Biotech Stock Roundup: MYOV & KRTX Surge, Pipeline Updates From VRTX, CELG & More
Zacks Investment Research | Nov 19, 2019 09:31PM ET
It was a busy week for the biotech sector with updates from quite a few companies. Vertex Pharmaceuticals (NASDAQ:VRTX) announced positive data on its gene-editing therapy, while Celgene (NASDAQ: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 (NASDAQ:KRTX) and Myovant (NYSE: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 (NASDAQ: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 (NASDAQ:ALKS) 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 (
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