Following are a number of statements, made by various posters on this forum over the last few days, to which I’d like to reply.
QRX, whose large part of the company was being held by Walker suffered a demise like no other when they got rejected by FDA a few years ago… One would think that a investor who made his fortune on BIO's to become a billionaire would have enough advise on whether the stuff was going to work but walker did not know and the company failed.
Lang Walker’s fortune comes from his property empire, not from investing in biotechs. Investing in biotechs is a side interest for him and he is candid in saying that when he tells this to people, they look at him as if he is on drugs. But irrespective of Walker’s interest, his money or the quality of his advisers, no one can predict Phase 3 results or FDA decisions with certainty. If they could, deep-pocketed pharmas would never have Phase 3 trial failures or FDA knockbacks. But they do. Frequently.
Pharma have had the most recent results since March and the other trial data for years yet they haven't acted in any significant manner to date.
I assume that “act in a significant manner” means sign off on a deal. Neuren management flagged that partnering discussions wouldn't occur in earnest until after meeting with the FDA to sort out the Phase 3 Rett trial design. This decision on timing seems logical to me because the agreed upon trial design would have direct bearing on any deal value calculations made by both Neuren and any prospective partner.
Maybe the Instos & Pharma aren't interested because NEU have been all over the place chasing multiple targets instead of focusing their limited funds on one indication
That’s one view.
Here’s another from global management consulting firm, L.E.K., on the topic of “Strategies for Orphan Drug Makers”
Given the small patient populations for orphan diseases, it makes sense to develop drugs for an array of different diseases, instead of approaching the business as a one-off venture. Smaller companies might be tempted to direct all their resources toward a single product or disease. But to become a long-term presence in the orphan market, they need a strategic vision that carries them beyond their first product. In short, for large and small companies alike, the focus should be on sustained value creation.
….(pharma) are directing their interests into the pipeline of the hot in demand neurodegenerative disease indications such as Alzheimers & Parkinson's etc. (which have the most partnering activity).
I won’t dispute that every pharma would love to develop a successful treatment for Alzheimer’s
but …. at what cost? This time last year Eli Lilly announced failure of its Phase 3 trial in Alzheimer’s. It was a 3.5 year long trial in 2121 patients. I don’t know the trial’s full cost, but it would run into the hundreds of millions of dollars. Because it was such a big trial, in all senses of the word, announcement of its failure wiped US$10 billion off Eli Lilly’s market cap. In February this year Merck gave up on its pivotal Phase 2/3 trial in Alzheimer’s. That 2210 patient trial had commenced over 4 years earlier.
Personally, I don’t think pharma can afford to invest all of its interest in large, costly, blockbuster indications such as Alzheimer’s. And the evidence suggests that it’s not.
The Director for the FDA Office of Orphan Products Development
said last year
Developing drugs for rare diseases, once considered a rare phenomenon itself, has fast become a mainstay for many companies’ drug development pipelines.
Biopharmadive, on trends in orphan drug development, says
Many of the top rare disease drug manufacturers are not small, narrowly focused biotechs. Instead, most are the big pharma and big biotechs that dominate the industry elsewhere.
Currently, 40% of NME drug approvals are for orphan disease indications. Orphan diseases, including neurodevelopmental orphan indications such as Rett syndrome, are “hot in demand” by pharma in their own right.
Bell Potter has also been predicting a deal is imminent for years…..
It’s true that Bell Potter has been assuming an ultimate licence deal for a number of years now, but I don’t recall it being claimed at any stage that it was “imminent”. The assumption in the BP October update was for a Q1 2018 deal. Not quite the definition of “imminent” held by some here.
And if they were about to do a deal and negotiating with multiple parties, how could it possibly hurt to tell the market that….
Well, we do know the company has been talking with multiple interested parties because it has stated so (although I acknowledge that some won’t believe anything that management says). As to the question of “How could it possibly hurt to tell the market” if the company was “about to do a deal”? Mmm..
It is noteworthy that LW chose not to increase his holdings
This is known as a fact? What is known is that Neuren opted to run with Lanstead. But where has it been stated that this occurred because Lang Walker “chose not to increase his holdings”?
Big pharma funds are scarce and our future health care depends on them being directed efficiently.
Poor pharma, they are almost penniless….US pharmas in the S & P 500 alone have
over US$460 bn cash stockpiled overseas.
....to get a substantial from a pharma before the trial results is a mean task… they will engage NEU , discuss some partenrship options to some extent but will not sign the paper until p3 is concluded. Honestly, I feel that the deal would not happen until end of p3 with a positive outcome.
Yes, pharma would be taking on a level of risk if signing a deal prior to successful results in Phase 3 being announced. But pharma actually prices and runs with a level of risk all the time. To the extent that,
in 2016, 87% of in-licence deals signed by pharma were for assets at the “risky” Phase 2 clinical stage or earlier. Just 2% were signed at the post- Phase 3, ready-to-file stage.
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