We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Biotech Stock Roundup: LXRX Up on Regulatory Update, SAVA, LXEO Down on Updates
Read MoreHide Full Article
It was a regular week for the biotech sector with quite a few important pipeline and regulatory updates. Among these, Cassava Sciences, Inc. (SAVA - Free Report) and Lexeo Therapeutics (LXEO - Free Report) were down on leadership changes and study results, respectively.
Recap of the Week’s Most Important Stories:
SAVA Down on CEO Resignation: Cassava Sciences announced that Remi Barbier, the company’s chairman, president and CEO, has resigned from his positions. Consequently, the board has appointed Richard Barry as SAVA’s executive chairman and principal executive officer, with immediate effect. Cassava is looking for a permanent CEO and will separate the roles of its chairman and CEO. In addition, Lindsay Burns, Ph.D., SVP, Neuroscience, is also leaving the company. Shares of SAVA were down 29% following the news.
The company stated that its priority remains on developing a potentially effective treatment for Alzheimer’s disease and it will do so with transparency, accountability and the highest ethical business practices. SAVA also stated that the ongoing phase III trials are being run according to FDA protocols, and no one from the company knows or will ever know the subjects receiving a drug or placebo. No individual has access to sub-study results and other information that might be used to infer which patients are on placebo.
Blinding information is and will be held exclusively by a small group of professionals at SAVA’s CRO, Premier Research. Statistical analyses for these studies will be conducted by professional biostatisticians at Pentara Corporation.
Regulatory Update From Lexicon: Lexicon Pharmaceuticals (LXRX - Free Report) announced that the FDA has accepted its resubmitted new drug application (NDA) seeking approval for sotagliflozin as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes (T1D) and chronic kidney disease. A final decision is expected by Dec 20, 2024. If approved, the product will be marketed under the brand name Zynquista, with a commercial launch expected in early 2025. The company’s shares rose on the news.
Lexicon had initially submitted an NDA in 2018 for sotagliflozin in T1D indication. However, the FDA issued a complete response letter (CRL) to the NDA in 2019 after one of its advisory committees issued a split opinion regarding its approval. Management stated that the agency considers the resubmission as a complete response to this CRL.
Regulatory Update From Kyverna: Kyverna Therapeutics, Inc. (KYTX - Free Report) announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead pipeline candidate KYV-101. It did so for the treatment of patients suffering from refractory stiff-person syndrome (SPS).
KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases. SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms and legs, thereby affecting the ability to walk or move.
The RMAT designation should allow Kyverna to receive expert guidance on efficient drug development and the use of surrogate endpoints from senior FDA officials. Last month, the FDA cleared the company’s investigational new drug application for KYV-101, which is to be used for the treatment of SPS in Kyverna's trial, named KYSA-8.
Study Data From Lexeo: Lexeo Therapeutics reported positive interim data from two cohorts of the phase I/II SUNRISE-FA study evaluating LX2006, an investigational gene therapy for treating Friedreich ataxia (FA) cardiomyopathy. Data from the SUNRISE-FA study showed that treatment with LX2006 achieved a mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline. Treatment with the therapy was also well-tolerated across all study participants, with no treatment-related serious adverse event.
The interim data also showed that treatment with LX2006 demonstrated improvement in other key measures of cardiac status, including left ventricular wall thickness (an early indicator of an inefficient heart) and troponin I (a biomarker of myocardial injury), in the majority of participants at 12 months.
Based on the above results, Lexeo intends to seek FDA’s approval for LX2006 under the accelerated pathway. Management is currently dosing patients in the third dose cohort of the SUNRISE-FA study, which is also enrolling study participants.
However, the stock went down following the results as some investors were not impressed by the data. They believed that the results probably weren’t good enough to procure accelerated approval from the FDA.
Image: Bigstock
Biotech Stock Roundup: LXRX Up on Regulatory Update, SAVA, LXEO Down on Updates
It was a regular week for the biotech sector with quite a few important pipeline and regulatory updates. Among these, Cassava Sciences, Inc. (SAVA - Free Report) and Lexeo Therapeutics (LXEO - Free Report) were down on leadership changes and study results, respectively.
