Pharmaceutical R&D bets: picking pipeline winners in a risky field

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In an industry that frequently strives for breakthroughs, only a few winners emerge from the many attempts at the finish line.

And as markets embark on a “fragile recovery,” investors are looking for the least risky drug candidates and companies with the strongest data.

“We’re in a high-cost environment, which is driving risk aversion, so clinical data remains important,” said Andy Seid, a partner at Bioluminescence Ventures.

But prudent spending isn’t just for startups. Big Patent Cliff Looming Price Negotiation As the IRA kicks in, big pharmaceutical companies are also trying to cut costs, with layoffs and restructuring being the usual tactics, but pipelines are also frequently the target of cuts.

In April, Bristol-Myers Squibb Ban experimental drugs Roche also pulled new drugs from its pipeline as part of a $1.5 billion cost-cutting plan. One-fifth of the pipeline We cut operating expenses last year.

As companies face financial pressures, making strategic pipeline decisions is critical to lay the foundation for growth. But with so many drugs ultimately failing in development, how can company leaders determine whether their pipeline will be successful?

PharmaVoice asked leaders attending BIO’s annual international conference this week what criteria they use to make these tough choices. Whether they’re evaluating a potential new drug or considering investing in a partnership, here’s how they make those decisions.

Don’t miss out on highly processed targets

Dr. Gavin Samuels, chief operating officer of hub-and-spoke biotech company CinRX, owns several “CinCos,” including CinCor Pharma, which was recently acquired by AstraZeneca. Trading specializing in hypertension It’s worth up to $1.8 billion.

“Because we’re broad in our therapeutic areas, we have certain criteria for evaluation. It has to do with IP, robustness of data, signs of differentiation. It’s nothing unusual. But we’re trying to do something a little bit different, which is to find candidates that are not obvious, for a broad audience that is not obvious.

CinCor is a good example. The conventional wisdom was that hypertension wasn’t of interest to biotech companies because there were generic drugs on the market already. But a lot of heart disease patients weren’t reaching their treatment goals and their problem wasn’t being solved. So when we looked at the drug CinCor, an aldosterone synthase inhibitor made by Roche, we thought this might be the missing piece that could get a lot of patients closer to their treatment goals. Everyone else was getting out of hypertension, but we got involved, and through our IPO, our acquisition by AstraZeneca, and the data being generated in Phase 2, there was agreement that this was the case.”

Using high technology for early detection

Anthony Mancini, executive vice president and chief operating officer of antibody specialist Genmab, recently entered the ADC space. I picked up ProfoundBio It was acquired in April for $1.8 billion.

“We have great confidence in our candidate selection process because we use robotics to run through thousands of candidate combinations. Now we are integrating AI to further enhance, automate and speed up our work.”

From a discovery perspective, that’s not the norm. Often the configuration is done manually by smart postdocs. But the reason we’re so productive is because of this core science. It’s given us a 25% hit rate from IND to approval, which is typically less than 10%. We think that’s part of our secret sauce.”

Aiming for highly effective medicines and win-win partnerships

Stacey Feld, regional head of North America West for Johnson & Johnson Innovation. Feld is responsible for working with the external life sciences ecosystem to find what she calls “transformative assets” through partnerships and co-investments and inject them into J&J’s pipeline.

“Ultimately, data wins. We evaluate the transformative nature of science and technology and we focus on best-in-class or best-in-class assets and platforms. So that transformative element is one of the factors we use to evaluate deals.”

Also consider whether the team is trustworthy. How do you structure it based on that. If you’re talking about a platform and the company doesn’t necessarily have data in a particular area, suggest an experiment to validate it.

We are also committed to a belief: Is the relationship fair? In other words, are we each getting value from the relationship? It doesn’t necessarily have to be equal. It depends on our respective risks. But we want our companies and assets to be successful and to be able to bring them to market and to patients around the world.”

Trust senior staff

Bill Newell, CEO of Sutro Biopharma, a clinical-stage ADC specialist company with a lead candidate in mid-stage trials for ovarian cancer;

“We need to see if there is an unmet need in a particular patient population. We don’t always know, so we need to consider other molecules that may be in or developing. Through preclinical studies, we determine if it is significantly better than existing therapies, look at tolerability risks and see if there are side effects that can be managed. Whenever possible, we compare our preclinical data with that of other companies working on the same type of target, and that feeds into our assessment of feasibility and whether it will be best-in-class or first-in-class.”

(But) I have a very experienced CSO (Hans-Peter Gerber) who has the ability to see winners. He has been in ADCs for 25 years at Siegen and other companies. The ADC field has evolved through trial and error, and he has experienced that trial and error and has accumulated wisdom in looking at and interpreting the data.”

Leverage candidates with regulatory advantages

Dave Mazzo, CEO of Lisata Therapeutics, a clinical-stage company focused on developing treatments for certain types of advanced solid tumors, including pancreatic cancer and glioblastoma.

“First, there’s a fairly simple analysis: we look for signs and tumours where stroma is present and the current prognosis is dire. These are the toughest cancers, and some would say all drugs fail. But there is a scientific rationale for pursuing these targets, and also regulatory and commercial strategies, especially in the early stages of clinical research.

We aim for indications that qualify for orphan drug or pediatric drug designation, because that can potentially expedite regulatory review. It also makes us eligible for grant funding from foundations in those fields. It’s also easier to do trials in high unmet need designations, where patients are asking for something new, than in, say, breast cancer, where there are a lot of options and a lot of good stuff. For pancreatic cancer, the treatments have been around for decades, without even a slight improvement in outcomes. Think about it like a patient.

Ultimately, the goal is to expand clinical studies into larger non-rare indications.”

Focus on the mission

Andy Seid, partner at Bioluminescence Ventures, said: Debuted last year It is focused on accelerating drug development and supporting “mission-driven” and platform-based companies.

“The first question I ask to determine if I’m in the right partnership agreement relies on understanding the fundamental situation of the company. It starts with solidifying your core strategy: what asset are you developing and why is it important? Companies need to start with a problem and then build a solution from there. Its strategy needs to be tied to its mission.”

As a venture capital investor, I try to answer this question: What will address an unmet patient need and provide independent value creation for the company? You need a clear, well-structured strategy that the CEO is not distracted from. And the company needs to be focused first. If pharma comes later, that’s great, but don’t aim to partner with pharma from the get-go. Let them come to you.”

Stick to the basics

Robert Jacks, CEO and president of Sparrow Pharmaceuticals, a clinical-stage biotechnology company with a lead candidate aimed at treating endocrine disorders caused by excess cortisol.

“I used to work in business development at Pfizer, and the questions we would hear often were: ‘Do you own this? Can you manufacture it? Will someone pay for it? In other words, do you have the intellectual property rights? Can you manufacture it easily? Will doctors prescribe it?'”

I work for a company that hopes to collaborate with big pharma in the future, so it’s good to know what they think.”