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Biotech Stock Roundup: NVAX Vaccine Approval, ALNY, ATHA Stock Dive on Updates & More
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It was a busy week for the biotech sector, with a lot of important data readouts and regulatory updates. Among these, Novavax (NVAX - Free Report) and Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) were in the spotlight. While NVAX grabbed focus on obtaining the FDA nod for its latest COVID-19 vaccine, ALNY disappointed investors with its study data.
Recap of the Week’s Most Important Stories:
NVAX Gets FDA Approval for Vaccine: The FDA granted emergency use authorization (EUA) to an updated version of Novavax’s protein-based COVID-19 vaccine for individuals aged 12 years and older. Following this nod, Novavax’s updated jab is the only non-mRNA vaccine option available to people in the United States. The same has been designed to target the JN.1 strain.
The updated composition is based on guidance from the FDA issued in late June, which advised COVID-19 vaccine manufacturers to update their respective COVID-19 vaccines to target the KP.2 strain if it is feasible. The guidance is also an update on the FDA’s prior advice issued in early June that recommended updating COVID-19 vaccines to target the JN.1 strain.
The EUA was granted based on data from non-clinical studies that showed that the updated vaccine composition was also effective against the current circulating strains, including KP.2.3, KP.3, KP.3.1.1 and LB.1.
ALNY Down on Data: Alnylam Pharmaceuticals announced detailed data from the phase III HELIOS-B, which evaluated its investigational RNAi therapeutic, Amvuttra (vutrisiran), for an expanded indication. The HELIOS-B study intends to facilitate the label expansion of Amvuttra for treating transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
Per the company, the HELIOS-B study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in the overall and the monotherapy population. As a component of the primary endpoint, treatment with vutrisiran led to a 30% reduction in mortality in the monotherapy population in the double-blind period. Per management, this reduction was non-significant. Investors were not impressed by the data and ALNY’s stock most likely declined on the same.
Vutrisiran is already approved in the United States for treating adult patients with polyneuropathy of hereditary ATTR amyloidosis under the brand name Amvuttra.
ATHA Stock Tanks on Data: Shares of Athira Pharma, Inc. (ATHA - Free Report) plunged after the company announced disappointing top-line data from the phase II/III LIFT-AD study evaluating its pipeline candidate, fosgonimeton. The drug is used for the treatment of mild-to-moderate Alzheimer’s disease (AD).
The LIFT-AD study investigated once-daily fosgonimeton (40 mg) subcutaneous injection versus placebo over a treatment period of 26 weeks in mild-to-moderate AD patients.
The study did not meet the primary endpoint which was the Global Statistical Test (GST), a combination of results from measures of cognition and function. The study also failed to meet the key secondary endpoints of changes in cognition and function, plasma biomarkers of neurodegeneration, protein pathology and neuroinflammation.
In pre-specified subgroups of patients with moderate AD or who are carriers of the APOE4 gene, cognition and function improved or stabilized in patients who received fosgonimeton compared with a placebo. In prespecified biomarker analyses, cognition and function, the two key measures of GST, directionally favored treatment with fosgonimeton over placebo.
Vaxcyte Stock Soars on Study Data: Shares of Vaxcyte (PCVX - Free Report) skyrocketed after it reported positive top-line results from a phase I/II study on VAX-31, which is an investigational 31-valent pneumococcal conjugate vaccine (PCV). The study was conducted on adults aged 50 years and older.
Data from the study showed that study participants who received VAX-31 achieved robust opsonophagocytic activity immune responses for all 31 serotypes across all three dose levels (low, medium and high) in a six-month evaluation period. While the vaccine met or exceeded the regulatory immunogenicity criteria for all the serotypes at the high and middle dose levels, it did not meet the same for two serotypes at the low dose level.
