It was a busy week for the biotech sector. The FDA approved Amgen’s (AMGN - Free Report) biosimilar for a leading breast cancer drug, Herceptin. Meanwhile, Alexion’s (ALXN - Free Report) Ultomiris was approved in Japan. Regeneron(REGN - Free Report) presented positive data on an investigational bispecific monoclonal antibody for lymphoma.
Recap of the Week’s Top Stories:
Amgen Gets Approval for Biosimilar Kanjinti: Amgen and its biosimilar collaboration partner, Allergan announced that the FDA has approved Kanjinti (trastuzumab-anns), their biosimilar of Roche’s blockbuster breast cancer drug, Herceptin. The biosimilar was approved for all approved indications of the reference drug, Herceptin — HER2 overexpressing adjuvant and metastatic breast cancer, and HER2 overexpressing metastatic gastric cancer or gastroesophageal junction adenocarcinoma.
Amgen has a collaboration agreement with Allergan for the worldwide development and commercialization of four oncology antibody biosimilars. Kanjinti is the second drug to receive FDA approval under this agreement.
In addition, Amgen announced five-year overall survival (OS) analysis from the single-arm, phase II BLAST study on leukemia drug, BLINCYTO. The study evaluated Blincyto in patients with minimal residual disease (MRD)-positive acute lymphoblastic leukemia (ALL). Results from the study showed a median OS of 36.5 months for Blincyto-treated patients with a median follow-up of 59.8 months. More than half of the patients who achieved a complete MRD response following the first cycle of Blincyto treatment were alive at five years.
Amgen currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.
Regeneron Presents Positive Data on Lymphoma Candidate: Regeneron announced positive early-stage data for pipeline candidate, REGN1979. The data will be presented at the 24th Congress of the European Hematology Association (EHA). REGN1979, an investigational bispecific monoclonal antibody, is being evaluated in patients with relapsed or refractory (R/R) B-cell non-Hodgkin lymphoma (B-NHL). The data also includes patients with R/R diffuse large B-cell lymphoma (DLBCL), who had progressed after CAR-T therapy. The primary objective was to assess the safety, tolerability and dose-limiting toxicities of the candidate. High response rates observed with REGN1979 in both relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma were encouraging. Moreover, two patients, who failed CAR-T therapy and received REGN1979 80 mg, achieved complete response.
The R/R follicular lymphoma (FL) grades 1 to 3a treatment arm showed an overall response rate of 93% (13 of 14 patients) in patients, who received doses of 5 mg or more, with a complete response rate of 71% (10 of 14 patients). In DLBCL patients treated with REGN1979 80 mg to 160 mg, an overall response rate of 57% was observed. In R/R DLBCL patients, whose disease progressed after CD-19 directed CAR-T therapy, two out of four achieved a complete response. The company will initiate a phase II program later in the month for a potential registration, and proactively evaluate active REGN1979 doses in indolent and aggressive non-Hodgkin lymphoma.
Alexion’s Ultomiris Gets Approval in Japan: Alexion announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor administered every eight weeks, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The drug is already approved in the United States. The approval was based on comprehensive results from two phase III studies, which included 441 patients who had either never been treated with a complement inhibitor before, or who had been stable on Alexion’s lead drug Soliris. Results showed that the efficacy of Ultomiris administered every eight weeks was non-inferior to the efficacy of SOLIRIS administered every two weeks on all 11 endpoints.
Earlier, Alexion announced positive, long-term data from the extension of the phase III study on Ultomiris and Soliris in complement inhibitor-naïve, adult patients with PNH. The data demonstrated that Ultomiris administered every eight weeks provided consistent efficacy and safety through 52 weeks, with no cases of breakthrough hemolysis associated with incomplete C5 complement inhibition. The data was presented at the Annual Congress of the European Hematology Association. Ultomirus was studied in the largest-ever phase III program in PNH.
Savara Crashes on Late-Stage Study Failure: Savara (SVRA - Free Report) crashed after it announced that its lead pipeline candidate, Molgradex, failed to meet the primary endpoint in the pivotal phase III study — IMPALA — evaluating it in autoimmune alveolar pulmonary proteinosis (aPAP), a rare lung disorder. The IMPALA study evaluated Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), in patients with aPAP compared to placebo for improvement in alveolar-arterial oxygen gradient (A-aDO2) as its primary endpoint. The study evaluated two dose administrations — once daily continuous administration of and once daily in seven-day intermittent cycles — of 300 µg Molgradex over 24 weeks. Data from the study demonstrated average improvement of 12.1 mmHg in patients who were administered Molgradex once daily continuously compared with an improvement of 8.8 mmHg for placebo. However, the treatment difference of 4.6 mmHg failed to meet the primary endpoint.
Molgradex also failed to achieve improvement of statistical significance in two key secondary endpoints – the six-minute walk distance (6MWD) and requirement for whole lung lavage (“WLL”).
Abeona Gets FDA Fast Track Designation for Gene Therapy: Abeona Therapeutics (ABEO - Free Report) announced that the FDA has granted Fast Track designation to its gene therapy ABO-202 program. ABO-202 is administered as a one-time adeno-associated virus 9 (AAV9) gene therapy that delivers a functional copy of the PPT1 gene to cells of the central nervous system and peripheral organs. It is being evaluated for CLN1 disease, which is also known as Infantile Neuronal Ceroid Lipofuscinosis or infantile Batten disease. The company is planning to initiate a phase I/II study evaluating ABO-202 in patients with CLN1 disease and will provide guidance on the timing of the study later in the year.
The NASDAQ Biotechnology index gained 4.67% in the last five trading sessions. Among the major biotech giants, Alexion gained 4.46% in the period. Over the past six months, shares of Celgene have surged 45.81% whereas Biogen stock has lost 20.95%. (See the last biotech stock roundup here: Biotech Stock Roundup: Celgene Submits MS Drug to FDA, INSY & CBAY Crash)
What's Next in Biotech?
Stay tuned for more pipeline and regulatory updates.
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