July 19, 2023
Barry Bunin, PhD
Founder & CEO
Collaborative Drug Discovery
Drug Discovery is Fueled by Small Companies. That’s the take-home message that Derek Lowe provides in his recent blog for Science, titled “The Drugs of 2015 to 2021.” He points to an article in Nature analyzing drug approvals (both small molecules and biologics) from the 20 largest drug companies during 2015-2021, looking for trends in where new drug discoveries have been coming from. Lowe notes that of 323 FDA approvals during the period, 138 came from the 20 largest companies, while 165 came from small companies with solo approvals. Taking a closer look at the 138 approvals granted to the 20 largest companies, he notes: “A key number is that 90 of those 138 approvals (65%) came from somewhere else than the company that filed them—some (40% of the 90) were from acquisitions of smaller companies, while others were from collaborations and in-licensing. And since I always get asked about this, only 7% of the total approved drugs from the top 20 companies had academic origins. … So it’s safe to say—and it’s been safe to say for quite a while now—that the business model of the big drug companies is to come up with what they can on their own, but to rely on outside compounds for the majority of their new approvals.” See the Pharmaceutical KnowledgeBase (see: PharmaKB.com) for more extensive approved and clinical drugs’ competitive financial and scientific data.
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“Eli Lilly’s Experimental Alzheimer’s Drug Slows Disease, Data Show.” That’s the headline from The Washington Post report on Donanemab, and how the once-a-month treatment administered intravenously, slowed cognitive and functional decline by about 35 percent, compared with a placebo, over 18 months, according to data published in JAMA, the peer-reviewed journal of the American Medical Association. Company officials said the results showed the benefits of treating Alzheimer’s as early as possible. The New York Times reports: The trial outcomes were “the most positive we have seen,” said Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation, a nonprofit that promotes research on treatments for Alzheimer’s. He said therapies that target amyloid, as Donanemab does, “will serve as the first line of defense in the arsenal of drugs needed to treat this disease,” and he predicted the Food and Drug Administration would approve the medication within months. FIERCE Biotech in an article headlined “Lilly Reveals How Alzheimer’s Drug Shapes Up Against Leqembi” writes “Improvements over placebo continued to grow even after patients stopped taking the drug, but safety and certain subgroups are concerns.”
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“A One-Time Shot for Type 2 Diabetes? A Biotech Company Is On It.” That’s the from WIRED about Fractyl Health which is developing a gene therapy alternative to drugs like Ozempic and Wegovy to control blood sugar and body weight without repeated injections, and thus hoping to prevent this with a one-and-done treatment that could last for years. The company wants to deliver an artificial gene to the pancreas that continuously produces the GLP-1 hormone so there’s no need for weekly injections. Working with mice bred to develop type 2 diabetes, scientists gave a single infusion of the gene therapy to one group and weekly injections of semaglutide to another. After 10 weeks, they found that the gene therapy decreased fasting blood sugar by 70 percent, slightly more than semaglutide, which lowered blood sugar by 64 percent. Scientists with the company presented the findings at the American Diabetes Association conference at the end of June, along with separate findings that the therapy also reduced body weight in mice by 23 percent compared to control mice. There are plenty of questions, though. WIRED quotes Daniel Drucker, an endocrinologist and professor of medicine at the University of Toronto, who is skeptical about the company’s plans for injecting the therapy directly into the human pancreas. “The pancreas is a very fragile and important organ,” Drucker said. “If it’s poked or prodded, it can induce inflammation.” The article also notes the potential expense of gene therapies. But a one-and-done, if effective and affordable, would be a step forward. The company is said to be aiming to begin an initial human trial by the end of 2024.
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World Health Organization Names Aspartame a Possible Cause of Cancer in Humans. The World Health Organization’s International Agency for Research on Cancer has declared that aspartame, an artificial sweetener widely used in diet drinks and low-sugar foods, could possibly cause cancer, though the FDA and a second WHO committee defended the sugar substitute as safe, according to The New York Times. Dr. William Dahut, Chief Scientific Officer of the American Cancer Society, which led one of the key studies the WHO relied on, said the findings should be considered alongside the WHO’s report earlier this year that indicated artificial sweeteners offered no help in achieving weight loss or protection from other chronic conditions.
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AI-Enabled Study Design. While there’s been plenty of writing about AI-powered drug discovery, Forbes carries an article titled “AI Poised To Revolutionize Drug Development,” that points to how AI can help with clinical trials protocol design and automated data collection. The author writes: “AI-enabled study design could help optimize and accelerate the writing of appropriate targeted clinical trial protocols. It would promise to decrease the number of amendments, increase the likelihood of success, and improve overall efficiencies, in addition to reducing patient burden. AI algorithms can analyze historical clinical trial data to identify potential areas for protocol optimization, such as selecting appropriate endpoints, sample sizes, and study durations. By leveraging AI's ability to analyze complex data, researchers can design more efficient and informative trials.” AI can also help develop innovative ways of collecting trial data and reducing the need for patients to visit hospital sites. “Body sensors and wearable devices such as bracelets, heart monitors, patches, and sensor-enabled clothing, can monitor vital signs and other information from patients’ homes. Advanced algorithms would help reveal real-time insights into study execution and patient adherence.” The article even referred to a study by the FDA titled “Artificial Intelligence and Machine Learning (AI/ML) for Drug Development.”
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Barry A. Bunin, PhD, is the Founder & CEO of Collaborative Drug Discovery, which provides a modern approach to drug discovery research informatics trusted globally by thousands of leading researchers. CDD Vault® is a hosted biological and chemical database that securely manages your private and external data.