Kiniksa touts 3rd ap­proval for ag­ing Ar­c­a­lyst — a good haul for just $5M in cash it paid Re­gen­eron for the drug

Kiniksa bet $5 mil­lion cash back in 2017 that Re­gen­eron’s Ar­c­a­lyst could help pa­tients with re­cur­rent peri­cardi­tis, a painful au­toin­flam­ma­to­ry heart dis­ease. Now, more than three years lat­er, that bet con­tin­ues to pay off.

Reg­u­la­tors ap­proved Ar­c­a­lyst (rilona­cept) to treat re­cur­rent peri­cardi­tis and re­duce the risk of re­cur­rence in pa­tients 12 years and old­er, Kiniksa said on Thurs­day. The Bermu­da-based biotech is plan­ning a com­mer­cial launch — its very first — this April, and will split the prof­its 50/50 with Re­gen­eron.

“We’re clear­ly very ex­cit­ed to have the ap­proval of our first com­mer­cial prod­uct,” gen­er­al man­ag­er Ross Moat said in a call with in­vestors on Thurs­day. “Not on­ly does Ar­c­a­lyst be­come the first and on­ly ap­proved drug in re­cur­rent peri­cardi­tis, but it has the po­ten­tial to be­come the stan­dard of care.”

The FDA’s de­ci­sion was based on re­sults from the Phase III RHAP­SODY tri­al, in which pa­tients giv­en Ar­c­a­lyst achieved a 96% re­duc­tion in the risk of a re­cur­rent peri­cardi­tis event, with a p-val­ue of 0.0001, ac­cord­ing to Kiniksa. Me­di­an time to treat­ment re­sponse was 5 days, with a 97% treat­ment re­sponse rate. For pa­tients in the Ar­c­a­lyst arm, 92% of tri­al days were “pain free or at most ex­pe­ri­enc­ing min­i­mal pain,” com­pared to 40% of days for those on the place­bo.

John Paoli­ni

“Me­di­an time to first oc­cur­rence in the Ar­c­a­lyst arm could not be cal­cu­lat­ed as there were not enough re­cur­rent events dur­ing the ob­ser­va­tion pe­ri­od to al­low for the num­ber to be cal­cu­lat­ed,” CMO John Paoli­ni said dur­ing the in­vestor call.

The most com­mon ad­verse events were in­jec­tion site re­ac­tions and up­per res­pi­ra­to­ry tract in­fec­tions.

On Fri­day morn­ing, $KN­SA shares hov­ered around $20.83 apiece.

Re­cur­rent peri­cardi­tis typ­i­cal­ly presents with chest pain and is of­ten linked to changes in elec­tri­cal con­duc­tion and some­times peri­car­dial ef­fu­sion, or the buildup of flu­id around the heart, ac­cord­ing to Kiniksa. The re­cur­rent dis­ease af­fects about 40,000 pa­tients in the US each year, 14,000 of whom ex­pe­ri­ence a sec­ond re­cur­rence.

“Our fo­cus is on the 14,000 pa­tients on their sec­ond re­cur­rence, who have the high­est un­met need,” Moat said. “These pa­tients have failed on cur­rent sys­temic non-tar­get­ed treat­ments and re­quire a so­lu­tion that di­rect­ly ad­dress­es in­ter­leukin-1 al­pha and be­ta, the un­der­ly­ing dri­vers of peri­car­dial in­flam­ma­tion.”

Ar­c­a­lyst, which tar­gets IL-1α and IL-1β, was first ap­proved in 2008 for cry­opy­rin-as­so­ci­at­ed pe­ri­od­ic syn­dromes (CAPS), in­clud­ing fa­mil­ial cold au­toin­flam­ma­to­ry syn­drome and Muck­le-Wells syn­drome. It got an­oth­er OK in De­cem­ber 2020 for the main­te­nance of re­mis­sion of de­fi­cien­cy of IL-1 re­cep­tor an­tag­o­nist (DI­RA). Kiniksa con­trols full sales and dis­tri­b­u­tion of the drug for all three in­di­ca­tions in the US.

“Whilst our pro­mo­tion­al ef­forts will be fo­cused on re­cur­rent peri­cardi­tis, we re­main ful­ly com­mit­ted to serv­ing pa­tients who are and will be treat­ed with Ar­c­a­lyst across all of the in­di­ca­tions,” Moat said.

Moat says the com­pa­ny has hired a car­di­ol­o­gy spe­cial­ist sales team of 27 peo­ple, who will be “ready to roll over the com­ing days.” Ar­c­a­lyst costs $20,000 per month, a price Moat said is “con­sis­tent with oth­er spe­cial­ty bi­o­log­ics.”

“We aim to se­cure broad ac­cess with min­i­mal pay­er re­stric­tions for our tar­get pop­u­la­tion,” he added.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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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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.

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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David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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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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Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”