Biotechs are at the mercy of several make-or-break catalysts that invariably have a big impact on stocks.
A case in point was the strong upside in Biogen Inc.'s BIIB stock earlier this month when its Alzheimer's treatment Aduhelm was approved.
Details on key catalytic events, a timeline when these will likely materialize and the magnitude of impact these can have on stocks are always good knowledge to possess for an informed investor.
Among a slew of catalysts, Benzinga has culled out a handful based on screening criteria, including blockbuster potential, rare disease/breakthrough therapies and the ability to move stocks in a big way.
Novavax "Shot" In The Arm
Catalyst: EUA For COVID-19 Vaccine
Catalyst Timeline: Q3 (expected)
Novavax, Inc. NVAX is on the cusp of getting conditional approval for its coronavirus vaccine candidate, NVX-CoV2373. This could be the fruition of its labor for over a year now.
The company recently reported positive results for the U.S. leg of the Phase 3 trial of its vaccine candidate. Having already initiated a rolling emergency use authorization application submission, conditional approval could come any time soon.
The FDA is expected to schedule an Adcom meeting following which it is likely to authorize the emergency use of NVX-CoV2373. If things go well, the investigational vaccine will be the fourth to be authorized for use in the U.S.
Novavax's vaccine candidate should be on the market by the fourth quarter, competing with the established COVID-19 vaccines in a declining developed market but an expanding middle- and lower-income market globally, SVB Leerink analyst Geoffrey Porges said in a note.
"The vaccine offers refrigerator storage and distribution, relatively low manufacturing cost, similar efficacy and excellent (probably better) tolerability," he added.
Novavax shares have nearly halved from its all-time high of $331.68 in early February. Approval and subsequent commercialization could give the shares the much-needed momentum they lost temporarily due to the inordinate delay in the release of U.S. late-stage trial data.
FibroGen's Anemia Drug Faces Adcom Test
Catalyst: Adcom Meeting For Anemia Drug
Catalyst Timeline: July 15
FibroGen, Inc. FGEN shares plummeted on April 7 when the company conceded it had fudged safety data with regard to Roxadustat, which it is co-developing with AstraZeneca plc AZN.
FibroGen's shares have recovered since then, although they are still trading below the pre-disclosure levels.
Roxadustat, which is being evaluated as a treatment option for anemia associated with chronic kidney disease, will face an Adcom test on July 15, when the Cardiovascular and Renal Drug Advisory Committee will review the NDA.
The drug is already approved in Japan and China, and sales results, especially from China, are encouraging. FDA decision could ultimately follow in the third or fourth quarters.
Sanofi Under Pressure to Take Pompe Disease Treatment Past Finish Line
Catalyst: PDUFA Date For Pompe Disease Treatment
Catalyst Timeline: Aug. 16
French pharma giant Sanofi SA SNY has a key PDUFA goal date of Aug. 18 for its enzyme replacement therapy avalglucosidase alfa for Pompe disease.
FDA accepted the biologic license application for priority review in mid-November and set a PDUFA goal date of May 18. In early May, the company communicated a 3-month delay in the review period.
Pompe disease is a rare degenerative muscle disorder, affecting about 3,500 people in the U.S. Avalglucosidase alfa is designed to improve the delivery of acid alpha-glucosidase enzyme to muscle cells. If approved, the drug would offer a potential new standard of care for patients with Pompe disease.
Smaller rival Amicus Therapeutics, Inc. FOLD has a competing therapy in its pipeline, which has shown superior clinical results relative to Sanofi's. Amicus has had a pre-BLA meeting with the FDA, and it said in early May the rolling submission of the BLA is on track for completion by the second quarter.
Additionally, several gene therapy candidates are in development for Pompe's disease, either in preclinical or early-stage trials.
The pressure is now on Sanofi to get its candidate past the finish line at the earliest.
Bristol-Myers Squibb Seeking Fresh Lease of Life For Opdivo
Catalyst: Opdivo Label Expansion
Catalyst Timeline: Sept. 3
Bristol-Myers Squibb Company's BMY product portfolio is heavily weighted with oncology drugs. In the first quarter ended March 2021, the company derived more than 60% of its revenues from its oncology portfolio.
