Over the past week, mergers and acquisitions have grabbed headlines in the biotech sector. Shares of Spark Therapeutics and Clementia soared on buyout offers from Roche and Ipsen. Meanwhile, as the earnings cycle draws to an end, regular pipeline updates and data read outs are also in the news.
Recap of the Week’s Top Stories:
Spark, Clementia Gain on Merger News: Shares of gene therapy developer, Spark Therapeutics ONCE, soared significantly after Swiss pharma giant Roche Holdings announced that it will acquire the former for $4.3 billion to foray into the gene therapy space. Spark Therapeutics` lead clinical asset is SPK-8011, a novel gene therapy for the treatment of haemophilia A, which is expected to start phase III in 2019.
Shares of Clementia Pharmaceuticals Inc. CMTA surged after French biopharma company Ipsen offered to buy the former for $1.31 billion to boost its rare diseases portfolio. Per the agreement, Ipsen will pay $25.00 per share in cash upfront on completion of the transaction, for an initial aggregate consideration of $1.04 billion.
The agreement also includes deferred payments on the achievement of a future regulatory milestone in the form of a contingent value right (CVR) of $6.00 per share upon FDA acceptance of the NDA filing for Clementia’s late-stage drug candidate, palovarotene, for the treatment of multiple osteochondromas (MO), representing an additional potential payment of $263 million.
FDA Grants Priority Review to Celgene’s Revlimid: Celgene Corporation CELG announced that the FDA has granted Priority Review designation for its supplemental New Drug Application (sNDA) for the label expansion of lead drug Revlimid (lenalidomide) in combination 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.
The sNDA was based on results from the randomized, double-blind, phase III AUGMENT study, which evaluated the efficacy and safety of the investigational combination versus rituximab plus placebo in patients with relapsed/refractory follicular and marginal zone lymphoma. A Marketing Authorization Application (“MAA”) for the combination has been submitted to the European Medicines Agency (“EMA”) for the treatment of relapsed/refractory follicular and marginal zone lymphoma.
Concurrently, the company also announced updated timing for the anticipated submission of a Biologics License Application (BLA) with the FDA for pipeline candidate luspatercept in adult patients with anemia related to very low to intermediate myelodysplastic syndromes (“MDS”) with ring sideroblasts who require red blood cell transfusions, and in adult patients with anemia related to beta-thalassemia who require regular red blood cell transfusions. Celgene intends to submit the BLA in April 2019.
Celgene currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Regeneron and Partner Sanofi Disagree with Court Verdict: Regeneron REGN and Sanofi voiced their disagreement with the verdict from the U.S. District Court for the District of Delaware relating to rival Amgen’s patents. The jury upheld the validity of three of the five asserted claims of two Amgen U.S. patents covering antibodies targeting PCSK9 inhibitor, Repatha.
However, the jury agreed with Regeneron and Sanofi for two of the five asserted claims, finding they were invalid based on lack of written description. Nevertheless, Regeneron and Sanofi intend to file post-trial motions with the District Court over the next few months, seeking to overturn the jury verdict and also request a fresh trial. Earlier in the month, the District Court dismissed Amgen's claim for willful infringement.
Crispr and Vertex Began Dosing in Thalassemia Study: CRISPR Therapeutics and partner Vertex Pharmaceuticals Incorporated VRTX announced that the first patient has been treated with CTX001 in a phase I/II clinical study of patients with transfusion-dependent beta thalassemia (TDT). CTX001 is an experimental, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies.
Both the companies are also evaluating CTX001 for the treatment of severe sickle cell disease (SCD) and also announced that the first patient has been enrolled in a phase I/II clinical study of CTX001 in severe SCD in the United States. The patient is expected to be infused with CTX001 in mid-2019.
Alkermes Initiates Study, FDA Accepts NDA: Alkermes plc ALKS initiated ARTISTRY-2, a new clinical study of ALKS 4230 administered subcutaneously as monotherapy and in combination with the PD-1 inhibitor Keytruda in patients with advanced solid tumors. The study will explore the safety, tolerability and efficacy of ALKS 4230 administered subcutaneously and assess once-weekly and once-every-three-week dosing schedules.
Earlier, Alkermes and Biogen Inc. announced that the FDA has accepted for review the New Drug Application (NDA) for diroximel fumarate (BIIB098), a novel oral fumarate in development for the treatment of relapsing forms of multiple sclerosis (MS) with a target action date in the fourth quarter of 2019. Upon approval, Biogen intends to market diroximel fumarate under the brand name Vumerity, which has been conditionally accepted by the FDA and will be confirmed upon approval.
The NASDAQ Biotechnology index gained 1.7% in the past five trading sessions. Among the major biotech biggies, Biogen gained 4.2% of value. Over the past six months, shares of Regeneron have rallied 12.2% while the Gilead stock has declined 6.2%. (See the last biotech stock roundup here: Biotech Stock Roundup: ICPT Strong on NASH Drug Data, Collaborations & More)
What's Next in Biotech?
Stay tuned for more earnings news along with regular pipeline updates.
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