Mergers and acquisitions continue to grab headlines in the biotech sector. Shares of Nightstar Therapeutics NITE soared after Biogen BIIB announced an agreement to acquire the former. Meanwhile, as the reporting cycle draws to a close, regular pipeline updates and data read-outs are also in news. Celgene Corporation CELG obtained a priority review for its drugs from the FDA while CHMP gave a positive opinion on the label expansion of Regeneron’s Dupixent.
Recap of the Week’s Top Stories:
Biogen to Acquire Gene Therapy Company Nightstar: Biogen announced that it has entered into an agreement to acquire London based clinical-stage gene-therapy company, Nightstar Therapeutics. Per the deal, Biogen will pay $25.50 in cash for each share of Nightstar or approximately $800 million. Biogen expects to complete the buyout by mid-2019.
Nightstar is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. The company has two potentially first-in-class mid- to late-stage candidates and other preclinical programs. The company’s lead asset, NSR-REP1, is being evaluated for the treatment of choroideremia (CHM) in an ongoing phase III STAR study. Data from the same is expected in the second half of 2020. The second candidate is NSR-RPGR, currently evaluated for the treatment of X-linked retinitis pigmentosa (XLRP).
The phase I/II results from the dose escalation portion of the XIRIUS investigation on NSR-RPGR demonstrated an increase in central retinal sensitivity. The phase II/III dose expansion part of the XIRIUS analysis is currently underway. The preclinical pipeline includes NSR-ABCA4 for Stargardt disease and potential programs targeting Best vitelliform macular dystrophy (Best disease) and other genetic forms of retinitis pigmentosa. The transaction is expected to accelerate Biogen’s foray into ophthalmology space.
Celgene Gets Priority Review for Myelofibrosis Drug: Celgene announced that the FDA has accepted its New Drug Application (NDA) for fedratinib and granted a priority review to the same. Fedratinib, a highly selective JAK2 inhibitor, is being evaluated for treating patients with myelofibrosis. The FDA has set an action date of Sep 3, 2019. The NDA was based on encouraging outcomes from a randomized, placebo-controlled, phase III probe (JAKARTA) on patients with primary or secondary myelofibrosis and a single-arm, open-label phase 2 evaluation (JAKARTA2) of patients with primary or secondary myelofibrosis, previously exposed to Jakafi. The candidate also enjoys an Orphan Drug designation for secondary and primary myelofibrosis. The company also plans to evaluate fedratinib in combination with luspatercept.
The FDA also granted a priority review designation to Celgene’s supplemental New Drug Application (sNDA) for its lead drug — Revlimid — combined with rituximab (R²) for the treatment of patients with previously treated follicular and marginal zone lymphoma. The FDA has set its action date as Jun 27, 2019.
Celgene currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
CHMP Provides Positive Opinion on Label Expansion of Dupixent: Regeneron Pharmaceuticals, Inc. REGN and partner Sanofi announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive response to Dupixent (dupilumab) for asthma. The CHMP recommended Dupixent’s approval for use in adults and adolescents aged 12 years or older as an add-on maintenance treatment for severe asthma with type 2 inflammation, characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO). Such patients are inadequately controlled with high dose inhaled corticosteroid plus another medicinal product for maintenance therapy.
A decision from the European Commission is expected in the coming months. Dupixent is already approved in the European Union for administering to adults with moderate-to-severe atopic dermatitis, who are candidates for systemic therapy. A potential nod for asthma will broaden the sales potential of the drug. Last October, the FDA also approved Dupixent as an add-on maintenance therapy for patients with moderate-to-severe asthma, aged 12 years or above with an eosinophilic phenotype or oral corticosteroid-dependent asthma. Meanwhile, the FDA has granted a priority review to the sBLA for atopic dermatitis in adolescent patients and set a target action date of Mar 11, 2019.
Immunogen Crashes as Late-Stage Study Disappoints: Shares of ImmunoGen IMGN plunged significantly after the company performed dismally in a late-stage study on its ovarian cancer candidate. The FORWARD 1 study evaluated the efficacy of mirvetuximab soravtansine as a single-agent therapy compared with chemotherapy for treating patients with platinum-resistant ovarian cancer, whose tumors express high or medium levels of FR alpha and who have received up to three prior treatment regimens.
The phase III study failed to meet the primary endpoint of progression-free survival (PFS). It did not achieve the PFS goal in the entire study population apart from the pre-specified subset of patients with high FRα expression. Although the confirmed overall response rate was higher for mirvetuximab soravtansine than for chemotherapy, there was no significant difference in PFS. The company will further assess data from the FORWARD I study to determine potential future steps with a monotherapy approach.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology index inched up 0.8% in the past five trading sessions. Among the major biotech biggies, Regeneron gained 2.6% of value. Over the past six months, shares of Alexion have rallied 14.8% while the Gilead stock has declined 10.2%. (See the last biotech stock roundup here: Biotech Stock Roundup: ONCE, CMTA Soar on Merger News, Other Pipeline Updates)
What's Next in Biotech?
Stay tuned for regular pipeline updates.
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