BIO Notebook: Woodcock Calls For Doing The Right Thing, Dealmaking Remains Constrained

Woodcock: Don’t Do The Wrong Study, Even If FDA Tells You To

Janet Woodcock said she has told drug developers not to blindly follow agency advice, an unusual comment from a former US Food and Drug Administration acting commissioner and long-time Center for Drug Evaluation and Research director.

“What I’ve been advising people is never do the wrong study,” Woodcock said at a June 18 BIO International Convention session on optimizing clinical trial design and execution for rare disease.

Even if the FDA told the sponsor to do a particular study or a randomized controlled study, if it’s not going to work, “don’t do it,” Woodcock said.

“If it’s going to be underpowered and you’re likely to fail, do not do that study,” she added. If it is the wrong endpoint, “do not do that study.”

Instead, “do the right thing,” she said.

Companies should focus on conducting studies they believe can generate solid data, regardless of the FDA’s hesitancy.

“In many of these interventions, because the disease is so dire, the treatment effect is quite large,” Woodcock argued. “You will carry the day with your data.”

For very small, heterogenous diseases, Woodcock advises sequential n of 1 studies, which involve following an individual, conducting a run-in phase and picking that person’s serious symptoms to track.

“Prospectively designate and write it down, what a clinically significant effect would be,” she said. “And then do that again and again” for other patients.

If a sponsor does this once and a person who was bedridden walks, “nobody’s going to be that impressed,” Woodcock said. “But if you do it seven times and five out of seven” walk, “you’re going to carry the day.”

Woodcock argued her advice is consistent with the FDA’s drug approval authority.

“The statute governing efficacy says adequate and well controlled investigations that will convince experts,” she said. “It does not say anything about a p value. It does not say anything about randomization. It says adequate and well controlled and there are different ways to do that.”

“So sure, meet with the FDA. Totally agree with that,” Woodcock said. But her message was do not just blindly follow any advice.

She has heard from multiple groups that followed FDA advice and the study failed.

“And that’s just a tragedy, because often with a rare disease, it can’t happen again,” Woodcock said.

Woodcock, in partnership with the Haystack Project, has been helping craft legislation that would establish a third path for rare disease drugs to meet the FDA’s “substantial evidence” standard without a randomized controlled trial.

RCTs are “fit for purpose for large populations,” Woodcock said, but not fit for rare diseases.

The smaller the size of the study arms, the more likely bias toward the null hypothesis occurs, which means an increased chance of not finding a treatment effect even if a product is effective, Woodcock said.

“And that’s very sad, because it’s some of our most needy populations who don’t have any treatments, and yet, the designs that we currently use make it much less likely that we’ll find an effect when there is one,” she said. “It’s a math issue, it’s not ideological, it’s mathematical.”

PwC: Regulatory, Policy Uncertainty Constrain Dealmaking

Life sciences deal volume and total valuation has been sluggish approaching the midpoint of 2025, even compared to the below-average numbers posted during the first quarter, PwC said in its “US Deals 2025 midyear look” report, issued June 18 in tandem with the BIO meeting.

Author Roel van den Akker, PwC’s US pharma and life sciences deals leader, said heightened regulatory uncertainty and several US policy issues are constraining dealmaking activity, despite an environment where scientific progress, numerous potential near-term drug approvals and healthy biopharma company balance sheets should be producing a healthy stream of transactions.

“Overall, we continue to believe that the near-term prospects for M&A in the sector remain robust,” van den Akker said in the report. “But dealmakers face a multifaceted regulatory and geopolitical set of challenges, which require pharmaceutical and life science companies to demonstrate significant agility, comprehensive due diligence and strategic foresight in managing their dealmaking activities and overall corporate strategies.”

In addition to a tight capital market yielding very few initial public offerings for life sciences firms, the report cited the uncertainty created by the Trump administration’s “most favored nation” executive order on prescription drug prices, potential impacts of program and workforce reductions at the US Food and Drug Administration and other agencies, and policy decisions related to Health and Human Services Secretary Robert F. Kennedy Jr.’s “Make America Healthy Again” initiative as leading to “further complicated revenue forecasting and valuation modeling.”

