Vertex leads in CRISPR Therapeutics partnership with USD 900 Million upfront

Vertex has boosted a deal with CRISPR Therapeutics to USD 900 million upfront as the companies compete against bluebird bio to the market with a novel gene-editing therapy for sickle cell disease and beta-thalassemia called CTX001.

Boston-based Vertex will reimburse CRISPR USD 900 million upfront with a potential for USD 200 million in milestone payments if the therapy is granted. Vertex will be responsible for 60% of the program costs and receive the same in profits from future global sales of CTX001. The new deal is a 10% increase from the former agreement.

CTX001 is under development as a potential cure for the blood diseases sickle cell disease and transfusion-dependent beta-thalassemia.

Vertex and CRISPR are currently in a race against bluebird bio to market a new treatment option for patients with the two conditions. Whereas, bluebird bio is ahead in its regulatory plans for Zynteglo, with already European approval. bluebird also announced that German cost regulators could not find a price for its gene therapy that bluebird would agree to, so it has been pulled into that market.

KalVista Pharmaceuticals provides a regulatory update for the Phase 2 clinical trial 

KalVista Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) has notified them regarding a clinical hold on the submitted Phase 2 clinical trial of KVD824.

KVD824 is KalVista’s oral product candidate being developed for prophylactic treatment of hereditary angioedema (HAE). An Investigational New Drug Application was submitted earlier in 2021 for a Phase 2 clinical trial to assess KVD824 as a potential prophylactic treatment to prevent HAE attacks. The FDA letter demands further information and analysis related to specific preclinical studies of KVD824 submitted to bolster the planned Phase 2 trial. Refinements were also proposed to the required KVD824 Phase 2 study protocol. Neither new studies were asked, nor did it suggest that new data be generated to commence the Phase 2 trial.

Andrew Crockett, Chief Executive Officer of KalVista said that they intend to fully comply with the requests and recommendations provided by the FDA. KalVista has formerly reported data from first-in-human and formulation studies of KVD824 that were conducted in the UK. A total of 121 subjects have received KVD824 as single doses up to 1280 mg and up to 14 days of twice-daily dosing of 600 mg and 900 mg. In both studies, adverse event rates were similar in the placebo and active arms, no subjects withdrew, and no serious adverse events were observed. 

KalVista will pursue to work closely with the FDA on the overall development of KVD824 and will give further updates as appropriate.

Ovid dumps lead program after dull results showing in Angelman syndrome

Ovid Therapeutics is pulling the plug on its lead program, OV101, a treatment under development for Angelman syndrome and Fragile X syndrome. Now, the company’s zeros in on its earlier-stage pipeline.  

Ovid CEO Dr Jeremy Levin said that they are disappointed with the outcome for OV101. Ovid’s earlier-stage pipeline comprises OV882, a research-stage program in Angelman syndrome that works differently than OV101. While the latter functions by activating the GABAA receptor, the former is a gene-silencing treatment based on short hairpin RNA. The company is also working on research-stage genetic therapies for neurological disorders linked to mutations in the KIF1A genes and a preclinical-stage GABA aminotransferase inhibitor for seizures related to tuberous sclerosis complex, a genetic condition that causes benign tumors to form in several parts of the body, and infantile spasms. 

OV101, also called gaboxadol, has had a difficult path. The company reported in 2018, phase 2 data from 88 patients showing that after 12 weeks of treatment, the drug beats placebo on one measure but flunked to do so on 16 other metrics. Ovid’s stock price nosedived more than 30% that day, but where investors saw 16 missed endpoints, the company found many lessons. 

As Ovid designed the phase 2 study, the company met with patients and families and experts to distil more than 200 tools to gauge symptoms down to 17 endpoints, Levin told in an interview.

Virta Health pulls in USD 133 Million to expand its diabetes reversal platform

Virta Health, a startup that proffers a program for diabetes reversal, has banked USD 133 million of equity financing led by Tiger Global.

The Series E financing boosts the company’s valuation to USD 2 billion from USD 1.1 billion back in December, as per the company.

San Francisco-based Virta Health wishes to use the financing to scale its virtual metabolic clinic and approach reversing chronic metabolic conditions, including type 2 diabetes, prediabetes, and obesity.

The fresh capital will bolster support growth in research and product innovation and enable Virta’s commercial team to meet the demand for diabetes reversal from employers, health plans, and government, as told by executives. 

Sami Inkinen, Virta Health co-founder and CEO, said that the norm in diabetes care has been of more medications, more weight gain, and more suffering, but Diabetes reversal changes this. They have paved the way for people to live medication and diabetes-free, and this investment is about reaching even more people and taking diabetes reversal mainstream.

According to company executives, the investment in Virta Health and the company’s continued growth signals a shift in how chronic diseases are considered and treated.

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