Biotech Stock Roundup: IRON, VERV Down on Study Updates, BMYs Drug News & More

Pipeline and regulatory updates continue to be in focus in the biotech sector. While nothing much came out of bigwigs apart from a few, shares of clinical-stage biotechs like Disc Medicine, Inc. IRON and Verve Therapeutics VERV plunged on disappointing updates.

Recap of the Week's Most Important Stories:

Updates From BMY: Bristol Myers Squibb BMY announced that the European Commission has approved a label expansion of Reblozyl (luspatercept). The EC expanded the drug's label to include the first-line treatment of adult patients with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS). The approval of Reblozyl covers all European Union EU member states.

The latest approval is based on results of the late-stage COMMANDS study, wherein Reblozyl demonstrated superior efficacy compared to epoetin alfa in the study's primary endpoint of concurrent red blood cell transfusion independence and hemoglobin increase.

Safety results were consistent with previous MDS studies and in line with expected symptoms in this patient population. Reblozyl is already indicated in the EU for the treatment of adult patients with transfusion-dependent anemia due to very low, low and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. It is also indicated for the treatment of adult patients with anemia, associated with transfusion-dependent and non-transfusion-dependent beta-thalassemia.

Earlier, Bristol Myers announced that a late-stage study on orally-administered Zeposia (ozanimod) in Crohn's disease (CD) indication failed to achieve its primary endpoint. This late-stage study was the first of the two induction studies in the phase III YELLOWSTONE clinical program, which evaluated Zeposia in 600 adult patients with moderate to severe active CD over a 12-week treatment period. Initial results from the study showed that treatment with Zeposia failed to meet the primary endpoint of clinical remission at week 12.

BMY currently has a Zacks Rank #3 (Hold).

IRON Down on Study Results:  Shares of Disc Medicine, Inc., a clinical-stage biopharmaceutical company, plunged after the company announced mixed results from a phase II study of lead pipeline candidate, bitopertin, in patients with erythropoietic protoporphyria EPP. The AURORA study is a randomized, double-blind, placebo-controlled phase II study that enrolled 75 adult subjects with EPP. The participants were randomized 1:1:1 to receive 20 mg of bitopertin, 60 mg of bitopertin, or placebo once daily for 17 weeks.

Treatment with bitopertin resulted in statistically significant reductions in toxic metabolite protoporphyrin IX (PPIX), the primary endpoint, and significant improvements in the rate of phototoxic reactions with pain and the Patient Global Impression of Change. The 60 mg dose reached statistical significance compared to placebo. The 20mg cohort achieved a 21.6% reduction in whole blood PPIX levels and the 60mg dose achieved a 40.7% reduction in whole blood PPIX levels. The placebo group had a mean increase of 8.0%.

However, the study did not achieve its key secondary endpoint of cumulative time in sunlight on days without pain. Results showed that biopterin-treated patients recorded a mean of 175.1 hours at 20 mg and 153.1 hours at 60 mg compared with 133.9 hours for the placebo of cumulative total time in sunlight between 10 am and 6 pm on days without pain, as observed over the four-month treatment period. While the magnitude of the improvement in the biopterin-treated patients was comparable to that observed in the BEACON study, the benefit in the placebo arm in the AURORA trial was greater than expected.

Disc Medicine will conduct an analysis of its final data set and work with investigators, regulators and patient advocacy groups to define the optimal registrational endpoints moving forward.

VERV Down on Study Pause:  Verve Therapeutics's shares plunged after the company announced updates from the Heart-1 phase Ib study on investigational gene editing medicine VERVE-101. The candidate is designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver to reduce disease-driving low-density lipoprotein cholesterol (LDL-C).

VERVE-101 is being evaluated in the phase Ib study with trial endpoints of safety and tolerability as well as changes in blood PCSK9 protein and low-density lipoprotein cholesterol (LDL-C) levels in patients suffering from heterozygous familial hypercholesterolemia (HeFH), established atherosclerotic cardiovascular disease (ASCVD) and uncontrolled hypercholesterolemia.

A total of 13 participants have been dosed in the study. Among these, six have been dosed at 0.45 mg/kg of VERVE-101. Data showed that the VERVE-101 demonstrated time-averaged LDL-C reductions ranging between 21% and 73%, and averaging 46% for the first five participants in this cohort, with follow-up to at least 28 days.

However, the sixth participant experienced a serious drug-related adverse event. This patient, treated in the 0.45 mg/kg cohort, experienced a grade 3 drug-induced transient increase in serum alanine aminotransferase as well as a serious adverse event of grade 3 drug-induced thrombocytopenia within the first four days after dosing.  Per management, the abnormalities resolved fully within a few days and the participant did not experience any bleeding or other symptom related to the laboratory abnormalities.

Consequently, the company has decided to pause enrollment in this study in consultation with the independent data and safety monitoring board. Verve will now focus on the development of its other candidate, VERVE-102, and the initiation of the Heart-2 clinical trial.

VNDA Up on Drug Approval:  Vanda Pharmaceuticals Inc.'s VNDA shares rose after the company obtained FDA approval of Fanapt (iloperidone) tablets for the acute treatment of manic or mixed episodes associated with bipolar I disorder in adults. The approval of Fanapt for bipolar disorder was based on data from a pivotal study, which evaluated around 400 patients with bipolar I disorder. Data from the study showed that patients who were treated with Fanapt experienced a larger improvement in the core symptoms of mania than patients who received placebo at the end of week four, and this difference was statistically significant.

The drug is already approved in the United States for the acute treatment of patients with schizophrenia. The approval for Fanapt in the given indication could present a significant commercial opportunity for Vanda Pharmaceuticals.

Performance

The Nasdaq Biotechnology Index has dropped 3.15% in the past four trading sessions. Among the biotech giants, Moderna's shares have lost 8.48% during the same time frame. Over the past six months, shares of VRTX have risen 18.44%.

What's Next in Biotech?

Stay tuned for pipeline updates.

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