Do they just do PH3 in PMS?
And make everyone wait 3 years? And then sell. I cant see it. (I'd do it commercially, but not medically). And I just have this deep feeling that the NEU board want to have a purpose first and foremost. And to deliver to patients before shareholders. (even though they are currently doing both)
There is a further factor for Neuren (and Acadia and any other potential acquirer of Neuren) to consider when deciding if and when to proceed with Phase 3 development in the various indications for NNZ-2591.
That factor is the Inflation Reduction Act (IRA), something
discussed here in August last year. Under the current legislation, introduced by the Biden Administration, if a small molecule drug has received orphan drug designation in a second indication (and NNZ-2591 has orphan drug designation in all four indications currently being trialled by Neuren), then, 7 years following the date of FDA marketing approval in the first indication, that drug becomes eligible for mandatory price negotiation, with price control coming into play 2 years later.
It can be argued that the impact on sales of the first indication would be limited – nine years after approval most drugs would be facing generic competition anyway. However, sales of any further indications are likely to be impacted. The median time from novel orphan drug approval to the next follow-on indication is ~ 4 years. The drug in that second indication would potentially face price control after just 5 years. The potential impact is even greater for any further indications. Hence, the current law heavily disincentivizes drug companies from developing drugs in multiple rare diseases.
As explained by a representative from the National Organization of Rare Disorders (NORD), “The problem now is that the way the IRA is written, once these orphan drugs apply for a second designation, even though there is no revenue associated with it, they will become negotiable. We think that it has a tremendous chilling effect on the pipeline, in particular for the small biotech where we get a lot of really big innovation in the orphan drug space.”
There is some hope is on the horizon.
Lawmakers introduced the
Optimizing Research Progress Hope and New Cures (ORPHAN Cures Act) to the US House of Reps last September and the Senate in October. The sponsors of the Bill are proposing that reference to exemption from the IRA for drugs approved in “only one rare disease or condition” be struck out and replaced with the words “one or more rare diseases or conditions”. However, as we know, the wheels of government turn slowly at the best of times, this is a US election year, and there can be no certainty that the Bill will pass both Houses anyway.
In the meantime, the current legislation applies and will likely inform decision-making by Neuren, Acadia and any potential acquirer.
NORD Position on IRA/CMS Drug Negotiation Price Program (rarediseases.org)The Inflation Reduction Act: Unintended Consequences - Bio.NewsBILLS-118hr5539ih.pdf (govinfo.gov)ORPHAN Cures Act would boost rare disease R&D - Bio.News
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