MedNess: bite-size biopharma and medtech news

EMA Grants Orphan Drug Designation to CPI-613® (Devimistat) for Treatment of Patients with Relapsed or Refractory Burkitt's Lymphoma
“Relapsed or refractory Burkitt’s lymphoma can be a devastating form of cancer that is in need of effective treatment options,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to.”

SAMETA Global Ph 3 Trial of Savolitinib in Combination with PD-L1 Inhibitor IMFINZI® initiated in Patients with MET-Driven Advanced Papillary RCC

• SAMETA global Ph 3 study of savolitinib (ORPATHYS® in China), an oral, potent, and highly selective small molecule inhibitor of MET, a receptor tyrosine kinase, in combination with IMFINZI® (durvalumab) in patients with MET-driven advanced papillary renal cell carcinoma (“PRCC”).
• The first patient received their first dose on October 28, 2021.
• The Phase III trial is an open-label, randomized, controlled study in treatment-naïve patients with MET-driven, unresectable and locally advanced or metastatic PRCC, to evaluate the efficacy and safety of savolitinib in combination with IMFINZI®, compared to single agent IMFINZI® or single agent SUTENT® (sunitinib), an oral multi-kinase inhibitor considered the standard-of-care treatment option in PRCC. The primary endpoint of the study is median progression free survival (“PFS”). Other endpoints include median overall survival (“OS”), objective response rate (“ORR”), duration of response (“DoR”), 6-months and 12-months disease control rate (“DCR”), time to second progression (PFS2), safety, pharmacokinetics (“PK”) and quality of life.
• Additional details may be found at clinicaltrials.gov, using identifier NCT05043090.

Mammoth Biosciences and Vertex Pharmaceuticals enter collaboration to develop gene therapies
Vertex Pharmaceuticals entered a collaboration with Mammoth Biosciences to use its proprietary ultra-small CRISPR systems to discover and develop novel in vivo gene-editing therapies for two undisclosed genetic diseases. Mammoth’s CRISPR platform consists of a proprietary toolbox of novel, ultracompact Cas enzymes, including Cas14 and Casɸ. The small size of these Mammoth systems, together with further optimized parameters, have the potential to facilitate advanced delivery, which may increase the scope of in vivo gene-editing for genetic diseases.

Merck signed a voluntary licensing agreement to Let Other Drug Makers Make Its COVID Pill
Recently, Merck and its partner Ridgeback Biotheraputics announced that the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) has granted authorization in the United Kingdom (U.K.) for Molnupiravir, the first oral antiviral medicine authorized for the treatment of mild-to-moderate COVID-19 in adults with a positive SARS-CoV-2 diagnostic test and who have at least one risk factor for developing severe illness. Molnupiravir cut hospitalizations and deaths by half among patients with early symptoms of COVID-19. Merck announced its application with the U.S. Food and Drug Administration (FDA) for Emergency Use Authorization (EUA) of molnupiravir is under review and decisions that could come within weeks.
As part of its commitment to widespread global access, Merck announced that it has entered into a licensing agreement with the Medicines Patent Pool to increase broad access for molnupiravir in low- and middle-income countries. The agreement will allow the Medicines Patent Pool to grant further licenses to qualified companies who are approved to make the drug. Neither drug maker will receive royalties under the agreement for as long as the World Health Organization deems COVID-19 to be global emergency. Molnupiravir is the first pill that has been shown to treat the disease. Charles Gore, the executive director of the Medicines Patent Pool, said the early results for molnupiravir were “compelling” and that he hoped this first voluntary licensing agreement for a COVID-19 treatment would lead to others.
While the competitor Pfizer claims its Covid-19 pill reduces the chance of hospitalization and death by 89 percent, though has not mentioned anything about the licensing agreement yet. Despite repeated requests from governments and health officials, no vaccine makers have agreed to a similar deal.