MedNess: bite-size biopharma and medtech news

FDA accepted IND application to begin clinical studies of RM-1995 in patients with advanced cutaneous squamous cell carcinoma or with SCCHN
“We are very excited to have achieved this regulatory milestone and delighted to be moving RM-1995 into the clinic,” said Mickey Mikitani, Chief Executive Officer of Rakuten Medical, Inc. “RM-1995 has the potential to attack the cancer in a novel way, targeting Tregs. Based on data from preclinical studies, RM-1995 photoimmunotherapy treatment is expected to result in acute killing of Tregs within the tumor. We are developing drugs using various antibodies in our drug discovery program based on Rakuten Medical’s IlluminoxTM platform, and RM-1995 is the second pipeline drug developed on this platform. We will continue to advance our business with a sense of speed so that we can deliver these important treatments to as many patients as possible.”

First Patient Dosed in Ph 1/2 Clinical Trial of Tipifarnib + Alpelisib Combination in Head and Neck Squamous Cell Carcinoma
“Despite the approval of immunotherapy, the treatment of recurrent and metastatic HNSCC remains a significant unmet need,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “The KURRENT trial builds on the impressive clinical activity reported for tipifarnib as a monotherapy in HRAS mutant HNSCC and represents an opportunity to significantly expand the potential patient population and target mechanisms of drug resistance.”

Generation Bio’s non-viral gene therapy for hemophilia A faces a setback
Generation Bio’s proprietary genetic medicine technology comprises a closed-ended DNA (ceDNA) delivered via a novel, cell-targeted lipid nanoparticle (ctLNP). In July 2021 the company announced that it would incorporate its novel, proprietary rapid enzymatic synthesis (RES) for ceDNA production into its pipeline programs which has since improved ceDNA purity to 99%. Generation Bio’s hemophilia gene therapy involves getting factor VIII (the clotting protein absent in hemophilia patients) to be expressed in the liver. Using its RES-derived ceDNA delivered by ctLNP in mice, the company was able to generate a 205% human factor VIII expression. But in their major clinical trial in monkeys which would eventually facilitate FDA to approve human clinical trials, only a 2% expression for the protein was seen. This is a far-off number from expectation. The company stated in its press release that it plans to optimize certain parameters to be able toa chieve similar numbers as those observed for mice preclinical studies.