Recap of the Week’s Most Important Stories:
SAVA Down on CEO Resignation: Cassava Sciences announced that Remi Barbier, the company’s chairman, president and CEO, has resigned from his positions. Consequently, the board has appointed Richard Barry as SAVA’s executive chairman and principal executive officer, with immediate effect. Cassava is looking for a permanent CEO and will separate the roles of its chairman and CEO. In addition, Lindsay Burns, Ph.D., SVP, Neuroscience, is also leaving the company. Shares of SAVA were down 29% following the news.
The company stated that its priority remains on developing a potentially effective treatment for Alzheimer’s disease and it will do so with transparency, accountability and the highest ethical business practices. SAVA also stated that the ongoing phase III trials are being run according to FDA protocols, and no one from the company knows or will ever know the subjects receiving a drug or placebo. No individual has access to sub-study results and other information that might be used to infer which patients are on placebo.
Blinding information is and will be held exclusively by a small group of professionals at SAVA’s CRO, Premier Research. Statistical analyses for these studies will be conducted by professional biostatisticians at Pentara Corporation.
Regulatory Update From Lexicon: Lexicon Pharmaceuticals (LXRX - Free Report) announced that the FDA has accepted its resubmitted new drug application (NDA) seeking approval for sotagliflozin as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes (T1D) and chronic kidney disease. A final decision is expected by Dec 20, 2024. If approved, the product will be marketed under the brand name Zynquista, with a commercial launch expected in early 2025. The company’s shares rose on the news.
Lexicon had initially submitted an NDA in 2018 for sotagliflozin in T1D indication. However, the FDA issued a complete response letter (CRL) to the NDA in 2019 after one of its advisory committees issued a split opinion regarding its approval. Management stated that the agency considers the resubmission as a complete response to this CRL.
Lexicon currently carries a Zacks Rank #3 (Hold).You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Regulatory Update From Kyverna: Kyverna Therapeutics, Inc. (KYTX - Free Report) announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead pipeline candidate KYV-101. It did so for the treatment of patients suffering from refractory stiff-person syndrome (SPS).
KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases. SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms and legs, thereby affecting the ability to walk or move.
The RMAT designation should allow Kyverna to receive expert guidance on efficient drug development and the use of surrogate endpoints from senior FDA officials. Last month, the FDA cleared the company’s investigational new drug application for KYV-101, which is to be used for the treatment of SPS in Kyverna's trial, named KYSA-8.
Study Data From Lexeo: Lexeo Therapeutics reported positive interim data from two cohorts of the phase I/II SUNRISE-FA study evaluating LX2006, an investigational gene therapy for treating Friedreich ataxia (FA) cardiomyopathy. Data from the SUNRISE-FA study showed that treatment with LX2006 achieved a mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline. Treatment with the therapy was also well-tolerated across all study participants, with no treatment-related serious adverse event.
The interim data also showed that treatment with LX2006 demonstrated improvement in other key measures of cardiac status, including left ventricular wall thickness (an early indicator of an inefficient heart) and troponin I (a biomarker of myocardial injury), in the majority of participants at 12 months.
Based on the above results, Lexeo intends to seek FDA’s approval for LX2006 under the accelerated pathway. Management is currently dosing patients in the third dose cohort of the SUNRISE-FA study, which is also enrolling study participants.
However, the stock went down following the results as some investors were not impressed by the data. They believed that the results probably weren’t good enough to procure accelerated approval from the FDA.
Performance
The Nasdaq Biotechnology Index has gained 3.88% in the past five trading sessions and Incyte’s shares have risen 7.53%. In the past six months, shares of MRNA have rallied 23.63%. (See the last biotech stock roundup here: Biotech Stock Roundup: KYMR, IDYA, QURE Soar on Study Data, HLVX Tanks on Study Failure)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.