The Whens and Whys of Top Biotech M&A Deals, page-5

This one seems to have flown under the radar with a total value up to USD $1.1b for a phase 3 ready drug to treat a rare disease with a prevalence of 1 in 24000. In comparison, PMS is 1/8000 to 1/15000 whereas PHS is 1/34000 to 1/41000.

Post successful FDA meeting in Sep, Neuren could be phase 3 ready for PMS and MOST LIKELY phase 3 ready soon after for Pitt Hopkins too given they both fall under the same FDA division.

On 1st Aug, Otsuka Commits USD $800M in M&A Deal Bringing a Phase 3 Ready Metabolic Disorder Drug. The Japanese pharma company could shell out up to $325 million more if the lead drug candidate of privately held Jnana achieves development and regulatory milestones.

Interestingly, there are already two treatments available for this rare disease with $484.7m combined sales in 2023. NNZ2561 on the other hand, has the potential to be the first in the market to treat both PMS and Pitt Hopkins.

Two approved PKU therapies are also available, both from BioMarin Pharmaceutical: Kuvan is a small molecule that helps the process of breaking down phenylalanine while Palynziq is an engineered version of the enzyme needed to break down the amino acid. These therapies don’t work for all PKU patients and some who take Palynziq struggle to maintain the required regimen of daily injections. For 2023, BioMarin reported $484.7 million in combined revenue for the two products.

The drug has the potential to treat the spectrum of PKU patients but they have only so far conducted a Phase 1b trial for a different indication. In contrast, Neuren has 3 successful phase 2 trials so far and more to be announced soon

Jnana has said its drug has potential to treat the spectrum of PKU patients from mild to severe disease. The acquisition agreement comes six months after the biotech announced JNT-517 demonstrated clinical proof of concept in a Phase 1b trial, paving the way for a pivotal study planned for the second half of 2025. In addition to being safe and well tolerated, the interim results from 13 patients — eight dosed with the study drug and five with placebo — showed a statistically significant 51% average reduction in phenylalanine levels in the blood. Results also showed a rapid onset of effect that was sustained throughout the 28 days of dosing.