It was a busy week for the biotech sector, with important treatment approvals and other pipeline updates. Quite a few companies offered major updates on their key candidates. Among these, Vertex’s (VRTX Quick QuoteVRTX - Free Report) shares hit record high on positive news from a phase II study on its pain candidate and gene-editing deal.

Recap of the Week’s Most Important Stories:

FDA Nod for bluebird’s SCD Therapy:  bluebird bio, Inc. (BLUE Quick QuoteBLUE - Free Report) obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (SCD) in patients aged 12 and older who have a history of vaso-occlusive events (VOEs). The FDA approved lovo-cel under the brand name Lyfgenia.

The therapy’s label is based on data from patients from the phase I/II HGB-206 study. Safety data supporting the application includes data from 54 patients who initiated stem cell collection. Efficacy of the gene therapy was backed by data from 36 patients in the phase I/II HGB-206 Group C study following enhancements to the treatment and manufacturing processes made through the course of the clinical development program.

Data showed 32 patients were evaluable for the endpoints of complete resolution of VOEs and severe VOEs in the 6-18 months post-infusion, including eight adolescent patients. Severe vaso-occlusive events were resolved in 94% of patients in this cohort and 88.2% experienced no VOE at all.

However, shares of bluebird were down despite receiving the FDA’s approval for lovo-cel. This is primarily because of the boxed warning of hematologic malignancy issued with Lyfgenia’s label.

Vertex, CRISPR Win FDA Approvals:  Vertex Pharmaceuticals Inc. (VRTX Quick QuoteVRTX - Free Report) and CRISPR Therapeutics (CRSP Quick QuoteCRSP - Free Report) announced that the FDA has approved Casgevy (exagamglogene autotemcel [exa-cel]) for the treatment of SCD for patients aged 12 years and older with recurrent vaso-occlusive crises (VOCs).  Per the companies, Casgevy became the first CRISPR-based gene-editing therapy to be approved in the United States.

Vertex is leading the global development, manufacturing and commercialization of Casgevy in partnership with CRISPR Therapeutics. Vertex will make a $200 million milestone payment to CRISPR following the FDA’s approval of Casgevy, which will be capitalized and amortized to the cost of sales.

Vertex also announced positive results from a mid-stage dose-ranging study of VX-548 in people with painful diabetic peripheral neuropathy (DPN). VX-548 is an investigational oral, selective NaV1.8 inhibitor that is highly selective for NaV1.8 relative to other NaV channels. Results showed that treatment with the NaV1.8 inhibitor VX-548 led to a statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the Numeric Pain Rating Scale (NPRS) at the end of week 12.

Secondary and other endpoints were supportive of the study’s primary endpoint. More than 30% of patients treated with VX-548 achieved more than 50% reduction in all dose groups, and more than 20% of patients in the mid-and high-dose groups achieved a greater than 70% reduction in the weekly average of NPRS at week 12 compared to baseline.

The candidate was generally well tolerated. Consequently, Vertex plans to advance VX-548 into pivotal development in diabetic peripheral neuropathic pain following discussions with regulators. Shares of Vertex surged on the impressive phase II results of this non-opioid pain drug, which, if successfully developed, will be a significant boost to VRTX’s portfolio, making the candidate an important alternative to otherwise available opioid pain drugs. The surge was attributable to a licensing deal with genome editing company, Editas Medicine (EDIT Quick QuoteEDIT - Free Report) .

CRISPR Therapeutics currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Editas, Vertex Collaborate: Editas Medicine entered into a license agreement with Vertex Pharmaceuticals. Per the terms, Vertex will obtain a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and beta-thalassemia, including the recently approved SCD therapy Casgevy.

The infusion of cash from Vertex from this license agreement extends Editas Medicine’s cash runway into 2026. Editas Medicine is the exclusive licensee of certain CRISPR patent estates for making human medicines.

The company recently announced new safety and efficacy data for 17 patients treated with EDIT-301, now known as enizgamglogene autogedtemcel (reni-cel), from RUBY and EdiTHAL studies.

In both the RUBY and EdiTHAL trials to date, reni-cel has been well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant in all patients. All RUBY patients have been free of VOEs since treatment with reni-cel, while EdiTHAL patients had an early and robust increase of total hemoglobin, above the transfusion independence threshold of 9 g/dl. The promising preliminary results underscored the potential of this gene therapy, particularly in the safety aspect. The company continues dosing additional patients and will share further updates in mid-2024.

ACAD Surges on Patent Ruling: Acadia Pharmaceuticals Inc. (ACAD Quick QuoteACAD - Free Report) announced that the U.S. District Court in Delaware passed a judgment strongly in favor of the company in its litigation against MSN Laboratories Pvt. Ltd., MSN Pharmaceuticals, Inc. and other abbreviated new drug application filers.

The court granted summary judgment to Acadia confirming the validity of the '740 composition of matter patent of lead drug for Nuplazid (pimavanserin). The ruling prevents generic drug manufacturers like MSN Laboratories from making low-cost generic versions of Acadia’s lead-marketed drug, Nuplazid.  The ‘740 composition of matter patent protects Nuplazid exclusivity until 2030. Acadia’s stock surged on the favorable court ruling. The company markets two forms of Nuplazid, a 34mg capsule and a 10mg tablet, which are also protected by issued patents. The 34mg capsule is protected by multiple issued formulation patents until 2038, while the 10mg tablet is protected by an issued method of use patent until 2037.

Performance

The Nasdaq Biotechnology Index has gained 5.96% in the past five trading sessions. Among the biotech giants, Vertex has gained 14.58% during the period. Over the past six months, shares of Moderna have plunged 38.58%. (See the last biotech stock roundup here: Biotech Stock Roundup: EXEL Partners RCUS for Study, PHVS & EYPT Gain on Study Data).

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What's Next in Biotech?

Stay tuned for more pipeline updates.