The results from the phase I/II study equal or even exceed the immune responses generated by Pfizer’s Prevnar 20, which is the current standard of care in the PCV space. Data from the study showed that VAX-31 was shown to be well-tolerated by study participants and demonstrated a safety profile similar to Prevnar 20 across all three dose levels. VAX-31 covers 31 serotypes, which includes all the 20 serotypes found in Pfizer’s Prevnar 20.
Following the announcement, shares of Vaxcyte hit an all-time high price of $119.27.
DYN Stock Down on Updates: Shares of Dyne Therapeutics, Inc. (DYN) tumbled on Sep. 3 on two key announcements.
DYN reported mixed data from its ongoing phase I/II study, the DELIVER trial of DYNE-251, in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody that binds to the transferrin receptor 1, which is highly expressed on muscle.
Data reported includes six-month biomarker and functional data from 8 male patients enrolled in the 20 mg/kg (approximate PMO dose) cohort who were randomized to receive DYNE-251 or placebo once every four weeks and 12-month functional data from six participants in the 10 mg/kg cohort.
Patients treated with 20 mg/kg of DYNE-251 once every four weeks reported a mean absolute dystrophin expression of 3.71% of normal levels (unadjusted for muscle content), more than 10 times higher than the 0.3% reported in a study of the weekly standard of care, eteplirsen. The DYNE-251 treated group reached 8.72% mean absolute dystrophin when muscle content was adjusted. This level was greater than that reported by peptide conjugate PMOs in clinical development.
Dyne stated that DYNE-251 demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. The majority of treatment-emergent adverse events were mild or moderate. However, two participants at the 40 mg/kg dose level experienced adverse events potentially related to the study drug. Per the company, both participants recovered.
Post the data readout, DYN announced that its chief medical officer (CMO), chief operating officer and chief business officer are leaving the company. Nonetheless, the CMO, Wildon Farwell, will remain through the end of 2024.
Image: Bigstock
Biotech Stock Roundup: NVAX Vaccine Approval, ALNY, ATHA Stock Dive on Updates & More
It was a busy week for the biotech sector, with a lot of important data readouts and regulatory updates. Among these, Novavax (NVAX - Free Report) and Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) were in the spotlight. While NVAX grabbed focus on obtaining the FDA nod for its latest COVID-19 vaccine, ALNY disappointed investors with its study data.
Recap of the Week’s Most Important Stories:
NVAX Gets FDA Approval for Vaccine: The FDA granted emergency use authorization (EUA) to an updated version of Novavax’s protein-based COVID-19 vaccine for individuals aged 12 years and older. Following this nod, Novavax’s updated jab is the only non-mRNA vaccine option available to people in the United States. The same has been designed to target the JN.1 strain.
The updated composition is based on guidance from the FDA issued in late June, which advised COVID-19 vaccine manufacturers to update their respective COVID-19 vaccines to target the KP.2 strain if it is feasible. The guidance is also an update on the FDA’s prior advice issued in early June that recommended updating COVID-19 vaccines to target the JN.1 strain.
The EUA was granted based on data from non-clinical studies that showed that the updated vaccine composition was also effective against the current circulating strains, including KP.2.3, KP.3, KP.3.1.1 and LB.1.
ALNY Down on Data: Alnylam Pharmaceuticals announced detailed data from the phase III HELIOS-B, which evaluated its investigational RNAi therapeutic, Amvuttra (vutrisiran), for an expanded indication. The HELIOS-B study intends to facilitate the label expansion of Amvuttra for treating transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
Per the company, the HELIOS-B study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in the overall and the monotherapy population. As a component of the primary endpoint, treatment with vutrisiran led to a 30% reduction in mortality in the monotherapy population in the double-blind period. Per management, this reduction was non-significant. Investors were not impressed by the data and ALNY’s stock most likely declined on the same.
Vutrisiran is already approved in the United States for treating adult patients with polyneuropathy of hereditary ATTR amyloidosis under the brand name Amvuttra.
