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Biotech Stock Roundup: SAGE Plunges on Study Failure, AGEN Down on Regulatory Update & More
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It was a low-key week for the biotech sector, with nothing important from the bigwigs. Regular pipeline and other updates continue to be in the news. Among these, Sage Therapeutics, Inc. (SAGE - Free Report) is facing the brunt of investors due to the failure of a mid-stage study.
Recap of the Week’s Most Important Stories:
SAGE Plummets on Study Failure: SAGE and partner Biogen Inc. (BIIB - Free Report) announced the failure of a mid-stage study on oral investigational drug SAGE-324 (BIIB124) as a potential treatment in essential tremor (ET).
The phase II KINETIC 2 dose-range study did not demonstrate a statistically significant dose-response relationship in change from baseline to day 91 based on the primary endpoint, The Essential Tremor Rating Assessment Scale (TETRAS) Performance Subscale (PS) Item 4 (upper limb) total score in participants with ET.
Additionally, there was no statistically significant difference demonstrated for any dose of SAGE-324 as compared to placebo in the change from baseline to day 91 on the TETRAS PS Item 4 Total Score or the TETRAS Activities of Daily Living Composite Score.
Owing to the dismal results, SAGE and Biogen will close the ongoing open-label safety study of SAGE-324 in ET and do not plan to conduct any further clinical development of the same. Shares of SAGE plummeted on the news.
AGEN Tanks on Data: Agenus, Inc. (AGEN - Free Report) announced the outcome of its end-of-phase II (EOP2) meeting with the FDA regarding the accelerated development of its immunotherapy combination, botensilimab (BOT) and balstilimab (BAL).
The BOT/BAL combination is being studied for the treatment of adult patients with relapsed/refractory microsatellite stable colorectal cancer (r/r MSS CRC) with no active liver metastases.
The FDA discouraged the accelerated approval pathway for the BOT/BAL combination in r/r MSS CRC. As per the regulatory body, the objective response rate (ORR) observed in the phase II study might not convert to survival benefit.
AGEN announced top-line interim data from a phase II study that evaluated the BOT/BAL combination in r/r MSS CRC. The interim data demonstrated the best activity at 75 mg BOT/240mg BAL combination, which led to an ORR of 19.4%. Also, at the same dose, 90% of the patients were alive.
Per the press release, the company has reached an agreement with the FDA on the proposed dose of the BOT/BAL combination for a phase III study. The agreed dosing of the BOT/BAL combination regimen is BOT (75mg) once every six weeks for up to four doses, in combination with BAL (240mg), once every two weeks for up to two years.
The FDA has also proposed to include a BOT monotherapy arm in the phase III study. AGEN is currently looking for a partner in the United States to conduct the study.
BMRN’s Drug Label Expansion: BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has approved the label expansion of Brineura. The drug is now approved to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
The drug is already approved in symptomatic children, aged 3 years and older, with late infantile CLN2 disease. This expanded indication now includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or presymptomatic.
The supplemental biologics license application is supported by data from the phase II study 190-203, evaluating Brineura treatment over the span of approximately three years in children aged 1-6 years at baseline, including eight children less than 3 years of age.
Image: Bigstock
Biotech Stock Roundup: SAGE Plunges on Study Failure, AGEN Down on Regulatory Update & More
It was a low-key week for the biotech sector, with nothing important from the bigwigs. Regular pipeline and other updates continue to be in the news. Among these, Sage Therapeutics, Inc. (SAGE - Free Report) is facing the brunt of investors due to the failure of a mid-stage study.
Recap of the Week’s Most Important Stories:
SAGE Plummets on Study Failure: SAGE and partner Biogen Inc. (BIIB - Free Report) announced the failure of a mid-stage study on oral investigational drug SAGE-324 (BIIB124) as a potential treatment in essential tremor (ET).
The phase II KINETIC 2 dose-range study did not demonstrate a statistically significant dose-response relationship in change from baseline to day 91 based on the primary endpoint, The Essential Tremor Rating Assessment Scale (TETRAS) Performance Subscale (PS) Item 4 (upper limb) total score in participants with ET.
Additionally, there was no statistically significant difference demonstrated for any dose of SAGE-324 as compared to placebo in the change from baseline to day 91 on the TETRAS PS Item 4 Total Score or the TETRAS Activities of Daily Living Composite Score.
Owing to the dismal results, SAGE and Biogen will close the ongoing open-label safety study of SAGE-324 in ET and do not plan to conduct any further clinical development of the same. Shares of SAGE plummeted on the news.
AGEN Tanks on Data: Agenus, Inc. (AGEN - Free Report) announced the outcome of its end-of-phase II (EOP2) meeting with the FDA regarding the accelerated development of its immunotherapy combination, botensilimab (BOT) and balstilimab (BAL).
The BOT/BAL combination is being studied for the treatment of adult patients with relapsed/refractory microsatellite stable colorectal cancer (r/r MSS CRC) with no active liver metastases.
The FDA discouraged the accelerated approval pathway for the BOT/BAL combination in r/r MSS CRC. As per the regulatory body, the objective response rate (ORR) observed in the phase II study might not convert to survival benefit.
AGEN announced top-line interim data from a phase II study that evaluated the BOT/BAL combination in r/r MSS CRC. The interim data demonstrated the best activity at 75 mg BOT/240mg BAL combination, which led to an ORR of 19.4%. Also, at the same dose, 90% of the patients were alive.
Per the press release, the company has reached an agreement with the FDA on the proposed dose of the BOT/BAL combination for a phase III study. The agreed dosing of the BOT/BAL combination regimen is BOT (75mg) once every six weeks for up to four doses, in combination with BAL (240mg), once every two weeks for up to two years.
The FDA has also proposed to include a BOT monotherapy arm in the phase III study. AGEN is currently looking for a partner in the United States to conduct the study.
BMRN’s Drug Label Expansion: BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has approved the label expansion of Brineura. The drug is now approved to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
The drug is already approved in symptomatic children, aged 3 years and older, with late infantile CLN2 disease. This expanded indication now includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or presymptomatic.
The supplemental biologics license application is supported by data from the phase II study 190-203, evaluating Brineura treatment over the span of approximately three years in children aged 1-6 years at baseline, including eight children less than 3 years of age.
BMRN currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Performance
The Nasdaq Biotechnology Index has lost 0.75% in the past five trading sessions and BMY’s shares have risen 3.50%. In the past six months, shares of MRNA have rallied 16.89%. (See the last biotech stock roundup here: Biotech Stock Roundup: LXRX Up on Regulatory Update, SAVA, LXEO Down on Updates)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.