BioCryst wins ap­proval for HAE pill and charges half-a-mil­lion dol­lars, but will any­one take it?

Six years af­ter the FDA OK’d their flu an­tivi­ral peramivir, BioCryst has notched an­oth­er ap­proval.

The North Car­oli­na-based biotech an­nounced Thurs­day that the FDA had ap­proved bero­tral­stat, a dai­ly pill meant to pre­vent painful episodes in pa­tients with the rare dis­ease hered­i­tary an­giode­ma (HAE). The mol­e­cule, which Biocryst has been study­ing in the clin­ic since 2013, will be sold un­der the name Or­ladeyo.

De­spite in­fe­ri­or ef­fi­ca­cy, BioCryst will charge $485,000 for Or­ladeyo, rough­ly in line with oth­er treat­ments on the mar­ket. The price tag sur­prised RBC’s Bri­an Abra­hams, who had pro­ject­ed a $300,000 tag.

The ques­tion is how many pa­tients will ac­tu­al­ly buy it. The mol­e­cule met its pri­ma­ry end­point in a 96-pa­tient Phase III tri­al last year, suc­cess­ful­ly re­duc­ing the num­ber of painful in­ci­dences by 44% com­pared with place­bo. But the stock fell pre­cip­i­tous­ly af­ter BioCryst an­nounced the re­sults, as in­vestors com­pared that 44% with the re­duc­tions shown by oth­er drugs on the mar­ket.

BioCryst sought to carve out a niche as one of the on­ly oral op­tions for pa­tients, an al­ter­na­tive to the in­jecta­bles that have dom­i­nat­ed the mar­ket. But the drug looked like it might be in­fe­ri­or enough that few pa­tients would switch and risk an in­crease in HAE at­tacks, which can be fa­tal.

Take­da’s Takhzy­ro, for ex­am­ple, had shown an 87% re­duc­tion in painful events, or rough­ly dou­ble.

Abra­hams laid out the sit­u­a­tion plain­ly in a note late Thurs­day night. He pre­dict­ed around 12% of mild HAE, 9% in mod­er­ate, and 7% of se­vere pa­tients would take the drug, lead­ing to sales around $170 mil­lion per year. Af­ter BioCryst an­nounced the price, he said sales may be as high as $280 mil­lion.

“This marks an im­por­tant mile­stone for BCRX, though it is not sur­pris­ing and was most­ly ex­pect­ed giv­en the straight­for­ward, sta­tis­ti­cal­ly sig­nif­i­cant da­ta, lack of an Ad­Comm, and high con­fi­dence pub­licly ex­pressed by man­age­ment as re­cent­ly as yes­ter­day,” he said. “The re­al test will be com­mer­cial up­take and what pro­por­tion of HAE pa­tients will be drawn by Or­ladeyo’s dif­fer­en­ti­at­ed oral ad­min­is­tra­tion rather than the cur­rent­ly avail­able every-2week SC that has twice the la­beled ef­fi­ca­cy.”

BioCryst $BCRX shares im­me­di­ate­ly spiked post-mar­ket Thurs­day but have since slid to $4.77, or 35 cents be­low yes­ter­day’s $5.12 close.

With BioCryst’s pill un­der­whelm­ing, oth­er com­pa­nies, such as the Swiss start­up Phar­varis, are try­ing to come up with their own oral op­tions. BioCryst, mean­while, is try­ing to see how ef­fec­tive­ly their pill can not on­ly pre­vent at­tacks but stop them when they oc­cur.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.