relapsed marginal zone lymphoma. It fetched revenues of $155.5 million in the third quarter, a 136% year-over-year growth.

Merck's Wonder Cancer Drug On Track For Another Approval?

Company: Merck & Co, Inc. MRK
Type of Application: sBLA
Candidate: Keytruda
Indication: lung cancer
Date: Jan.29

Merck’s regulatory application for Keytruda as an adjuvant treatment of patients with stage 1b, 2 or 3a non-small cell lung cancer following complete surgical resection was accepted for review in mid-June 2022.

The application was supported by data from the Phase 3 KEYNOTE-091 trial, which demonstrated a significant improvement in disease-free survival for patients regardless of PD-L-1 expression. However, another co-primary endpoint of disease-free survival in patients whose tumors express PD-L1 did not reach statistical significance.

Adcom Meetings

FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee is scheduled to meet on Jan. 9 to discuss Regeneron Pharmaceuticals Inc.’s REGN sBLA for aflibercept solution for intravitreal injection. The supplement was submitted in response to FDA’s pediatric written request for studies of aflibercept in the treatment of retinopathy of prematurity.

The Antimicrobial Drug Advisory Committee of the FDA will discuss on Jan. 24 Cidara Therapeutics Inc.’s CDTX NDA for rezafungin lyophilized powder for injection for the treatment of candidemia and invasive candidiasis in adults.

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