Rare Disease Day and BIO CEO recaps

Happy March. We recap yesterday’s Rare Disease Day hearing—and the bipartisan support for a few key BIO priorities—and look at a few things you might have missed at the BIO CEO and Investor Conference. (552 words, 2 minutes, 45 seconds)

Lawmakers back bipartisan legislation supporting rare disease research

At yesterday’s House Energy and Commerce Health Subcommittee hearing on Rare Disease Day, lawmakers considered three of BIO’s legislative priorities that would support rare disease R&D.

What they discussed:

• The ORPHAN Cures Act (H.R. 5539) would fix a flaw in the Inflation Reduction Act (IRA) to ensure orphan drugs are exempt from price controls, even if approved for two or more indications.
• The Creating Hope Reauthorization Act (H.R 7384) would renew the Rare Pediatric Disease Priority Review Voucher (PRV) program, which supports treatments for rare diseases that disproportionately impact children.
• The Maintaining Investments in New Innovation (MINI) Act (H.R. 5547) would increase protection from price controls for drugs made with genetically targeted technology to 13 years instead of 9. 

What they said:

• Subcommittee Chair Brett Guthrie (R-KY): “Research and development into therapies designed to treat rare diseases is challenging,” which makes investment “risky and unpredictable.”
• Rep. Nanette Barragan (D-CA): “Rare Disease patients experience inequities in diagnosis and care,” and the problem is greater for people of color.
• Rep. John Joyce, M.D. (R-PA): The ORPHAN Cures and MINI acts “represent narrow and concise fixes to the IRA” to promote innovation and bring patients new treatments.
• Rep. Michael Burgess, M.D. (R-TX): Uncertainty around Pediatric Disease Priority Review Vouchers “adds another layer of complexity to this space,” making investment into research harder to obtain.

The bottom line: These small policy fixes, especially tweaks to the IRA “are important steps and will make a meaningful difference in the lives of the millions of Americans living with rare diseases,” said Rep. Guthrie. 

Read more on Bio.News.

Watch highlights:

A few more things from the BIO CEO & Investor Forum in New York…

On investment: Companies must differentiate themselves, demonstrate rapid growth potential, and provide clear communication to secure funding. Investors prioritize proven clinical data and a solid path to commercialization, said panelists on Monday.

What they’re saying: “Financing right now is hard,” said Kate Walsh, Managing Director, Life Science and Healthcare at HSBC Bank USA. “Really know in this market to try and take on more capital because you know that things are not going to go as planned. There are going to be bumps along the way, and then also, ideally having real data inflection points metered out along the way to help de-risk your technology, help the investors get comfortable, and find more opportunities to finance the company.”

On M&A: In a challenging market, M&A is crucial—especially for early-stage biotechs, said panelists Monday. Strategic M&A, coupled with a focus on core value offerings and therapeutic areas of high growth potential, can position firms for success in a competitive landscape.

On obesity drugs: The metabolic field, particularly advancements in GLP-1s, is a significant opportunity for innovation and investment, agreed panelists Tuesday. Despite challenges, such as concerns about tolerability issues and weight loss durability, ongoing R&D offers hope for patients and the investment landscape.

What they’re saying: “It’s about time that obesity is considered a medical condition and not just something that one thinks about in cosmetic terms,” said Jon Isaacsohn, founder & CEO of CinFina, which is working to develop long-acting GLP-1s.

Watch: BIO’s VP for External Affairs, Michele Oshman, talks about the important role of mission-driven investors:

President Biden’s Friday: Meeting with Prime Minister Giorgia Meloni of Italy at the White House, before heading to Camp David for the weekend.

What’s Happening on Capitol Hill:Partial shutdown avoided—enjoy the weekend.