It was a busy week for the biotech sector with updates from quite a few companies. While Intercept Pharmaceuticals ICPT obtained Priority Review for its New Drug Application (NDA) for NASH drug, CymaBay CBAY plunged on termination of NASH study. Amgen AMGN upped its guidance after buying Otezla whereas Global Blood Therapeutics GBT obtained FDA approval for its SCD drug. Meanwhile, ChemoCentryx CCXI soared after it announced positive data from a late-stage study on its rare disease candidate.
Recap of the Week’s Most Important Stories:
Intercept NDA for NASK Drug Gets Priority Review: Intercept Pharmaceuticals announced that the FDA has accepted its New Drug Application (NDA) for obeticholic acid (OCA) seeking accelerated nod for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH). The agency also granted a Priority Review to the same. Notably, the regulatory authority generally grants priority review to drugs with potential to treat a serious condition and if approved, would provide a significant improvement in safety or effectiveness. The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of Mar 26, 2020, for the NDA. The agency indicated that it currently plans to hold an advisory committee meeting to discuss the application. However, the date for the meet has not been finalized and the timeline for the NDA review is subject to change.
CymaBay Plunges on Termination of NASH Study: Shares of CymaBay plummeted significantly after the company announced that it was terminating its phase IIb study of seladelpar in patients with NASH and its recently initiated phase II study of the drug in patients with primary sclerosing cholangitis (PSC). Moreover, it is putting a hold on all studies of seladelpar in patients with primary biliary cholangitis (PBC). The decision to terminate these studies was based on initial histological findings observed in the phase IIb study of seladelpar for NASH after data showed some patients experiencing some liver damage. These planned, blinded, histological assessments of the first tranche of liver biopsies in the study showed atypical histological findings including histology characterized as an interface hepatitis presentation with or without biliary injury.
Alexion Gets Label Expansion Nod for Soliris in Japan: Alexion Pharmaceuticals’ ALXN Soliris received Japan’s Ministry of Health, Labour and Welfare’s (MHLW) approval for a label expansion. The drug is now approved for neuromyelitis optica spectrum disorder (NMOSD) in adult patients, who are anti-aquaporin-4 (AQP4) antibody positive. Soliris is the first and the only approved medication for this indication in Japan. The nod was based on comprehensive results from the phase III, randomized, double-blind, placebo-controlled PREVENT study. Soliris was approved for the treatment of NMOSD in adult patients, who are anti-AQP4 antibody-positive, by the FDA in June 2019 and the European Commission (EC) in August.
Alexion currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Amgen Buys Otezla, Lifts Outlook: Amgen announced that it has completed the previously-declared acquisition of Celgene’s blockbuster psoriasis drug Otezla, which the latter had to divest in order to complete its merger with Bristol-Myers. Amgen had to pay Celgene $13.4 billion in cash or approximately $11.2 billion net of anticipated future cash tax benefits for Otezla and certain related assets and liabilities. Otezla is approved to treat psoriasis, psoriatic arthritis and oral ulcers associated with Behçet's disease
Following this buyout, the company raised its overall guidance for 2019. Amgen now expects total revenues in the range of $23.1-$23.3 billion, previously expected within $22.8-$23.0 billion. Adjusted EPS is expected in the band of $14.5 -$14.7 compared with the previous range of $14.20-14.45.
Biogen Completes Enrollment in Retinal Disorder Study: Biogen BIIB announced that it has completed enrollment in the global phase III STAR study on its gene therapy, timrepigene emparvovec, currently being developed to treat choroideremia (CHM). CHM is a rare, inherited retinal disorder that causes progressive loss of vision due to degeneration of the choroid and retina. This mainly affects males. Timrepigene emparvovec (previously BIIB111) was added to Biogen’s portfolio in June as part of its acquisition of Nightstar Therapeutics. The STAR study enrolled 170 adult males with CHM. The primary endpoint of the study is the proportion of patients who will experience an improvement of at least 15 letters from baseline in best corrected visual acuity (BCVA) at 12 months post the treatment, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity protocol.
Global Blood Gets FDA Approval for SCD Drug: Global Blood Therapeutics announced that the FDA has approved its lead product candidate Oxbryta (voxelotor) as an oral, once-daily treatment for sickle cell disease (SCD) in patients aged 12 years and above. The approval comes almost three months ahead of the scheduled action date of Feb 26, 2020. SCD is a chronic, inherited blood disorder that affects hemoglobin levels. Following this nod, Oxbryta became the first approved therapy that directly inhibits sickle hemoglobin polymerization, a major cause for SCD.
ChemoCentryx Gains on Study Results: ChemoCentryx surged significantly after it announced encouraging top-line data from the phase III study ADVOCATE evaluating its orally-administered selective complement 5a receptor inhibitor, avacopan. The pivotal study evaluated avacopan in patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA vasculitis), a rare disease affecting small blood vessels. Data from the study showed that patients in the study treated with avacopan achieved both primary endpoints of clinical remission at weeks 26 and 52. Clinical remission was statistically superior in the avacopan arm compared to SOC at 52 weeks. The company is planning to file a regulatory application seeking approval for avacopan to treat ANCA vasculitis in the United States and Europe next year.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index gained 4.44% in the past five trading sessions. Among the biotech giants, Biogen gained 6.15% in the period. Over the past six months, shares of Amgen have gained 38.73% whereas the Alexion stock has declined 10.61%. (See the last biotech stock roundup here (See the last biotech stock roundup here: Biotech Stock Roundup: MYOV & KRTX Surge, Pipeline Updates From VRTX, CELG & More)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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Global Blood Therapeutics, Inc. (GBT) : Free Stock Analysis Report
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