Esperion releases positive PhII results; Safety warnings at India, China manufacturing sites spell trouble for global supply chain
→ After lining up a new $200 million funding deal for the projected commercial launch of bempedoic acid next year, Esperion announced today positive results from their Phase II randomized, double-blind parallel-group study of bempedoic acid/ezetimibe combination tablet in comparison to ezetimibe and placebo. 179 patients with hypercholesterolemia and type II diabetes were enrolled. The 12-week study met its primary and secondary endpoints.
→ The Pharmaceutical Journal has conducted an analysis that shows the majority of warnings from European and US drug regulators are to manufacturing sites in India and China. The data indicate from early 2018 until August 2019 that the FDA’s office of manufacturing quality published 75 warning letters to pharmaceutical manufacturers that violated its safety and/or quality standards (49% of which could result in enforcement action were sent to companies in China or India). Similarly, the EMA issued 22 compliance notices, of which 14 (64%) were for manufacturers in India or China.
Cases such as the recalling of thousands of batches of valsartan-containing products that were contaminated with potential carcinogens in June 2018 from two factories in China and the 2016 factory explosion in China resulting in severe shortages of the API for piperacillin-tazobactam are raising concerns of the global supply chain from experts.
“Where I am concerned is the concentration in production of APIs — that is what should keep people awake at night,” says Warwick Smith, director-general of the British Generic Manufacturers Association. “If you only have one or two manufacturers for APIs for a critical product, that is a real weakness in the supply chain.”
A parliamentary report found that India and China are among the top five providers of UK medicines outside the EU. A spokesperson for the Medicines and Healthcare products Regulatory Agency (MHRA) said that it was not owing to lower standards that these manufacturers were receiving such high numbers of notices, but because of their larger market share and that there isn’t a significant difference in the number of serious non-compliance issues identified at UK site in comparison to non-EU countries.
“According to data provided by the MHRA, 5% of inspections of manufacturers in India found “critical deficiencies” in 2017 and 2018, compared with 4% of manufacturers in the UK. However, the data also revealed that 6% of inspections of manufacturers in India resulted in the regulator issuing a statement of non-compliance (SNC). This is compared with 0.8% of inspections of manufacturers in the UK.”
→ Cambridge, Massachusetts-based Harbour BioMed is teaming up with CRO Pharmaceutical Product Development (PPD) to develop Harbour’s therapeutics in the fields of oncology and immunology. The two companies recently completed an Australian ethics committee submission for HBM4003, an anti-cancer immunotherapy to target solid tumors — with a plan for Phase I trials in Australia and the US.
→ Bain-backed SpringWorks — which closed a series B round in April, is now gunning for an IPO — has scored breakthrough therapy status from the FDA for its experimental drug, nirogacestat, which is a gamma-secretase inhibitor under development to help patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.
→ Soligenix $SGNX has updated the late-stage program for its experimental drug — SGX942 — for mucositis, which is characterized by damage done to the mucosa by anticancer therapies. On Wednesday, the company said an independent data monitoring panel recommended it increase enrollment by 70 patients to increase the total to 260 subjects. The panel’s recommendation indicates that “a beneficial SGX942 effect has been observed — however, to maintain the rigorous assumption of 90% statistical power for the primary efficacy endpoint, an increase was required to take into account any potential variability and/or distribution changes observed in the Phase 3 study patient population that may have differed from the initial protocol design assumptions,” Soligenix said. Topline results from the study are still expected in the first half of 2020, it added.