Late last year, the Galien Foundation highlighted several drug discovery innovations in its annual Prix Galien USA Award Winners, which specifically highlighted drugs from Regeneron and Amgen as well as a platform from Exscientia and the incubators BioLabs and LabCentral.
The foundation recently hosted a webinar featuring several executives from the respective winning companies discussing their view on their respective drug discovery innovations.
1. Inmazeb: The first FDA-approved Ebola drug
The Galien Foundation chose Regeneron’s (Nasdaq:REGN) Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) as the best biotechnology product of 2022. The antibody cocktail became the first FDA-approved treatment for Ebola (Zaire Ebolavirus) for pediatric and adult patients in 2020.
There were several hurdles involved in developing Inmazeb, said Neil Stahl, EVP of R&D at Regeneron Pharmaceuticals. “The first thing we had to do was to make the antibody cocktail. We had 20 years of technology lined up behind us that allowed us to screen thousands of antibodies and make a three-antibody mixture and get it ready for the clinic in five months,” Stahl said.
In 2018, the company began shipping the then-investigational Ebola treatment to Congo for use in an outbreak. “The outbreak was in a very remote part of the Congo, which was rather violent at the time,” Stahl said. Researchers in Congo had to close two clinical sites due to invasions. “Nonetheless, this brave and dedicated group of clinical researchers did the study and compared our drug to three other drugs,” Stahl added.
The researchers stopped the study early after Regeneron’s investigational biotech cocktail surpassed ZMapp, a cocktail of chimeric monoclonal antibodies.
The development of Inmazeb was more of a humanitarian success than a commercial success, Stahl said. The U.S. government, however, paid for Regeneron’s supply of the drug.
“The benefit to patients was actually quite large,” Stahl said. “My favorite number is that if you got the drug before having symptoms for more than five days, it dropped the mortality from 65% to 10%.”
2. Why Lumakras is the best pharmaceutical agent of 2022
FDA granted accelerated approval to Lumakras for adults with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC) on May 28, 2021. Mutations of the Kirsten rat sarcoma (KRAS) gene are associated with various cancer types. Lumarkras was the first KRAS protein inhibitor to win regulatory backing.
“Before Lumakras was approved, patients living with KRAS G12C-mutated, non-small cell lung cancer had very few treatment options,” said Angela Coxon, vice president of oncology research at Amgen, in a webinar with the Galien Foundation.
The scientific community has spent some four decades working to identify KRAS inhibitors. “I’m really proud to say that the Amgen researchers uncovered how to make this elusive mutation druggable,” Coxon said.
Amgen is working on a broad KRAS G12C inhibitor development program active across five continents. “We’ve now got 10 different combination regimens that we’re pursuing, really trying hard to bring this molecule to as many patients with KRAS G12C as possible,” Coxon said.
To date, Amgen has treated more than 6,500 patients internationally with KRAS G12C in clinical development programs and commercial use.
3. Exscientia’s quest to select the right drug for the right patient
Exscientia plc (Nasdaq:EXAI) won the Prix Galien USA 2022 Award for Best Digital Health Solution for its AI-driven precision medicine platform.
The category was new for the Galien Foundation.
Exscientia has worked for about 10 years to develop its AI-based functional precision medicine platform. For the past five, the company has studied it clinically.
“Now, we’ve got the first drugs that have been generated by AI in clinical trials,” said Andrew Hopkins, CEO of Exscientia, in a Galien Foundation webinar.
While the pharma industry continues to explore the use of AI to fuel drug discovery, “the other big application has been, ‘How AI can help us select the right patient for a drug?’” Hopkins said.
Part of Exscientia’s mission is to contribute to the development of precision medicine. “We can use AI to precision engineer and design the right drug, but also, we can use it to help select the right patients,” Hopkins added.
Exscientia demonstrated the ability of functional precision oncology to improve patient outcomes in the landmark EXALT-1 prospective interventional study focused on patients with late-stage hematological cancer patients.
Researchers involved analyzed tumor samples, exploring a single-cell functional precision medicine approach to guide treatment selection. “We could truly then identify which drug actually would help the right patients,” Hopkins said.
The patients who joined the study had already failed at least two lines of therapy. About a quarter of the group of patients who received a drug selected by AI algorithms were progression-free survivors almost four years after the beginning of the study. Hopkins says the EXALT-1 study is likely the first example showing that an AI system can improve outcomes in oncology.
4. BioLabs’ and LabCentral’s push to democratize early-stage drug discovery
The Galien Foundation selected BioLabs and LabCentral as winning drug discovery innovations in their new “Incubators, Accelerators and Equity” category.
Johannes Fruehauf, the founder, president and executive director of LabCentral, noted that the new category “follows the reality of drug development.”
Cambridge, Massachusetts–headquartered LabCentral is a non-profit company founded in 2013.
Drug discovery innovations represent a collaborative activity that is difficult for single companies to fulfill alone. “We represent the innovators here that are often in distributed small entrepreneurial teams,” Fruehauf said. “These are academics converted into entrepreneurs taking their ideas forward.”
Founded in 2012, LabCentral has “brought about a change in the way that people build biotech companies,” Fruehauf said. “It’s now much faster. It’s much cheaper. You don’t have to own a lab in the first year or two of your existence, and you can check the scientific hypothesis before you spend large amounts of dollars.”
LabCentral offers a membership-based network of shared labs in biotech clusters in the U.S. and Europe.
To date, LabCentral has aided the discovery of 80 drugs that are now in clinical trials.
“We cheer on our innovators, and we try to give them the best environment to be successful — to be faster, more capital efficient and have fun while they’re doing this important work,” Fruehauf said.