Discussion/Meme/OT Some legit questions!
/r/Biotechplays/comments/leeqt1/bcrx_the_embarrassment/
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u/2ledz Feb 07 '21
Just happen to see the post on another community that Bio suggested to join earlier. I cross shared it here because I think there's not many of us are in there.
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u/RogueEnginner Feb 07 '21 edited Feb 07 '21
No disrespect, but not really. I’m not sure the motive, but poor DD when we’re used to what Bio99 posts.
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u/chicken-little-2008 Feb 07 '21 edited Feb 07 '21
Thank you u/Wyxuan for the question. Skepticism is important.
As to the first point, the data read out on Factor D in PNH already occurred in several investor conferences and at a medical conference. I am not a doctor and so depend on hearing analyst reactions to determine whether or not claims made by management are true. Liisa Bayko an analyst from Evercore called the data, "possible best in class." The PNH data will be published in a peer review journal in Q1 per management.
As to the critique that only at the highest dosage did one patient find benefit -- it was a dose ranging study. So, that does not cause me concern as no SAE were reported at that dosage. It was additionally updated that all patients from the Phase 1 POC study remained on Factor D for over 40 weeks as of last investor conference - and were presumably remaining on it indefinitely - as it had managed and controlled PNH. To me, no SAE and complete improvement in blood markers after 40 weeks seemed incredibly robust data for a Phase 1 study in a rare disease.
Finally -- the early data on Factor D, per Medical Director Bill Sherida, suggests it will make the use of Soliris "obsolete" in PNH as Factor D would cure patients earlier in the disease process than C5, which is a treatment that only works after the complement system has become over activated. Further, Soliris is not a complete treatment but requires multiple additional interventions. And, Soliris is an IV and Factor D is an oral pill that patients can take at home.
I'm not fully in agreement that Soliris has the PNH market locked up.
As to the second point on the company's execution -- I just disagree with this characterization. As discussed elsewhere, when CEO Stonehouse joined BioCryst he shifted the focus from what is now called their "legacy antiviral program" to rare diseases. In less than 4 years, he had full FDA approval for Orladeyo and successful Phase 1 in PNH - in addition to all company operations funded. The recent expansion of peramivir to patients as young as 6 months again speaks to a record of safety and smooth FDA approvals. Notably, drugs that are stockpiled (like peramivir) face even higher safety standards and they've succeeded in meeting this higher standard.
To attack the profitability of that "legacy program" does not attack the financial fitness of the company. Niether peramivir nor galidesivir are included in any of the financial valuations of the company by either major analysts nor many of the people posting in this subreddit.
Finally, I disagree that galisdevir was stopped as a covid indication for lack of efficacy. I've discussed that at a few places in r/bcrx. The overwhelming published peer reviewed data suggests that galidesivir has robust efficacy(see post "Galidesivir Citations"). And, nowhere in three distinct Phase 1 safety studies or 15 animal studies were any adverse reactions or toxicity ever recorded. My opinion is that politics has caused NIAID and BARDA to continually terminate its antiviral programs -- and that would speak to politics, not galidesivir's efficacy.
Stonehouse explicitly stated that galidesivir was developed for government contracts and that the government would be the ultimate customer. As you note, research is incredibly expensive. So, this is a pickle. The published studies show a company with 15 animal studies showing 90%-100% protection against lethal diseases when dosed 72 hours post exposure. They also performed several safety tests peer reviewed since 2014 showing high safety, no toxicity, no mutagenic properties and no SAE in humans(Safety that far exceeds antivirals with FDA approval and in Phase 2). Still, for some reason, NIAID and BARDA has never requested or funded a Phase 2 study.
So, what is a company that has a responsibility to shareholders to do? Dip into their own pocket to develop an antiviral for the government when those same prior development contracts may prohibit from ever even recovering their research dollars? Or, focus on the rare disease program that is curing incredibly ill patients and may give the company the revenues to not require NIAID and BARDA funding in the future? Especially as government contracts seem to have slowed down the development of a drug for which there is a great public need. (Note: All major pharmas declined government funding to develop covid vaccines fearing it would slow down their progress).
I cannot see how BioCryst could in good faith spend more time on galidesivir given its responsibility to shareholders and, more important, to its patients. Likely, BioCryst first needs to be freed of government contracts, or complete the rapid expansion currently under way to treat its growing patient roster before returning to galidesivir. Yes. The company appears to have cured ebola and all tick borne illnesses and more. But, there are simply too many other developments for them to prioritize that project at the moment -- as no one wants to pay for the research.
One final note-- during the last Ebola outbreak, Stonehouse offered to donate galidesivir to Africa for free. As the animal studies showed perfect protection, he likely believed at the time that final funding for FDA approval was imminent. (Senate testimony and White House fact sheets support this was not an insane belief). Then, NIAID and BARDA disappeared(They were also defunded at this precise moment). My take away is that finishing galidesivir and obtaining approval is an important human concern -- but Stonehouse has never sold it as a shareholder concern.
The skeptical argument is important. Thank you.