Am­gen com­mits to low-cost Repatha; AZTher­a­pies bags Smith Ther­a­peu­tic­s' CAR-Treg tech for neu­roin­flam­ma­tion

→ As Am­gen tracks mod­est growth of its Repatha fran­chise and braces for the ar­rival of cheap­er cho­les­terol-low­er­ing med­i­cines in the mar­ket, its com­mer­cial team is go­ing one step fur­ther to try and boost adop­tion of its PC­SK9 drug.

Repatha’s list price will now be $5,850 across the board, not co-ex­ist­ing with the high­er cost op­tion. First in­tro­duced in Oc­to­ber 2018, that num­ber rep­re­sent­ed a 60% dis­count to the orig­i­nal $14,100 per year.

“We have seen sig­nif­i­cant im­prove­ments in ac­cess and af­ford­abil­i­ty, but not all Medicare pa­tients are ben­e­fit­ting from these im­prove­ments be­cause some Medicare Part D plans have not tran­si­tioned to the low­er list price op­tion of Repatha,” said Mur­do Gor­don, EVP of glob­al com­mer­cial op­er­a­tions, in a state­ment. “We are dis­con­tin­u­ing the orig­i­nal list price op­tion so that pay­ers and Medicare Part D health plans have clar­i­ty and can do their part: cov­er the low­er list price op­tion of Repatha to help every pa­tient pre­scribed Repatha fill their pre­scrip­tion at an af­ford­able, low fixed dol­lar co-pay.”

That rhetoric, fo­cused on costs to pa­tients and mid­dle­men’s roles, has been a key talk­ing point among bio­phar­ma com­pa­nies chas­tised for high drug prices.

David El­maleh AZTher­a­pies

While Repatha has per­formed bet­ter than Pralu­ent, the ri­val PC­SK9 ther­a­py from Re­gen­eron and Sanofi, sales are still far off from the block­buster fig­ures an­a­lysts had ex­pect­ed. And not on­ly has Am­gen lost a re­cent court fight to push that com­peti­tor out, it’s al­so fac­ing a new threat from The Med­i­cines Com­pa­ny’s in­clisir­an — an RNAi ther­a­peu­tic tout­ed as a su­pe­ri­or al­ter­na­tive to the an­ti­bod­ies.

Har­vard pro­fes­sor David El­maleh is adding CAR-Treg to his ar­se­nal of neu­roin­flam­ma­tion treat­ments at AZTher­a­pies through an ac­qui­si­tion of Smith Ther­a­peu­tics. The lit­tle biotech — al­so based in Boston — was found­ed by Philip Ash­ton-Rickardt, a for­mer Im­pe­r­i­al Col­lege Lon­don aca­d­e­m­ic who’s join­ing AZTher­a­pies as SVP of im­munol­o­gy. The pre­clin­i­cal CAR-Treg pro­grams he’s bring­ing to the com­pa­ny re­volves around im­muno­sup­pres­sive cells de­signed to damp­en mi­croglial ac­tiv­i­ty, there­by re­duc­ing in­flam­ma­tion.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Samantha Du, Zai Lab CEO

Any­one still look­ing for a CD47? Zai Lab shelves PhI pro­gram af­ter re­view­ing 'com­pet­i­tive land­scape'

Over the past few years, the promise of blocking CD47 — a “don’t eat me” signal co-opted by cancer cells — has sent drugmakers big and small into a frenzy. But one biotech is now bowing out.

Zai Lab is deprioritizing ZL-1201, its CD47 inhibitor, scrapping plans for a Phase II trial. It will now “pursue out-licensing opportunities,” the company said in its Q2 update. The decision was based on a review of the competitive landscape, it added, without going into further details.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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BioCryst re­sumes en­roll­ment for im­mune dis­eases drug; Ea­gle buys stake in res­pi­ra­to­ry dis­ease biotech

In April, the FDA hit BioCryst with a partial hold, leading the biotech to pause enrollment on three of its rare disease clinical trials and sending its shares down 30%.

Last week, BioCryst said the partial clinical hold had been resolved and that it would resume enrolling its clinical trials for its drug, dubbed BCX9930, but with new protocols at a lower 400 mg dose.

BioCryst’s drug is a factor D inhibitor, which was being tested in an array of immune diseases. The biotech is running two pivotal trials for paroxysmal nocturnal hemoglobinuria, as well as a third trial looking at a set of three diseases — C3 glomerulopathy, IgA nephropathy and primary membranous nephropathy.