It was a busy week for the biotech sector with quite a few updates. While coronavirus treatment updates continue to grab the spotlight, other regulatory and pipeline updates were also in focus this week.
Recap of the Week’s Most Important Stories:
Vaxart Surges on COVID-19 Vaccine Selection for OWS: Shares of clinical-stage biotechnology company, Vaxart, Inc. VXRT, surged after it announced that its oral COVID-19 vaccine has been selected to participate in a non-human primate (NHP) challenge study. The study is organized and funded by Operation Warp Speed (OWS), a new national program, to provide substantial quantities of safe, effective vaccines for Americans by January 2021. The study is designed to demonstrate the efficacy of Vaxart’s oral COVID-19 vaccine candidate. Per the press release, this is the first oral COVID-19 vaccine, which will be tested by OWS in non-human primates.
Gilead Announces Pricing for Coronavirus Drug: Gilead Sciences, Inc. GILD announced pricing for experimental coronavirus drug, remdesivir. The company set a price for governments of developed countries at $390 per vial, which equates to $2,340 per patient as the vast majority of patients are expected to receive a five-day treatment course using six vials of remdesivir. In the United States, the same government price of $390 per vial will apply. However, due to the way the U.S. system is set up and the discounts expected by government healthcare programs, the price for private insurance companies will be $520 per vial in the country. For the developing countries, Gilead has entered into agreements with generic manufacturers to deliver treatment at a substantially lower cost. The company expects its investment in the development and manufacture of remdesivir to exceed $1 billion by the end of 2020. The pricing was lower than most estimates.
Earlier, remdesivir was recommended for approval by the European Medicines Agency (EMA)’s human medicines committee (CHMP). The CHMP has recommended granting a conditional marketing authorization to the drug under the brand name, Veklury, for the treatment of COVID-19 in adults and adolescents from 12 years of age with pneumonia who require supplemental oxygen.
Gilead currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Intercept Plunges on CRL for NASH Candidate: Intercept Pharmaceuticals ICPT announced the receipt of a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for obeticholic acid (OCA) seeking approval for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH).
Based on the data reviewed by the FDA, the agency issued a CRL as it determined that the predicted benefit of OCA based on a surrogate histopathologic endpoint remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH. Moreover, the agency recommended Intercept to submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study in support of potential accelerated approval and continue the long-term outcomes phase of the study.
The news was disappointing as Intercept’s OCA was a frontrunner in receiving approval for the treatment of NASH. Shares plunged on the same.
Alexion’s Ultomiris Approved in Europe: Alexion Pharmaceuticals, Inc. ALXN announced that the European Commission has approved the label expansion of its long-acting C5 complement inhibitor, Ultomiris, for a rare disease. The drug, administered every eight weeks, is approved for the treatment of adults and children with a bodyweight of 10 kg or above suffering from atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received its other drug, Soliris (eculizumab), for at least three months and have evidence of response to Soliris. The European Commission’s approval was based on data from two global, single-arm, open-label studies of Ultomiris — one in adults and another in children.
Separately, Alexion also announced positive top-line results from a late-stage study on Ultomiris. The phase III study evaluated subcutaneous (SC) formulation of Ultomiris in adults with paroxysmal nocturnal hemoglobinuria (PNH), a serious, ultra-rare blood disorder. The ongoing study met its primary objective of pharmacokinetic (PK)-based non-inferiority of Ultomiris SC versus the intravenous (IV) formulation of Ultomiris at day 71. The study is ongoing to assess the secondary endpoints, including safety, immunogenicity and various PK/PD, quality of life, device performance and efficacy measures.
Alexion intends to file for the approval of the SC formulation of the drug and device combination in PNH and aHUS indications in the United States and the EU, once this study is completed and 12-month safety data are collected.
Vertex Gets CHMP Opinion for CF Regimen: Vertex Pharmaceuticals Incorporated VRTX announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion recommending approval for its triple combination cystic fibrosis regimen, Kaftrio. The company is seeking approval in Europe for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis in patients aged 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in their CFTR gene. Though the European Commission is not bound by the opinion of the CHMP, it usually seconds CHMP recommendations in its final decision for approval in Europe. The CHMP recommendation was supported by positive data from two global phase III studies in CF patients.
Zogenix Gets FDA Approval for Rare Disease Drug: Shares of Zogenix, Inc. ZGNX were up after it announced the receipt of FDA approval for Fintepla (fenfluramine) oral solution, CIV for the treatment of seizures associated with Dravet syndrome in patients 2 years of age and older. Dravet syndrome is a rare childhood-onset epilepsy marked by frequent and severe treatment-resistant seizures. Fintepla significantly and substantially reduced convulsive seizure frequency in patients whose seizures were not adequately controlled on other medications, as observed in two phase III placebo-controlled clinical studies.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index lost 1.40% in the last five trading sessions. Among the biotech giants, Gilead gained 2.52% during this period. Over the past six months, shares of Regeneron have gained 66.89%. (See the last biotech stock roundup here: Biotech Stock Roundup: REGN Begins Coronavirus Study, VRTX, BIIB Give Updates)
What's Next in Biotech?
Stay tuned for more pipeline updates, with a focus on treatments for the novel COVID-19.
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