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Biotech Stock Roundup: BHVN, WVE, ZVRA Stocks Up on Updates, BIIB & UCBs Study Data & More
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The biotech sector was in focus last week, with regular pipeline and regulatory updates. Drug approvals were in the spotlight as well.
Here's a recap of the week’s most important stories:
Biogen, UCB Lupus Study Data
Biogen (BIIB - Free Report) and partner UCB announced that the phase III PHOENYCS GO study, which evaluated the investigational drug candidate, dapirolizumab pegol, for the treatment of moderate-to-severe systemic lupus erythematosus (SLE), met the primary endpoint by demonstrating clinical improvement in moderate-to-severe SLE.
The double-blind, placebo-controlled phase III PHOENYCS GO study evaluated dapirolizumab pegol as an add-on therapy to standard-of-care (SOC) versus placebo with SOC for treating SLE. Data from the study showed that dapirolizumab pegol, in combination with SOC, led to greater improvement in moderate-to-severe disease activity after 48 weeks compared with placebo in addition to SOC.
The study also demonstrated clinical improvements in key secondary endpoints measuring disease activity and flares. The safety profile of dapirolizumab pegol was similar to that seen in the previous studies.
Following the PHOENYCS GO study's success, BIIB and UCB plan to initiate a second phase III study, PHOENYCS FLY, on dapirolizumab pegol in 2024.
FDA Rejects VNDA’s NDA
Shares of Vanda Pharmaceuticals Inc. (VNDA - Free Report) were down after the FDA issued a complete response letter (CRL) to its new drug application (NDA) seeking approval for its pipeline candidate, tradipitant, for the treatment of symptoms in gastroparesis.
The NDA included evidence of the candidate’s efficacy from two placebo-controlled studies. However, per the CRL, the FDA called for Vanda to conduct additional studies on tradipitant with a design that was inconsistent with the advice of key experts in the field and not appropriate based on the scientific understanding of the disease.
Also, per management, the FDA delayed its decision by more than 185 days and failed to meet the requirements specified by the Food Drug and Cosmetic Act (“FDCA”). Notably, the FDCA requires the FDA to review an NDA and provide either an approval or a possible hearing within 180 days of the filing. The FDA failed to do either. VNDA also stated that it had repeatedly asked the FDA to hold an advisory committee meeting to discuss the NDA for tradipitant that the regulatory body declined.
Despite the FDA declining to approve tradipitant for treating symptoms in gastroparesis, the company will continue to pursue the marketing approval for tradipitant in the given indication.
BHVN Stock Up on Study Data
Biohaven Ltd. (BHVN - Free Report) announced positive top-line data from the pivotal BHV4157-206-RWE study, which evaluated its pipeline candidate, troriluzole, for the treatment of spinocerebellar ataxia (SCA), a rare and debilitating neurodegenerative disease. The study showed the efficacy of troriluzole on the mean change from baseline in the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) following three years of treatment.
Also, treatment with troriluzole led to statistically significant improvements in the f-SARA at years one and two.
Based on the positive data from the BHV4157-206-RWE study, along with previous safety and efficacy data, BHVN plans to submit a ND) for troriluzole in SCA with the FDA in the fourth quarter of 2024. Biohaven plans to launch troriluzole in the United States in 2025 upon a potential approval.
WVE Surges on Study Data
Wave Life Sciences (WVE - Free Report) announced positive interim data from a mid-stage study evaluating its investigational candidate, WVE-N531, to treat boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
Per the interim data readout from the phase II FORWARD-53 study, treatment with WVE-N531 for 24 weeks resulted in a mean muscle content-adjusted dystrophin expression of 9% and unadjusted dystrophin of 5.5%. Treatment with WVE-N531 for 24 weeks also resulted in myofiber regeneration and improvements in muscle health.
The interim data readout also showed significant decreases in creatine kinase and aspartate aminotransferase levels from baseline, signifying meaningful improvement in muscle health.
WVE expects to share 48-week data from the complete FORWARD-53 study of WVE-N531 for DMD in the first quarter of 2025 and receive feedback from regulators on a pathway to accelerated approval.
Shares of WVE skyrocketed following the data announcement.
ZVRA Stock Rises on Drug Approval
Zevra Therapeutics, Inc. (ZVRA - Free Report) obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC). The drug was approved under the brand name Miplyffa.
The FDA approved Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. In addition, Zevra announced that it has received a rare pediatric disease priority review voucher in conjunction with the approval.
The approval of Miplyffa for the treatment of NPC is based on the totality of the data in the NDA, which included additional evidence supporting trial endpoints, FDA-preferred analyses and additional confirmatory evidence, both clinical and nonclinical.
Zevra stock skyrocketed to a new 52-week high following the announcement.
Image: Bigstock
Biotech Stock Roundup: BHVN, WVE, ZVRA Stocks Up on Updates, BIIB & UCBs Study Data & More
The biotech sector was in focus last week, with regular pipeline and regulatory updates. Drug approvals were in the spotlight as well.
