GBT strikes a deal with Sanofi; Lilly’s IL-23 inhibitor shows promise; Glooko raises $30M; FDA uplifts hold on Solid’s DMD gene therapy

Posted on Mar 18, 2021 by Delveinsight

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Global Blood Therapeutics strikes a deal with Sanofi for sickle cell disease programs

As Global Blood Therapeutics works to bring its sickle cell disease drug, Oxbryta, to younger patients, the company is reinforcing its pipeline with a pair of early-stage programs from Sanofi.

In exchange for the global rights to the assets, Sanofi will get up to USD 353 million, including a USD 2.25 million upfront fee and potential development, regulatory and commercial milestone payments down the line as per securities filing.

The programs, licensed from Sanofi’s Bioverativ unit, work differently than Oxbryta, which halts the polymerisation, sickling, haemoglobin S, and abnormal type of the oxygen-carrying protein observed in sickle cell disease patients. 

One of the Sanofi programs utilises a different anti-sickling mechanism than Oxbryta, while the other uses a new approach to decrease inflammation and oxidative stress. These mechanisms could be complementary to that of Oxbryta, suggesting a potential for combinations as per the statement by GBT.

Eli Lilly’s IL-23 inhibitor mirikizumab shows late-stage promise in Ulcerative Colitis 

A phase 3 clinical trial of mirikizumab in ulcerative colitis has touched its primary endpoint, enhancing Eli Lilly’s plans of establishing the anti-IL-23p19 antibody in gastrointestinal disorders.

Lilly embarked on a wide R&D program that set it to position mirikizumab as a late entrant to the psoriasis space and an initial mover in gastrointestinal disorders. The top-line phase 3 results proffer partial confirmation of Lilly’s big stake in mirikizumab. In LUCENT 1, Lilly compares intravenous mirikizumab to placebo in patients with moderately to severely active ulcerative colitis who had been missed by conventional or biologic therapies.

After 12 weeks, the clinical remission rate was remarkably higher in the mirikizumab arm, causing the trial to touch its primary endpoint with a p-value of less than 0.0001. Subjects were classed as being in clinical remission when the control or resolution of colon inflammation led to the normalisation, or near-normalisation, of symptoms such as stool frequency and bleeding. 

Mirikizumab also smashes placebo against all key secondary endpoints, including bowel urgency and endoscopic remission with highly statistically significant p-values. Symptoms halted as early as four weeks after treatment. The responders to mirikizumab included patients who failed by JAK inhibitors and biologic therapies.

The top-line readout cues mirikizumab improves outcomes in ulcerative colitis patients who have exhausted current treatment options. However, the absence of numbers in the Lilly release makes it difficult to reckon how grand an impact mirikizumab could have on the market. 

Glooko raises USD 30 Million to expand digital diabetes management 

Digital diabetes management developer Glooko has raised USD 30 million to continue its growth, fueled by increasing demands for telehealth programs plus multiple partnerships maintained with health systems and biopharma industry companies over the past year.

Its software garners data from glucose meters and continuous monitors and from insulin pumps, pens and wearable activity trackers, which Glooko began proffering for free in early 2019. The company currently lists more than 3 million users in 27 countries.

Health Catalyst Capital led the Series D round with participation from its previous backers, Canaan Partners, Georgian, Novo Nordisk, Insulet and the Mayo Clinic. The proceeds will be slated to expand Glooko’s remote patient monitoring services spanning diabetes and obesity to other chronic conditions as well as to new offerings in clinical research.

FDA uplifts clinical hold on Solid Biosciences’ DMD gene therapy trial 

Solid Biosciences announced the U.S. Food and Drug Administration (FDA) lifted the clinical hold on its IGNITE DMD Phase I/II clinical trial.

The trial was formerly placed on hold in November 2019 due to safety concerns. A patient experienced a severe adverse event related to SGT-001, a novel adeno-associated viral vector-mediated gene transfer. Solid made various attempts to address the clinical hold, comprising adding measures to boost patient safety and updated manufacturing process improvements. In July of this year, the FDA asked for further manufacturing data, updated safety and efficacy information for all patients dosed, and direction on total viral load to be administered per patient. Solid Biosciences provided the requested information to the regulatory agency.

This week, the FDA apprised the company satisfactorily addressed all clinical hold questions and offered Cambridge, Mass.-based company the go-ahead to restart the study.

SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to direct the underlying genetic cause of Duchenne muscular dystrophy caused by mutations in the dystrophin gene, which led to the scarcity of dystrophin protein. SGT-001 delivers microdystrophin, a synthetic dystrophin gene that encodes for a functional protein surrogate expressed in muscles and stabilises vital associated proteins.

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