Aetion to collaborate with UK's NICE on RWE; 4D's cystic fibrosis gene therapy cleared for clinical trials
New York-based real world evidence, or RWE, firm Aetion is teaming up with the National Institute for Health and Care Excellence in the United Kingdom to analyze data such as hospital records, prescription records and more to study clinical effectiveness and fill gaps in evidence.
As Aetion CEO Carolyn Magill told Endpoints News, the company’s goal is to collect evidence that would be harder to get outside of a controlled setting, such as a clinical trial.
“So [in a clinical trial] you have a nurse tapping you on the shoulder saying, ‘Did you take your medication today? Did you always take it at the same point in time?’ Whereas once the drug is on the market, our regular lives get in the way,” Magill said.
Magill said that RWE helps capture what happens to populations who may be underrepresented in clinical trials — such as women of childbearing age, children, and those who have different combinations of chronic illnesses.
In April, after talking for several years, representatives from NICE and Aetion worked alongside representatives from the EMA and co-published a paper on a possible structure for organizing fragmented RWE recommendations worldwide to achieve comprehensive guidance. That collaboration marked Aetion’s first partnership with an international HTA body, according to a company statement announcing the collaboration.
“As NICE continues to deliver on our five-year strategic vision, including rapid, robust, and responsive technology evaluation, understanding how to make the best use of RWE is vital in helping us resolve gaps in knowledge and driving forward access to innovations for patients,” Páll Jónsson, NICE’s programme director for data, said in a statement.
4D’s aerosolized cystic fibrosis gene therapy cleared for human trials
Biotech 4D Molecular Therapeutics said today that their investigational, cystic fibrosis gene therapy 4D-710 has been cleared by the FDA for clinical trials with an approved IND.
4D expects to be able to start a Phase I/II clinical trial with approximately 18 patients before the end of the year.
“Ultimately, we believe 4D-710 has the potential to treat a broad cystic fibrosis patient population, including those patients treated with current CFTR modulators, all of which require daily dosing over the patient’s lifetime and generally result in only partial correction of lung function,” said 4DMT CEO and co-founder David Kirn in a statement.
This announcement comes just a few months after Roche severed their partnership with 4D, resulting in shares of $FDMT tanking 10%.
The non-profit Cystic Fibrosis Foundation will purchase 125,715 shares of 4D’s common stock for further development of the drug for approximately $4 million, according to a company statement.
CRO Inotiv buys out Plato BioPharma in $15 million deal
Indiana-based CRO Inotiv is acquiring in vivo research firm Plato Biopharma as part of an in vivo pharmacology expansion, according to a company announcement today.
In a $15 million deal with $10 million in cash, along with common shares and promissory notes making up the other $5 million, Inotiv expects to retain all 34 of Plato’s employees after the transaction is complete.
“The acquisition of PBI marks another milestone for Inotiv as we continue to execute on our strategy to build a complete range of contract research services,” said Inotiv chief strategy officer John Sagartz. “This addition allows us to support and accelerate PBI’s growth plans, while further expanding into more physiologic and pharmacologic offerings.”
“We look forward to joining the team and broadening Inotiv’s service offering,” Plato founder and CEO Craig Plato said in a statement. “Our core values and collaborative approach align well, and our focus on rigorous scientific execution and dedication to delivering exceptional client experience will continue to be paramount.”
Xi Jinping calls for improved and increased biosecurity measures against zoonotic disease transmission
Chinese President Xi Jinping called for “active defenses” last week to prevent animal-to-human transmission of diseases during his address to the Political Bureau of the CPC Central Committee.
In a group session on biosecurity, Xi, who is also the general secretary of the Communist Party of China (CPC) Central Committee, called for a management network to respond to any potential diseases that might arise in the future.
He also stressed more supervision over the safety of biological resources. Specifically, Xi pointed out a need to strengthen examination of imported goods, and urged punishment for all illegal acts.
Calling the buildup of biosecurity “a long-term and arduous task,” Xi urged continual efforts and promised to take part in world biosecurity governance.
EQRx to collaborate with newly-public Absci on protein-based therapeutics
EQRx announced a discovery collaboration with Absci today to advance protein-based therapeutics.
Absci went public just a few months ago in July, where it raised $200 million at $16 a share.
The two companies will collaborate to jointly engineer and develop several clinical candidates across multiple therapeutic areas, including oncology and immunology. Absci has the option to make additional investments at different stages of development in exchange for an increased share of product sales.
“We are excited to work with Absci towards our goal of providing innovative, cost-effective treatment options for patients,” said EQRx chief of Rx creation Carlos Garcia-Echeverria in a statement.