It was a busy week for the sector with updates from quite a few big biotechs. While Celgene (CELG - Free Report) won the FDA approval for its bone marrow cancer drug, Gilead’s (GILD - Free Report) marketing authorization application (MAA) for filgotinib for rheumatoid arthritis and Vertex’s (VRTX - Free Report) new drug application (NDA) for the triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor have been accepted by the European Medicines Agency (EMA) and the FDA, respectively. Meanwhile, Sarepta (SRPT - Free Report) plummeted as the FDA rejected its muscular dystrophy treatment.
Recap of the Week’s Most Important Stories:
Celgene Gets FDA Approval for Bone Marrow Cancer Drug: Celgene Corporation announced that the FDA has approved Inrebic (fedratinib) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. The drug provides a new, once-daily oral option to patients affected by rare bone marrow cancer. Between 16,000 and 18,500 patients are estimated to be living with myelofibrosis and 1.5 of every 100,000 people are expected to be diagnosed with the same annually. The approval was based on positive results from the JAKARTA2 study. The results of the study showed that 37% of patients treated with Inrebic 400 mg experienced a 35% or more reduction in spleen volume compared to just 1% (1 of 96) of patients who received placebo. However, Inrebic, an oral kinase inhibitor with activity against wild type and mutationally-activated Janus Associated Kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3), carries a boxed warning for serious and fatal encephalopathy, including Wernicke’s.
Vertex NDA for Triple Combination CF Treatment Accepted: Vertex Pharmaceuticals announced that the FDA has accepted its NDA for the triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor for the treatment of cystic fibrosis (CF). The agency has also granted Priority Review to the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of Mar 19, 2020. The NDA was supported by positive data from two global phase III studies in CF patients. Both studies showed statistically significant improvements in lung function, which was the primary endpoint, and in all key secondary endpoints.
Vertex currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Gilead's Application for RA Drug Accepted in Europe: Gilead Sciences and partner Galapagos announced that the EMA has accepted the MAA for JAK inhibitor candidate, filgotinib. The MAA is seeking approval of the drug as a treatment for rheumatoid arthritis (RA) in Europe. The MAA includes data from the phase III clinical program, FINCH, which comprises three studies. Data from the studies have shown that once-daily treatment with filgotinib improved clinical signs and symptoms, and achieved low disease activity and remission. The candidate also inhibited structural damage in different sub-populations of RA patients.
Regeneron Presents Data on Cholesterol Drug: Regeneron (REGN - Free Report) announced positive pivotal phase III results for evinacumab, an experimental angiopoietin-like 3 (ANGPTL3) antibody, in patients with homozygous familial hypercholesterolemia (HoFH). On average, patients entered the trial with LDL cholesterol levels of 255 mg/dL despite treatment with other lipid-lowering therapies. The study met its primary endpoint, showing that the addition of evinacumab to other lipid-lowering therapies decreased LDL cholesterol by 49% on average compared to lipid-lowering therapies. The FDA granted the Breakthrough Therapy designation to evinacumab for the treatment of hypercholesterolemia in patients with HoFH in 2017. Data will be submitted to regulatory authorities, starting with the FDA in 2020.
Nabriva Obtains FDA Approval for Pneumonia Drug: Nabriva (NBRV - Free Report) announced that the FDA has approved its NDA for both intravenous (IV) and oral formulations of Xenleta (lefamulin) to treat adult patients with community-acquired bacterial pneumonia (CABP). Notably, last December, the company submitted two NDAs to the FDA for both IV and oral formulations of lefamulin to treat CABP in the United States. The approval was based on data from two pivotal phase III studies (LEAP 1 and LEAP 2), which evaluated the safety and efficacy of Xenleta compared to moxifloxacin for treating the given patient population. The drug was generally well tolerated in both studies.
Vanda Gets CRL for Jet Lag Treatment: Vanda Pharmaceuticals (VNDA - Free Report) suffered a setback when it received a Complete Response Letter (CRL) from the FDA for the supplemental new drug application (sNDA) of Hetlioz for the treatment of jet lag disorder. Hetlioz is already approved in the United States and Europe for the treatment of non-24-hour sleep-wake disorder — a serious, rare and chronic circadian rhythm disorder. The CRL stated that the measures demonstrating improved sleep were of unclear clinical significance. The FDA made additional observations on various aspects of Vanda's sNDA.
Sarepta Plunges as FDA Denies Approval to Golodirsen: Shares of Sarepta Therapeutics plunged significantly after the FDA rejected the application for the approval of its exon 53 skipping candidate, golodirsen. The company had filed an NDA in December last year, seeking accelerated approval for the candidate as a potential treatment for Duchenne muscular dystrophy (DMD). The FDA issued a CRL against the NDA for golodirsen, citing two concerns — the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models. Sarepta stated that renal toxicity in pre-clinical models was observed in doses ten times the dose used in clinical studies. The renal toxicity issue raised in the CRL was not observed in the clinical trial — Study 4053-101 — which formed the basis for the golodirsen NDA.
The Nasdaq Biotechnology index lost 0.53% in the last five trading sessions. Among the biotech giants, Vertex gained 2.3% in the period. Over the past six months, shares of Amgen have gained 10.4%, whereas Biogen stock has declined 31%. (See the last biotech stock roundup here: Biotech Stock Roundup: Amgen’s Enbrel Patent Win, AMRN’s Setback & More)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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