The Week Ahead In Biotech (Dec. 27-Jan. 2): Data Releases, Regulatory Filings To Move Stocks

Biotech stocks advanced for a fifth straight week in thin holiday trading. The gains, however, were more modest amid worries concerning the emergence of a new strain of coronavirus in the U.K.

The news flow of the week comprised mixed clinical trial readouts, COVID-19 drug and vaccine announcements, a few M&A announcements and a small-ticket IPO.

Gene therapy stocks uniQure N.V. QURE and Voyager Therapeutics, Inc. VYGR came under pressure after the Food and Drug Administration placed their respective clinical programs on hold.

Ocugen Inc OCGN was some of the big news of the week, with the strength stemming from an agreement to co-develop a vaccine against SARS-CoV-2.

Here are the key catalysts for the week ahead:

PDUFA Dates

Viatris Inc VTRS, which was formed following the merger of the erstwhile generic pharma company Mylan and Pfizer Inc.'s PFE Upjohn generic unit, awaits FDA approval for its BLA for MYL-1402O, a proposed biosimilar to Avastin from Roche Holdings AG Basel ADR Common Stock RHHBY. This is being developed in partnership with India's Biocon. The PDUFA goal date is Dec. 27 (Sunday).

The FDA is scheduled to rule on Osmotica Pharmaceuticals PLC's OSMT NDA for Ontinua (arbaclofen) ER for treating spasticity in patients with multiple sclerosis. The investigational asset had faced an earlier rejection in 2016 due to manufacturing issues at clinical trial sites. Following a resubmission, the FDA has set a PDUFA action date of Dec. 29 (Tuesday).

Related Link: Moderna Says Coronavirus Vaccine's Effectiveness Will Likely Hold Against New SARS-CoV-2 Variants

Proposed Regulatory Filings

Atara Biotherapeutics Inc ATRA: Rolling biologic license approval, or BLA, submission of tab-cel for Epstein-Barr virus-positive post-transplant disorder planned to be initiated by end of 2020, with finalization of the submission planned for the third quarter of 2021.

Vertex Pharmaceuticals Incorporated VRTX: Submission of a supplemental new drug application for Trikafta is planned for the fourth quarter. Trikafta is a triple combination of elexacaftor, tezacaftor/ivacaftor and ivacaftor, in children, ages 6-11 years, with cystic fibrosis with certain mutations. It was approved for people, aged 12 years and older in late October.

Incyte Corporation INCY: A new drug application, or NDA, for ruxolitinib cream in atopic dermatitis is planned for the end of 2020.

Oyster Point Pharma Inc OYST: The company is on track to submit an NDA for OC-01 for dry eye disease in the fourth quarter.

Eyenovia Inc EYEN: An NDA submission for MicroStat (brand name: Mydcombi) for pharmacologic mydriasis is planned for the end of 2020.

Coherus Biosciences Inc CHRS: A BLA for the internally developed Humira biosimilar, CHS-1420, is expected by the end of 2020.

Stealth BioTherapeutics Corp MITO: An NDA submission for elamipretidein Barth syndrome is anticipated by year-end.

Cara Therapeutics Inc CARA: An NDA submission for Korsuva injection for the treatment of pruritus in hemodialysis patients, is planned for the fourth quarter.

Clinical Readouts — Pending Q4 Releases

RedHill Biopharma Ltd RDHL: Top-line data from the U.S. Phase 2 study of opaganib in 40 severe COVID-19 patients

Dynavax Technologies Corporation DVAX: Final immunogenicity data from the Phase 1 study of Heplisav-B in patients on hemodialysis

Immunic Inc IMUX: Phase 2 data for IMU-838 in primary sclerosing cholangitis

Altimmune Inc ALT: Results from this Phase 1b study NasoShield intranasal anthrax vaccine

Pending 2020 Year-End Releases

Catalyst Pharmaceuticals Inc CPRX: Top-line proof-of-concept data from the Phase 2 study of Firdapse in spinal muscular atrophy type-3

Aprea Therapeutics Inc APRE: Top-line data from the Phase 3 study of eprenetapopt with Bristol-Myers Squibb Co's BMY azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes patients

Bellerophon Therapeutics Inc BLPH: Top-line data from the Phase 2 dose escalation study of INOpulse in pulmonary hypertension-sarcoidosis

Merus NV MRUS: Clinical update on the Phase 1 trial of MCLA-158 in patients with solid tumors

Ultragenyx Pharmaceutical Inc RARE: Data from Cohort 4 of the Phase 1/2 study of investigational gene therapy DTX401 for glycogen storage disease Type Ia (a prophylactic steroid regimen is added to the same dose as used for Cohort 2 and 3)

IPO Quiet Period Expiries

Kinnate Biopharma Inc KNTE
Seer, Inc. SEER
Sigilon Therapeutics, Inc. SGTX
Silverback Therapeutics Inc SBTX

Related Link: Sarepta, Amicus Top Biotech Picks Ahead Of Key Binary Events: Analyst

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