Here's a recap of the week’s most important stories:
Biogen, UCB Lupus Study Data
Biogen (BIIB - Free Report) and partner UCB announced that the phase III PHOENYCS GO study, which evaluated the investigational drug candidate, dapirolizumab pegol, for the treatment of moderate-to-severe systemic lupus erythematosus (SLE), met the primary endpoint by demonstrating clinical improvement in moderate-to-severe SLE.
The double-blind, placebo-controlled phase III PHOENYCS GO study evaluated dapirolizumab pegol as an add-on therapy to standard-of-care (SOC) versus placebo with SOC for treating SLE. Data from the study showed that dapirolizumab pegol, in combination with SOC, led to greater improvement in moderate-to-severe disease activity after 48 weeks compared with placebo in addition to SOC.
The study also demonstrated clinical improvements in key secondary endpoints measuring disease activity and flares. The safety profile of dapirolizumab pegol was similar to that seen in the previous studies.
Following the PHOENYCS GO study's success, BIIB and UCB plan to initiate a second phase III study, PHOENYCS FLY, on dapirolizumab pegol in 2024.
FDA Rejects VNDA’s NDA
Shares of Vanda Pharmaceuticals Inc. (VNDA - Free Report) were down after the FDA issued a complete response letter (CRL) to its new drug application (NDA) seeking approval for its pipeline candidate, tradipitant, for the treatment of symptoms in gastroparesis.
The NDA included evidence of the candidate’s efficacy from two placebo-controlled studies. However, per the CRL, the FDA called for Vanda to conduct additional studies on tradipitant with a design that was inconsistent with the advice of key experts in the field and not appropriate based on the scientific understanding of the disease.
Also, per management, the FDA delayed its decision by more than 185 days and failed to meet the requirements specified by the Food Drug and Cosmetic Act (“FDCA”). Notably, the FDCA requires the FDA to review an NDA and provide either an approval or a possible hearing within 180 days of the filing. The FDA failed to do either. VNDA also stated that it had repeatedly asked the FDA to hold an advisory committee meeting to discuss the NDA for tradipitant that the regulatory body declined.
Despite the FDA declining to approve tradipitant for treating symptoms in gastroparesis, the company will continue to pursue the marketing approval for tradipitant in the given indication.
BHVN Stock Up on Study Data
Biohaven Ltd. (BHVN - Free Report) announced positive top-line data from the pivotal BHV4157-206-RWE study, which evaluated its pipeline candidate, troriluzole, for the treatment of spinocerebellar ataxia (SCA), a rare and debilitating neurodegenerative disease. The study showed the efficacy of troriluzole on the mean change from baseline in the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) following three years of treatment.
Also, treatment with troriluzole led to statistically significant improvements in the f-SARA at years one and two.
Based on the positive data from the BHV4157-206-RWE study, along with previous safety and efficacy data, BHVN plans to submit a ND) for troriluzole in SCA with the FDA in the fourth quarter of 2024. Biohaven plans to launch troriluzole in the United States in 2025 upon a potential approval.
WVE Surges on Study Data
Wave Life Sciences (WVE - Free Report) announced positive interim data from a mid-stage study evaluating its investigational candidate, WVE-N531, to treat boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.
Per the interim data readout from the phase II FORWARD-53 study, treatment with WVE-N531 for 24 weeks resulted in a mean muscle content-adjusted dystrophin expression of 9% and unadjusted dystrophin of 5.5%. Treatment with WVE-N531 for 24 weeks also resulted in myofiber regeneration and improvements in muscle health.
The interim data readout also showed significant decreases in creatine kinase and aspartate aminotransferase levels from baseline, signifying meaningful improvement in muscle health.
WVE expects to share 48-week data from the complete FORWARD-53 study of WVE-N531 for DMD in the first quarter of 2025 and receive feedback from regulators on a pathway to accelerated approval.
Shares of WVE skyrocketed following the data announcement.
ZVRA Stock Rises on Drug Approval
Zevra Therapeutics, Inc. (ZVRA - Free Report) obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC). The drug was approved under the brand name Miplyffa.
The FDA approved Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. In addition, Zevra announced that it has received a rare pediatric disease priority review voucher in conjunction with the approval.
The approval of Miplyffa for the treatment of NPC is based on the totality of the data in the NDA, which included additional evidence supporting trial endpoints, FDA-preferred analyses and additional confirmatory evidence, both clinical and nonclinical.
Zevra stock skyrocketed to a new 52-week high following the announcement.
Performance
The Nasdaq Biotechnology Index has lost 3.46% in the past five trading sessions and MRNA’s shares have lost 8.90%. In the past six months, shares of AMGN have rallied 11.41%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD, NUVL Stocks Surge on Study Data, MRNA Down on Updates)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.