Compounding those factors is the evolving US tariff policy landscape, “marked by legal disputes, international negotiations and economic implications, leading to market volatility and global economic risk.”

The sector remains focused primarily on bespoke bolt-on acquisition deals meant to strengthen product pipelines, van den Akker said.

People, Relationships Mattered In Novartis’s Anthos Buyout

Novartis took the BIO stage June 17 to discuss its partnerships in a panel discussion that included Ronny Gal, the company’s chief strategy and growth officer, alongside two investors and executives from two smaller firms that the big pharma has worked with, including Anthos CEO Bill Meury.

Novartis announced in February that it would pay $925m up front to buy Anthos for its Factor XIa inhibitor abelacimab, a Novartis drug previously out-licensed to the company.

“Early on in the conversation, I think we agreed on the topline price very, very quickly,” Gal said. “And then it was all about the downside.”

He said that the companies had to determine how Novartis would protect itself from the risk of ongoing Phase III abelacimab clinical trials not succeeding. The result was the acquisition included $925m up front and up to $2.15bn in additional payouts based on future milestone events.

Gal said that because he and Meury have known each other for many years, it was easy to find solutions to any dealmaking challenges.

“The one problem we had was their owner was Blackstone, and Blackstone is also a large company,” Gal said.

The multibillion-dollar investment firm had corporate rules it had to follow, as did Novartis, but any challenges eventually were resolved, he added.

“Ronny and I have known each other for a long time,” Meury said. “I think that was a big benefit. We trusted each other … I was transparent from the moment we started talking. All the threshold issues were put on the table. I wanted him to get as smart as I was about the company.”

Meury said Novartis did probably more due diligence on Anthos as a company than it did on the Phase II results that Anthos previously generated for abelacimab.

“I would not underestimate the importance of the quality of the business for the buyer,” he said. “When you’re running a business and if this is a path that you can end up going down, it’s important that most of these companies aren’t buying an asset. They’re buying a company. That means people and structure and process. And whether you can draw a line to value or not, doesn’t matter. I do think it can become a threshold issue for a potential acquirer.”

Generate Preparing First Phase III Program, Assessing Funding Options

Privately held Generate:Biomedicines plans to take its lead drug candidate, the anti-TSLP antibody GB-0895, into Phase III testing for asthma in October and is considering several funding options to fully interrogate the asset, but CEO Mike Nally told Scrip at BIO that an initial public offering is not on the table at this time.

Nally, like investors and advisors on a June 16 BIO panel, noted that the IPO window is essentially closed in the US for biopharmaceutical companies right now.

Generate, which combines artificial intelligence and wet lab research to develop various types of biologics, last raised venture capital in 2023 via a $273m series C round. The company also has signed deals with big pharma partners to fund its platform and drug development programs, which include an IL-13-targeting candidate for atopic dermatitis that Generate has in Phase I healthy volunteer testing. The company earned $50m up front under a collaboration signed with Amgen in 2022 and $65m up front to partner with Novartis in 2024.

“We have $330m in cash on our balance sheet today,” Nally said. “The combined cost of the [TSLP] program, if you were to look at all indications, all patient populations, right now we think it’s about a half a billion dollars. And so inevitably we will need to seek additional financing to support not just the program, but also what’s critical to us is that we don’t underinvest in the technology, because we think this is just a starting point.”

Nally said Generate is investigating whether it should pursue another private financing or an asset-specific financing, as well as an eventual partner for GB-0895, because the company does not have the capabilities at this time to commercialize the drug.

The product would go head-to-head against Amgen’s anti-TSLP antibody Tezspire (tezepelumab), which is approved for asthma and generated positive Phase III results in a second indication, chronic rhinosinusitis with nasal polyps, in November 2024.

Nally said Generate can start its Phase III trials for GB-0895 with its cash on hand and determine the best path forward for funding the full Phase III program across all indications, while still investing in its AI-based drug discovery and development platform plus additional clinical-stage biologics, including planned programs in oncology.

[Editor’s note: The BIO notebook is a joint effort by the Pink Sheet and Scrip.]