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Biotech Stock Roundup: BMY's Drug Approval, VRTX & CRSP Gene Therapy Get MHRA Nod
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With the conclusion of the third-quarter earnings for the biotech sector, the focus is back on regular drug approvals and other pipeline and regulatory updates. Quite a few companies came out with important updates on their key candidates
Recap of the Week’s Most Important Stories:
Arcellx Surges on Deal Expansion: Shares of Arcellx, Inc. (ACLX - Free Report) gained after the company and partner Kite, a Gilead company, announced an expansion of their existing agreement. The companies collaborated in December 2022 to co-develop and co-commercialize Arcellx's CART-ddBCMA candidate for the treatment of patients with relapsed or refractory multiple myeloma.
Kite has exercised its option to negotiate a license for Arcellx's ARC-SparX program, ACLX-001, in multiple myeloma. The companies also expanded the scope of the collaboration for Arcellx's CART-ddBCMA to include lymphomas.
Per the terms of the agreement, Arcellx will receive an upfront cash payment of $85 million at closing and an equity investment of $200 million. It will also be eligible for potential milestone payments, including the advancement of lymphoma and the license for ARC-SparX, as well as additional milestones, to offset prespecified development costs over a limited period of time.
This infusion of cash is expected to extend Arcellx’s cash runway into 2027. Gilead will have an approximate 13% stake following this investment.
Bristol Myers’ Drug Approval: Bristol Myers Squibb (BMY - Free Report) announced that the FDA approved pipeline candidate repotrectinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). Repotrectinib is a tyrosine kinase inhibitor (TKI) targeting ROS1 oncogenic fusions.
The candidate has been approved under the brand name Augtyro as an oral therapy. The approval is based on positive results from the TRIDENT-1 study that evaluated Augtyro in TKI-naïve and TKI-pretreated patients. Results showed Augtyro successfully achieved a high objective response rate and durable response.
The candidate was added to BMY’s portfolio with the acquisition of Turning Point Therapeutics in August 2022. The approval will add Augtyro to Bristol Myers’ growing and differentiated NSCLC portfolio, expanding the company’s presence in precision medicine.
Solid Biosciences Up on IND Clearance: Solid Biosciences Inc. (SLDB - Free Report) announced that it has obtained FDA clearance for an investigational new drug (IND) application for SGT-003, the company’s next-generation Duchenne Muscular Dystrophy (Duchenne) gene therapy candidate.
Consequently, SLDB plans to initiate a phase I/II study, SGT-003-101, to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. The study is a first-in-human, open-label, multicenter study. Solid Biosciences shares surged on the same.
SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients aged between four and six years of age with DMD. Long-term safety and efficacy will be evaluated for a total of five years following treatment.
Update From VRTX, CRSP: Vertex Pharmaceuticals Incorporated (VRTX - Free Report) and partner CRISPR Therapeutics (CRSP - Free Report) announced that the U.K. Medicines and Healthcare products Regulatory Agency (“MHRA”) has granted conditional marketing authorization to their CRISPR/Cas9 gene-edited therapy, exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The therapy has been approved under the brand name Casgevy for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises or TDT, for whom a human leukocyte antigen-matched related hematopoietic stem cell donor is not available.
The two global trials of Casgevy in SCD and TDT met their respective primary outcome of becoming free from severe VOCs or transfusion-independent for at least 12 consecutive months. These benefits are potentially expected to be life-long once achieved. Exa-cel was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.
Performance
The Nasdaq Biotechnology Index has lost 0.90% in the past five trading sessions. Among the biotech giants, Vertex has lost 7.33% during the period. Over the past six months, shares of Moderna have plunged 41.39%. (See the last biotech stock roundup here: Biotech Stock Roundup: BMYs Q3 Results, PTCT Up on Deal, BEAMs Restructuring News).
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What's Next in Biotech?
Stay tuned for more pipeline and earnings updates.
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Biotech Stock Roundup: BMY's Drug Approval, VRTX & CRSP Gene Therapy Get MHRA Nod
With the conclusion of the third-quarter earnings for the biotech sector, the focus is back on regular drug approvals and other pipeline and regulatory updates. Quite a few companies came out with important updates on their key candidates
Recap of the Week’s Most Important Stories:
Arcellx Surges on Deal Expansion: Shares of Arcellx, Inc. (ACLX - Free Report) gained after the company and partner Kite, a Gilead company, announced an expansion of their existing agreement. The companies collaborated in December 2022 to co-develop and co-commercialize Arcellx's CART-ddBCMA candidate for the treatment of patients with relapsed or refractory multiple myeloma.
Kite has exercised its option to negotiate a license for Arcellx's ARC-SparX program, ACLX-001, in multiple myeloma. The companies also expanded the scope of the collaboration for Arcellx's CART-ddBCMA to include lymphomas.
Per the terms of the agreement, Arcellx will receive an upfront cash payment of $85 million at closing and an equity investment of $200 million. It will also be eligible for potential milestone payments, including the advancement of lymphoma and the license for ARC-SparX, as well as additional milestones, to offset prespecified development costs over a limited period of time.
This infusion of cash is expected to extend Arcellx’s cash runway into 2027. Gilead will have an approximate 13% stake following this investment.
Bristol Myers’ Drug Approval: Bristol Myers Squibb (BMY - Free Report) announced that the FDA approved pipeline candidate repotrectinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). Repotrectinib is a tyrosine kinase inhibitor (TKI) targeting ROS1 oncogenic fusions.
The candidate has been approved under the brand name Augtyro as an oral therapy. The approval is based on positive results from the TRIDENT-1 study that evaluated Augtyro in TKI-naïve and TKI-pretreated patients. Results showed Augtyro successfully achieved a high objective response rate and durable response.
The candidate was added to BMY’s portfolio with the acquisition of Turning Point Therapeutics in August 2022. The approval will add Augtyro to Bristol Myers’ growing and differentiated NSCLC portfolio, expanding the company’s presence in precision medicine.
Solid Biosciences Up on IND Clearance: Solid Biosciences Inc. (SLDB - Free Report) announced that it has obtained FDA clearance for an investigational new drug (IND) application for SGT-003, the company’s next-generation Duchenne Muscular Dystrophy (Duchenne) gene therapy candidate.
Consequently, SLDB plans to initiate a phase I/II study, SGT-003-101, to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. The study is a first-in-human, open-label, multicenter study. Solid Biosciences shares surged on the same.
SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients aged between four and six years of age with DMD. Long-term safety and efficacy will be evaluated for a total of five years following treatment.
Update From VRTX, CRSP: Vertex Pharmaceuticals Incorporated (VRTX - Free Report) and partner CRISPR Therapeutics (CRSP - Free Report) announced that the U.K. Medicines and Healthcare products Regulatory Agency (“MHRA”) has granted conditional marketing authorization to their CRISPR/Cas9 gene-edited therapy, exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The therapy has been approved under the brand name Casgevy for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises or TDT, for whom a human leukocyte antigen-matched related hematopoietic stem cell donor is not available.
The two global trials of Casgevy in SCD and TDT met their respective primary outcome of becoming free from severe VOCs or transfusion-independent for at least 12 consecutive months. These benefits are potentially expected to be life-long once achieved. Exa-cel was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.
Performance
The Nasdaq Biotechnology Index has lost 0.90% in the past five trading sessions. Among the biotech giants, Vertex has lost 7.33% during the period. Over the past six months, shares of Moderna have plunged 41.39%. (See the last biotech stock roundup here: Biotech Stock Roundup: BMYs Q3 Results, PTCT Up on Deal, BEAMs Restructuring News).
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline and earnings updates.