Neuren Media and Analyst Coverage, page-1539

Looking at “comparative” deals is always interesting and while it can be useful, it can also be misleading. Doubtlessly, some will look at this list of acquisitions for biotechs with rare disease drugs at Phase 3 stage and conclude that Neuren is at risk of being taken out during Phase 3 for less than A$2 bn. Thus, I think that further examination of all the lower-priced acquisitions is warranted.


At first glance, the price Biogen paid to acquire Nightstar Therapeutics in 2019 (US$800 m) might seem surprisingly low. After all, as stated, they were acquiring a company with a Phase 3 asset, in a disease with an estimated US$1 to 5 bn market and “79,000 to 158,000 individuals globally.”

But Nightstar had just two clinical assets, with the second candidate only in Phase 1/2. Both drugs were gene therapies; thus able to be developed in one indication only.

Despite the seemingly high patient population, the company estimated a patient population of just 13,000 across both the US and the EU.

Phase 2 data in the lead asset raised questions as the drug had showed a dose inverse response. Unfortunately for Biogen, this translated to a failed Phase 3 trial.

Nightstar had no approved products and hence no commercial revenue.

Nightstar had also licensed in its assets and thus milestones and royalties were payable if the assets were successfully developed and commercialised.


Ipsen’s acquisition of Clementia Pharmaceuticals for US$1.3 bn in 2019 might also seem to have been a bargain for Ipsen. After all, at the time of acquisition, Clementia was aiming to file for FDA approval of its lead asset within months.

However, Clementia’s lead drug had only passed Phase 2 (with somewhat shaky results) and the FDA subsequently denied accelerated approval. Ipsen had to complete a Phase 3 trial first, leading to a 2-3 year delay. The drug received approval from the FDA but was denied EU approval.

The potential patient population for fibrodysplasia ossificans progressive was very small, with a US population of just 400 (900 globally).

Another drawback was that the major patent for Clementia’s lead drug (acquired from Roche) was due to expire within 2 years.

Like Nightstar, Clementia was a clinical-stage company with no approved products or commercial revenue.


Novo Nordisk acquisition of Forma therapeutics in 2022 for US$1.1 bn appears to be another bargain deal. Forma’s lead asset, for the treatment of sickle cell disease, was being tested in a Phase 2 and Phase 2/3 trial at the time of acquisition. The Phase 2/3 trial in 450 patients commenced in 2021 and was expected to take 6 years. Another large Phase 3 trial, estimated to take 5 years, has just recently commenced.

Though the lead indication, sickle cell disease, has a relatively large patient population (est. 100,000 in the US), there are already 5 or 6 drugs approved for the condition, including 2 gene therapies. The most common treatment costs