Opdivo, the company's lead cancer drug, is an immuno-oncology pitched against rival Merck & Co., Inc.'s MRK high-flying Keytruda.
Opdivo revenues fell 3% year-over-year to $1.72 billion in the first quarter compared to Keytruda's 19% growth.
Bristol-Myers Squibb is looking to extend the lifetime of its key oncology drug by expanding its label to include new indications.
The company has a key PDUFA catalyst coming up for Opdivo on Sept. 3.
The supplemental BLA accepted for priority review in late April seeks approval of Opdivo for the adjuvant treatment of patients with surgically resected, high-risk muscle-invasive urothelial carcinoma.
Enanta Chases NASH Success
Catalyst: Interim Phase 2b Readout For NASH Drug
Catalyst Timeline: Q3
Enanta Pharmaceuticals, Inc. ENTA is due to announce results from a blinded 12-week internal interim analysis on a subset of patients from the ARGON-2 Phase 2b study of EDP-305 in non-alcoholic steatohepatitis in the third quarter. This data, according to the company, is key to chart out the next steps in this program.
Immatics Due to Read Out Updated Early-stage Readout For ACTengine Cell Therapy Assets
Catalyst: Data update From the ACTengine Trials
Catalyst Timeline: 2H'2021
Immatics NV IMTX, a biopharma engaged in the development of T-cell directing cancer immunotherapies, is scheduled to release data updates from the ACTengine trials, including initial data from patients treated at the target dose.
Data from the Phase 1a dose-escalation data for IMA201 and IMA203 and Phase 1b dose-expansion data for IMA202 are due in the second half of 2021.
In March, the company released data from 10 patients showing signals of activity at doses expected to be sub-therapeutic.
"We will be looking to see responses in tumor types beyond synovial sarcoma at the higher dose levels in updates from these programs to better understand the broader potential for these assets," Chardan analyst Geulah Livshits said in a note.
Biotech Stocks In BofA's Coverage Having Second-half Catalysts
The following is the list of catalysts for biotech stocks covered by BofA Securities analyst Jason Gerberry:
Mirati Therapeutics Inc MRTX: Mirati's KRAS inhibitor adagrasib has a Phase 2 readout in second-line and above non-small cell lung cancer settings in the second half, Gerberry said in the note. This readout offers more apples-to-apples comparison with Amgen, Inc.'s AMGN sotorasib, which has already been approved, he added.
Galapagos ADR GLPG: The focus is on the Phase 2 data for the lead TOLEDO program for ulcerative colitis/rheumatoid arthritis and a TYK2 inhibitor for psoriasis, Gerberry said.
The analyst sees a 10%-15% upside to stock if positive pipeline momentum erodes negative sentiment post disappointing 2020 pipeline updates.
Jazz Pharmaceuticals PLC JAZZ: The Phase 3 Nabiximols multiple sclerosis spasticity study data is due in the second half and it will offer the first pipeline readout from the recently acquired GW Pharma, the analyst said. MS spasticity could reach peak sales of $450 million, he added.
Aerie Pharmaceuticals Inc AERI: Readout from the ongoing Phase 2b study of Aerie's TRPM8 in dry eye is a key but risky catalyst in the third quarter, Gerberry said.
Global Blood Therapeutics Inc GBT: The biopharma has a key proof-of-concept data readout for GBT021601 for the treatment of sickle cell disease, likely at the ASH meeting in December, the analyst noted.
Ionis Pharmaceuticals Inc. IONS: The company is likely to release Phase 3 topline data for tofersen in amyotrophic lateral sclerosis in fall 2021 and Phase 2b results for vupanorsen in severe hypertriglycemia in the fourth quarter, Gerberry said.
Beyondspring Inc. BYSI: Phase 3 data from the DUBLIN trial evaluating plinabulin/chemo in second-line, plus setting in NSCLC patients is on track for a mid-2021 readout, Gerberry said.
Arena Pharmaceuticals, Inc. ARNA: The biopharma is likely to release Phase 2 data for etrasimod in alopecia areata and Crohn's disease in the second half, BofA said.
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