BioCryst halts en­roll­ment in all stud­ies for its lead pipeline can­di­date — shares crash

BioCryst is tak­ing a beat­ing Fri­day morn­ing af­ter re­port­ing it would pause en­roll­ment in three stud­ies for its lead pipeline pro­gram.

In a terse state­ment put out right be­fore the mar­ket opened, the Durham, NC-based biotech said it dis­cov­ered el­e­vat­ed serum cre­a­ti­nine lev­els in some pa­tients, and will halt re­cruit­ment while it in­ves­ti­gates. El­e­vat­ed cre­a­tine lev­els can be a sign of kid­ney dys­func­tion.

The pa­tients were tak­ing the ex­per­i­men­tal drug known as BCX9930, an oral Fac­tor D in­hibitor be­ing eval­u­at­ed in three clin­i­cal stud­ies.

Af­ter Fri­day’s open­ing bell, BioCryst $BCRX shares fell more than 30%,  to $11.92.

Two of the biotech’s clin­i­cal stud­ies were ex­am­in­ing the ex­per­i­men­tal drug in parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH), one in an open-la­bel set­ting com­pared to an ac­tive com­para­tor and one con­trolled against place­bo. Both were be­ing eval­u­at­ed as a monother­a­py. BioCryst’s plan was to study BCX9930 in a range of com­ple­ment-me­di­at­ed dis­eases.

Ac­cord­ing to the stud­ies’ clin­i­cal­tri­ web­pages, BioCryst had ex­pect­ed to en­roll about 140 pa­tients be­tween the two tri­als, and it’s not im­me­di­ate­ly clear how many had tak­en the drug as of Fri­day’s up­date. A com­pa­ny spokesper­son de­clined to com­ment, say­ing BioCryst is not pro­vid­ing de­tails on the stud­ies.

The pause marks a set­back for the biotech’s pipeline a lit­tle over a year af­ter it won its first FDA ap­proval for Or­ladeyo, a dai­ly pill meant to pre­vent painful episodes in pa­tients with the rare dis­ease hered­i­tary an­gioede­ma (HAE). BioCryst moved quick­ly to cap­i­tal­ize on the ap­proval, sign­ing a $250 mil­lion deal with Roy­al­ty Phar­ma three days af­ter the green­light.

Late last year, the biotech al­so moved to raise some new cash, get­ting an­oth­er $350 mil­lion from Roy­al­ty and new in­vestors. Some of the mon­ey was planned for ad­vanc­ing BCX9930 to its NDA and build­ing out ad­di­tion­al clin­i­cal tri­als, BioCryst said at the time.

This ar­ti­cle has been up­dat­ed to in­clude BioCryst’s re­sponse to an End­points News re­quest for com­ment.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

Harpreet Singh, Immatics CEO

Im­mat­ics an­nounces mul­ti­ple pipeline changes with lat­est fi­nan­cial re­sults

The T-cell biotech Immatics is looking to make some changes to its pipeline.

Immatics released its 2022 financial results on Tuesday and announced that it’s planning to discontinue its program for IMA201, an experimental cell therapy for solid tumors that express the antigens known as MAGE4/8. It plans to shift focus to IMA401, a TCR bispecific which goes after the same target.

The German-based biotech said it will treat the remaining patients enrolled in the program before the discontinuation. No other reasons were given for the discontinuation. Endpoints News reached out to Immatics for more details but did not receive a response by press time.

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FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Growth hor­mone from No­vo Nordisk is in short­age over man­u­fac­tur­ing de­lays

Novo Nordisk’s growth hormone Norditropin is in shortage because of manufacturing delays, according to an FDA site that tracks drug shortages as well as the American Society of Health-System Pharmacists’ shortages list.

The FDA has shortages of the drug listed for its 5, 10, 15 and 30 mg doses, while the pharmacists’ group, also known as ASHP, reported shortages of the same doses, except for the 15 mg version.

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