ATHA Stock Tanks on Data: Shares of Athira Pharma, Inc. (ATHA - Free Report) plunged after the company announced disappointing top-line data from the phase II/III LIFT-AD study evaluating its pipeline candidate, fosgonimeton. The drug is used for the treatment of mild-to-moderate Alzheimer’s disease (AD).
The LIFT-AD study investigated once-daily fosgonimeton (40 mg) subcutaneous injection versus placebo over a treatment period of 26 weeks in mild-to-moderate AD patients.
The study did not meet the primary endpoint which was the Global Statistical Test (GST), a combination of results from measures of cognition and function. The study also failed to meet the key secondary endpoints of changes in cognition and function, plasma biomarkers of neurodegeneration, protein pathology and neuroinflammation.
In pre-specified subgroups of patients with moderate AD or who are carriers of the APOE4 gene, cognition and function improved or stabilized in patients who received fosgonimeton compared with a placebo. In prespecified biomarker analyses, cognition and function, the two key measures of GST, directionally favored treatment with fosgonimeton over placebo.
Athira currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Vaxcyte Stock Soars on Study Data: Shares of Vaxcyte (PCVX - Free Report) skyrocketed after it reported positive top-line results from a phase I/II study on VAX-31, which is an investigational 31-valent pneumococcal conjugate vaccine (PCV). The study was conducted on adults aged 50 years and older.
Data from the study showed that study participants who received VAX-31 achieved robust opsonophagocytic activity immune responses for all 31 serotypes across all three dose levels (low, medium and high) in a six-month evaluation period. While the vaccine met or exceeded the regulatory immunogenicity criteria for all the serotypes at the high and middle dose levels, it did not meet the same for two serotypes at the low dose level.
The results from the phase I/II study equal or even exceed the immune responses generated by Pfizer’s Prevnar 20, which is the current standard of care in the PCV space. Data from the study showed that VAX-31 was shown to be well-tolerated by study participants and demonstrated a safety profile similar to Prevnar 20 across all three dose levels. VAX-31 covers 31 serotypes, which includes all the 20 serotypes found in Pfizer’s Prevnar 20.
Following the announcement, shares of Vaxcyte hit an all-time high price of $119.27.
DYN Stock Down on Updates: Shares of Dyne Therapeutics, Inc. (DYN) tumbled on Sep. 3 on two key announcements.
DYN reported mixed data from its ongoing phase I/II study, the DELIVER trial of DYNE-251, in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody that binds to the transferrin receptor 1, which is highly expressed on muscle.
Data reported includes six-month biomarker and functional data from 8 male patients enrolled in the 20 mg/kg (approximate PMO dose) cohort who were randomized to receive DYNE-251 or placebo once every four weeks and 12-month functional data from six participants in the 10 mg/kg cohort.
Patients treated with 20 mg/kg of DYNE-251 once every four weeks reported a mean absolute dystrophin expression of 3.71% of normal levels (unadjusted for muscle content), more than 10 times higher than the 0.3% reported in a study of the weekly standard of care, eteplirsen. The DYNE-251 treated group reached 8.72% mean absolute dystrophin when muscle content was adjusted. This level was greater than that reported by peptide conjugate PMOs in clinical development.
Dyne stated that DYNE-251 demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. The majority of treatment-emergent adverse events were mild or moderate. However, two participants at the 40 mg/kg dose level experienced adverse events potentially related to the study drug. Per the company, both participants recovered.
Post the data readout, DYN announced that its chief medical officer (CMO), chief operating officer and chief business officer are leaving the company. Nonetheless, the CMO, Wildon Farwell, will remain through the end of 2024.
erformance
The Nasdaq Biotechnology Index has lost 1.64% in the past four trading sessions and BMY’s shares have risen 1.83%. In the past six months, shares of REGN have rallied 21.44%. (See the last biotech stock roundup here: Biotech Stock Roundup: REGN's Drug Approval, LXRX, RYTM Stocks Gain on Update & More)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.