NVAX vaccine approval, ALNY and ATHA shares plunge after updates and more

It was a busy week for the biotech sector with many important data and regulatory updates. Novavax NVAX and Alnylam Pharmaceuticals, Inc. ALNY was in the spotlight. While NVAX attracted attention for receiving FDA approval for its latest COVID-19 vaccine, ALNY disappointed investors with its trial data.

Summary of the most important stories of the week:

NVAX receives FDA approval for vaccine: The FDA granted emergency use authorization (EUA) to an updated version of Novavax's protein-based COVID-19 vaccine for people 12 years of age and older. Following this authorization, Novavax's updated vaccine is the only non-mRNA vaccine option available to people in the United States. The vaccine is specifically designed for the JN.1 strain.

The updated formulation is based on FDA guidance from late June that advised COVID-19 vaccine manufacturers to update their respective COVID-19 vaccines to combat the KP.2 strain when possible. The guidance is also an update to FDA's previous recommendation from early June that recommended updating COVID-19 vaccines to combat the JN.1 strain.

The EUA was granted based on data from nonclinical studies that showed that the updated vaccine formulation was also effective against currently circulating strains, including KP.2.3, KP.3, KP.3.1.1 and LB.1.

ALNY down due to data: Alnylam Pharmaceuticals announced detailed data from the Phase III HELIOS-B trial evaluating the RNAi therapeutic Amvuttra (vutrisiran) for an expanded indication. The HELIOS-B trial is designed to facilitate the expansion of the indication of Amvuttra for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

According to the company, the HELIOS-B study met its primary endpoint, showing a statistically significant reduction in all-cause mortality and recurrent cardiovascular events in the overall and monotherapy populations. As part of the primary endpoint, treatment with vutrisiran in the double-blind phase resulted in a 30% reduction in mortality in the monotherapy population. According to management, this reduction was not significant. Investors were not impressed with the data and ALNY stock has most likely declined as a result.

Vutrisiran is already approved in the United States under the brand name Amvuttra for the treatment of adult patients with polyneuropathy of hereditary ATTR amyloidosis.

ATHA shares collapse due to data: Shares of Athira Pharma, Inc. ATHA plunged after the company announced disappointing topline data from the Phase II/III LIFT-AD trial evaluating its pipeline candidate fosgonimeton, a drug used to treat mild to moderate Alzheimer's disease (AD).

The story continues

The LIFT-AD study evaluated once-daily subcutaneous injection of fosgonimeton (40 mg) compared with placebo over a treatment period of 26 weeks in patients with mild to moderate Alzheimer's disease.

The study did not meet the primary endpoint, the Global Statistical Test (GST), a composite of results from measures of cognition and function. The study also did not meet key secondary endpoints, namely changes in cognition and function, plasma biomarkers of neurodegeneration, protein pathology, and neuroinflammation.

In pre-specified subgroups of patients with moderate Alzheimer's disease or carriers of the APOE4 gene, cognitive and functional abilities improved or stabilized in patients receiving fosgonimeton compared with placebo. In pre-specified biomarker analyses, cognitive and functional abilities, the two key GST measures, clearly favored treatment with fosgonimeton over placebo.

Athira currently has a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Vaxcyte shares rise rapidly after study data: Shares of Vaxcyte PCVX rose sharply after the company reported positive results from a Phase I/II study of VAX-31, an investigational 31-valent pneumococcal conjugate vaccine (PCV), in adults aged 50 years and older.

Data from the study showed that study participants who received VAX-31 achieved robust immune responses with opsonophagocytic activity for all 31 serotypes at all three dose levels (low, medium and high) over a six-month evaluation period. While the vaccine met or exceeded regulatory immunogenicity criteria for all serotypes at the high and medium dose levels, it did not meet them for two serotypes at the low dose level.

The results of the Phase I/II study match or exceed the immune responses generated by Pfizer's Prevnar 20, the current standard of care in the PCV space. Data from the study showed that VAX-31 was well tolerated by study participants and had a similar safety profile to Prevnar 20 across all three dose levels. VAX-31 covers 31 serotypes, including all 20 serotypes included in Pfizer's Prevnar 20.

Following the announcement, Vaxcyte shares reached an all-time high price of $119.27.

DYN shares down after updates: Shares of Dyne Therapeutics, Inc. (DYN) crashed on September 3 due to two major announcements.

DYN reported mixed data from its ongoing Phase I/II trial, the DELIVER study, of DYNE-251 in patients with exon 51 skipping-susceptible Duchenne muscular dystrophy (DMD). DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody that binds to transferrin receptor 1, which is highly expressed in muscle.

Reported data include six-month biomarker and functional data from eight male patients in the 20 mg/kg (approximate PMO dose) cohort who were randomized to receive DYNE-251 or placebo every four weeks and 12-month functional data from six participants in the 10 mg/kg cohort.

Patients treated with 20 mg/kg DYNE-251 every four weeks had a mean absolute dystrophin expression of 3.71% of normal (unadjusted for muscle fraction). This value is more than ten times higher than the 0.3% seen in a study with the standard weekly drug eteplirsen. The DYNE-251-treated group achieved a mean absolute dystrophin expression of 8.72% when adjusted for muscle fraction. This value was higher than that seen with peptide conjugate PMOs in clinical development.

Dyne said DYNE-251 had a favorable safety profile based on the 54 participants in the DELIVER study. The majority of treatment-related adverse events were mild or moderate. However, two participants at the 40 mg/kg dose experienced adverse events that may have been related to the study drug. Both participants recovered, according to the company.

Following the release of the data, DYN announced that its Chief Medical Officer (CMO), Chief Operating Officer and Chief Business Officer will be leaving the company. However, the CMO, Wildon Farwell, will remain with the company until the end of 2024.

Performance

The Nasdaq Biotechnology Index has lost 1.64% over the past four trading days and BMY shares have gained 1.83%. Over the past six months, REGN shares have gained 21.44%. (For the latest biotech stocks overview, see: Biotech Stock Overview: REGN Drug Approval, LXRX and RYTM Shares Gain After Update and More)

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What’s next for biotechnology?

Stay tuned for further pipeline updates.

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