It was a busy week for the biotech sector with updates from quite a few companies. Vertex Pharmaceuticals VRTX announced positive data on its gene-editing therapy, while Celgene CELG obtained a positive Committee for Medicinal Products for Human Use (CHMP) opinion for Revlimid in combination with Rituxan for the treatment of adult patients with previously treated follicular lymphoma (FL). Meanwhile, both Karuna Therapeutics KRTX and Myovant MYOV surged on positive pipeline updates.
Recap of the Week’s Most Important Stories:
Vertex & CRISPR Announce Data on Gene-Editing Therapy: Vertex Pharmaceuticals and CRISPR Therapeutics CRSP announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy, CTX001, in the ongoing phase I/II clinical trials. One patient with transfusion-dependent beta thalassemia (TDT) received CTX001 in the first quarter and data for this patient reflect nine months of safety and efficacy follow-up. One patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for the same reflect four months of safety and efficacy follow-up. Data showed that the beta-thalassemia patient is transfusion independent with a total hemoglobin level of 11.9 g/dL and 10.1 g/dL fetal hemoglobin at nine months after the CTX001 infusion. The SCD patient is free of vaso-occlusive crises with a total hemoglobin level of 11.3 g/dL and 46.6% fetal hemoglobin at four months after the CTX001 infusion. Meanwhile, these studies are ongoing and patients will be followed for approximately two years following the infusion.
Alkermes to Acquire Rodin Therapeutics: Alkermes plc ALKS announced that it will acquire privately-held Rodin Therapeutics for an upfront cash payment of $100 million and up to $850 million in milestone payments. The deal is expected to close by the end of November. The deal will enable Alkermes to expand its central nervous system (CNS) development efforts into a wide range of neurodegenerative disorders through epigenetic control of synaptogenesis. Rodin is developing therapeutics for synaptopathies by designing molecules that target specific histone deacetylase (HDAC) complexes. Alkermes plans to advance investigational new drug (IND)-enabling activities for lead preclinical assets in the Rodin development candidate portfolio.
Alkermes currently has a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Celgene Gets Positive CHMP Opinion for Revlimid-Rituxan Combo: Celgene announced that the European Medicines Agency's CHMP has adopted a positive opinion, recommending the approval of Revlimid (lenalidomide) in combination with Roche Holding AG’s Rituxan (rituximab) (anti-CD20 antibody) (R²) for the treatment of adult patients with previously treated FL (Grade 1-3a). The CHMP positive opinion was mainly supported by results from the randomized, multi-center, double-blind, phase III AUGMENT study, which evaluated the efficacy and safety of the R² combination versus Rituxan plus placebo in patients with previously treated FL.
Meanwhile, Bristol-Myers Squibb Company announced that the U.S. Federal Trade Commission (FTC) has permitted to close the impending merger with Celgene.
VistaGen's Major Depressive Disorder Drug Disappoints in Phase II: VistaGen Therapeutics VTGN announced disappointing results from a mid-stage study, ELEVATE, on pipeline candidate, AV-101. The candidate, an NMDA (N-methyl-D-aspartate) receptor glycine site antagonist, was evaluated as an adjunctive treatment for major depressive disorder (MDD) patients, who had an inadequate response to a stable dose of standard antidepressant therapy (either a selective serotonin reuptake inhibitor or a serotonin norepinephrine reuptake inhibitor). The study was a phase II, double-blind, placebo-controlled, multi-center, sequential parallel comparison design study that evaluated the safety, tolerability and efficacy of AV-101 as an adjunctive treatment in MDD patients. The data showed that the AV-101 treatment arm did not differentiate from placebo on the primary endpoint (change in the Montgomery-Åsberg Depression Rating Scale (MADRS-10) total score compared to baseline).
Karuna Surges on Positive Results from Schizophrenia Treatment: Shares of clinical-stage biopharmaceutical company Karuna Therapeutics soared significantly after it announced positive results from a phase II study of KarXT for the treatment of acute psychosis in patients with schizophrenia. Data from the study showed that KarXT demonstrated a statistically significant and clinically meaningful 11.6 point mean reduction in total Positive and Negative Syndrome Scale (PANSS) score compared to placebo. The data also demonstrated good overall tolerability. A statistically significant reduction in the secondary endpoints of PANSS-Positive and PANSS-Negative scores were also observed. Moreover, KarXT was well tolerated in the study with similar discontinuation rates between KarXT (20%) and placebo (21%). The study data support advancing KarXT to phase III and continuing development in other neuro disorders.
Myovant Up on Prostate Cancer Data: Myovant Sciences’ shares skyrocketed after it announced that the phase III HERO study of once-daily, oral relugolix (120 mg) met the primary efficacy endpoint and all six key secondary endpoints in men with advanced prostate cancer. In the study, 96.7% of men achieved sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks. Five key secondary endpoints demonstrated superiority to leuprolide acetate, including rapid suppression of testosterone at day 4 and day 15, profound suppression of testosterone at day 15, rapid suppression of prostate-specific antigen (PSA) at day 15 and suppression of follicle-stimulating hormone (FSH) at week 24. Moreover, relugolix demonstrated non-inferiority to leuprolide acetate on sustained testosterone suppression through 48 weeks. The positive results support a New Drug Application (NDA) submission to the FDA in the second quarter of 2020 and future regulatory submissions in Europe and Japan.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index gained 2.63% in the last five trading sessions. Among the biotech giants, Vertex gained 5.46% in the period. Over the past six months, shares of Amgen have gained 36.97%, whereas the Alexion stock has declined 12.13%. (See the last biotech stock roundup here: Biotech Stock Roundup: SLDB Plunges, CELG Gets FDA Nod for Reblozyl & Other Updates)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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Celgene Corporation (CELG) : Free Stock Analysis Report
Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
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