The past week was busy for the biotech sector with several pipeline updates as the reporting cycle ended. Regeneron Pharmaceuticals, Inc. REGN won the FDA approval for the label expansion of its leading asthma drug Dupixent while Celgene CELG made a notable progress with its MS (multiple sclerosis) candidate.
Meanwhile, AMAG Pharmaceuticals AMAG suffered a setback with Makena.
Recap of the Week’s Top Stories:
Regeneron/Sanofi Get FDA Nod for Label Expansion: Regeneron and partner Sanofi SNY announced that the FDA has approved a label expansion of the asthma drug Dupixent (dupilumab). Dupixent, a targeted biologic therapy that inhibits signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), is now approved for the treatment of adolescent patients, aged 12-17 years with moderate-to-severe atopic dermatitis, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
The approval was based on positive results from the phase III study, which showed that treatment with Dupixent significantly improved skin lesions, reduced itching and helped clear the skin of patients. Hence, Dupixent is now approved for treating both adults and adolescents suffering atopic dermatitis.
The regulatory body also accepted the priority review of the supplemental Biologics License Application (sBLA) for Dupixent as an add-on maintenance treatment for adults with inadequately controlled severe chronic rhinosinusitis with nasal polyps (CRSwNP). A decision on the same is expected on Jun 26. Dupixent is already approved in combination with other asthma medicines for the maintenance treatment of moderate-to-severe asthma.
Celgene Submits Ozanimod’s MAA for Review in Europe: Celgene announced that it has filed a Marketing Authorization Application to the European Medicines Agency (EMA) for its pipeline candidate ozanimod. The company is seeking an approval for ozanimod, an oral, sphingosine 1-phosphate (S1P) receptor modulator, for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS). The application was based on results from the SUNBEAM and RADIANCE part B phase III, multicenter, randomized, double-blind, double-dummy, active-controlled studies. Celgene intends to submit a New Drug Application (NDA) to the FDA for the relapsing forms of MS (RMS) by the end of this month.
Celgene also announced updates on Abraxane. The FDA gave a nod to the immuno-oncology drug Tecentriq in combination with Abraxane for treating adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC), whose tumors express PD-L1, determined by an FDA-approved test. According to the company, this is the second FDA approval of a PD-1/PD-L1 antibody combined with Abraxane. However, the phase III Apact study, evaluating Abraxane in combination with gemcitabine following surgical resection (adjuvant treatment) in patients with pancreatic cancer did not achieve the primary endpoint of improvement in disease-free survival as confirmed by independent radiological review in comparison to gemcitabine alone.
Celgene currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
AMAG Slumps on Study Failure: Shares of AMAG Pharmaceuticals, AMAG plunged after the company announced top-line results from PROLONG (Progestin’s Role in Optimizing Neonatal Gestation), a study evaluating Makena in patients with a history of a prior spontaneous singleton preterm delivery. The PROLONG study did not show a statistically significant difference between the treatment and placebo arms for the co-primary endpoints. About 11% of women, who were administered Makena, delivered their babies 35 weeks into their pregnancy or earlier, which wasn’t substantially better than the 11.5% placebo participants. Further, the percentage patient population, which met the criteria for the pre-specified neonatal morbidity and mortality composite index, experienced an even smaller disparity compared with placebo patients. The PROLONG study was conducted as part of an approval commitment under the FDA’s “Subpart H” accelerated approval process.
Vertex’ Combo Study for CF Succeeds: Vertex Pharmaceuticals VRTX announced that two phase III studies evaluating its next-generation CFTR corrector, VX-445 in combination with tezacaftor and Kalydeco (ivacaftor) attained the primary endpoint of significant improvement in lung function in patients suffering from cystic fibrosis (CF). While one study is evaluating the triple combination regimen in patients with one F508del mutation and one minimal function mutation, the other study is evaluating patients with two F508del mutations.
In the study evaluating patients with one F508del mutation and one minimal function mutation, the interim data showed that the triple combination regimen led to a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline through week four of treatment as compared to placebo. The second study yielded a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week four when VX-445 was added in patients already receiving tezacaftor and ivacaftor compared with placebo plus tezacaftor and ivacaftor. Vertex believes that safety and efficacy profile observed in the study supports a potential NDA submission.
Zafgen Tanks on Discontinuation of Candidate: Shares of Zafgen ZFGN plummeted following the company’s decision to suspend plans to file an investigational new drug (IND) application for ZGN-1258. The candidate was being evaluated for rare metabolic disorders including Prader-Willi syndrome (PWS). This decision was based on a recent, unexpected finding in muscle tissue from four- and six-month long-term rodent toxicology studies. Nonclinical data showed degeneration and other anomalies in rat muscle tissue to different degrees in both vehicle and dose arms of the studies.
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: BIIB to Acquire Nightstar, Other Pipeline News & More)
What's Next in Biotech?
Stay tuned for regular pipeline updates.
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