A mixed bag of top-line phase II data prompted stellar stock results for Prokidney Corp. and its chronic kidney disease (CKD) and diabetes therapy, rilparencel. There were statistically significant and clinically meaningful top-line data from Group 1 showing participants’ annual kidney function decline slowed by 78% in those receiving two doses, one in each kidney. In Group 2, composed of patients who received one injection and only received another if their kidney function worsened, the annual decline in kidney function improved by 50%, which was not statically significant. The market took to the results as the company’s stock (NASDAQ:PROK) was up 402% at $3.05 per share.

Taiho’s pizuglanstat fails in phase III DMD trial

In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III Reach-DMD trial, a randomized, placebo-controlled, double-blind and open-label, extension study of pizuglanstat in patients with DMD showed no significant difference in the mean change from baseline to 52 weeks in the primary endpoint of time to rise from the floor in the ambulatory cohort of the study. A selective hematopoietic prostaglandin D synthase (HPGDS) inhibitor, pizuglanstat was discovered internally by Taiho and was being developed as a DMD treatment that could potentially be used regardless of the dystrophin gene mutation type. It was hoped it would control the decline in motor function in DMD patients by inhibiting HPGDS, which exacerbates the inflammatory response in DMD patients’ muscles.

BAFF/APRIL mechanism still hot among industry, academics

Aurinia Pharmaceuticals Inc. recently unveiled positive results from the phase I single ascending-dose study with aritinercept (AUR-200), the company’s dual inhibitor of B cell-activating factor (BAFF) and a proliferation-inducing ligand (APRIL). The experiment tried doses of 5 mg, 25 mg, 75 mg, 150 mg, 225 mg and 300 mg and placebo, given by subcutaneous injection, in 61 healthy subjects. Interest continues to grow among researchers across the board who are taking aim at B cell-mediated diseases via the BAFF/APRIL pathway.

UK industry encourages renewal of rare disease framework

Representatives of patients’ groups, industry bodies and venture philanthropy funders are calling for a renewal of the U.K. Rare Diseases Framework, to put fresh momentum behind translational research and clinical trials, streamline regulatory oversight and improve access to therapies. Pioneering science has led to “incredible breakthroughs” for some people with rare conditions, creating hope for others. But as things stand, huge hurdles sit between the promise of R&D and diagnoses and therapies for the vast majority of patients, concludes a new report looking at how to accelerate development of diagnostics and treatments for rare diseases.

With surface mimicry, molecular glues shed hairpin need

Degradation is a therapeutic strategy that could offer possibilities to get at currently undruggable target proteins. In targeted degradation, compounds induce interactions between a target protein and a protein that can tag the target for degradation. In principle, there are several pathways that could be used for such tagging; the most attention has gone to ubiquitin ligases, in particular cereblon, a protein that is part of a ubiquitin ligase complex and the target of several approved drugs.

Veritas, Mitsubishi Gas Chemical partner for new ASO candidates

Veritas In Silico Inc. (VIS) entered a joint research agreement with Mitsubishi Gas Chemical Co. Inc. (MGC) to use VIS’s mRNA structural motif analyzing platform technology, called Ibvis, to develop antisense oligonucleotide (ASO) drug candidates. The three-year R&D collaboration also entails the development of a manufacturing method for the potential ASO therapies.

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