In the hunt for a megablock­buster, Cel­gene plans FDA fil­ing in wake of PhI­II win for ozan­i­mod in MS

When Cel­gene bought out Re­cep­tos and its au­toim­mune drug ozan­i­mod back in 2015 for $7.2 bil­lion in cash, the big biotech bull­ish­ly fore­cast their drug would be worth $4 bil­lion to $6 bil­lion in peak sales. To­day, the ex­ec­u­tive crew start­ed to make good on that pro­jec­tion with a pos­i­tive read­out on the top-line da­ta from their Phase III mul­ti­ple scle­ro­sis study.

The com­pa­ny says it’s on track to file for an ap­proval of the S1P1 re­cep­tor an­tag­o­nist this year.

In­ves­ti­ga­tors weren’t hand­ing out any hard da­ta to­day. But the com­pa­ny says that ozan­i­mod de­liv­ered “sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­ing­ful im­prove­ments com­pared to Avonex for the pri­ma­ry end­point of ARR (the an­nu­al­ized re­lapse rate) and the mea­sured sec­ondary end­points of the num­ber of gadolin­i­um-en­hanc­ing MRI le­sions and the num­ber of new or en­larg­ing T2 MRI le­sions at month 12.”

A sec­ond Phase III study in MS will read out in Q2.

This is a tough mar­ket, though. To get any­where close to $6 bil­lion, ef­fi­ca­cy alone won’t cut it. Cel­gene will al­so have to prove a sub­stan­tial safe­ty ad­van­tage with their drug.

Ge­of­frey Porges, Leerink

Just a few days ago Ge­of­frey Porges at Leerink out­lined the strat­e­gy for ozan­i­mod.

Re­sults from the two Ozan­i­mod phase III tri­als in mul­ti­ple scle­ro­sis are due in the first half of this year, with phase III ul­cer­a­tive col­i­tis da­ta ex­pect­ed in the first half of 2018. CELG al­ways as­sumed ozan­i­mod would get same class la­bel­ing match­ing oth­er S1P1 in­hibitors such as Gilenya, but al­so an­tic­i­pate that the phase III safe­ty da­ta would be in the la­bel so that CELG could dis­cuss the po­ten­tial dif­fer­ences with physi­cians and pay­ers. CELG stat­ed the com­pa­ny needs to see “re­al­ly good ef­fi­ca­cy” and a sim­i­lar lack of car­dio and liv­er tox­i­c­i­ty as shown in the phase II tri­al. About 15% of pa­tients dis­con­tin­ue Gilenya due to these tox­i­c­i­ty is­sues. CELG stat­ed that pric­ing will be low­er in UC when the drug is launched in that in­di­ca­tion, and the com­pa­ny plans to use dis­counts by in­di­ca­tion or oth­er meth­ods to match the ex­ist­ing pric­ing in the MS and IBD in­di­ca­tions (at a list price sim­i­lar to the ex­ist­ing $90,000 Gilenya price).

The com­pa­ny said to­day that the safe­ty pro­file of the drug is in line with their Phase II re­sults. And they sound­ed pumped about the prospects of their in­flam­ma­tion and im­munol­o­gy pipeline.

Scott Smith, Cel­gene

Jef­feries’ Bri­an Abra­hams gave the com­pa­ny a quick thumbs up, not­ing that there’s a long way to go be­fore this drug lives up to ex­pec­ta­tions.

We cur­rent­ly mod­el out-year MS sales of ozan­i­mod of ~$1B, giv­en the po­ten­tial Gilenya gener­i­ciza­tion, in­creas­ing com­pe­ti­tion, and grow­ing pric­ing pres­sure in the MS space. While it is not clear whether CELG would de­tail the agent on their own, the com­pa­ny has re­cent­ly shown more open­ness to us­ing ozan­i­mod as a cor­ner­stone to build out a broad­er neu­rol­o­gy com­mer­cial/de­vel­op­ment plat­form. In IBD, we as­sume peak sales ap­proach­ing ~$3.5B in the out-years, and while key ph.III UC da­ta is not ex­pect­ed un­til 2018, we could see some ini­tial Crohn’s dis­ease da­ta that could help clar­i­fy its pro­file and ex­pand­ed IBD po­ten­tial.

“Peo­ple liv­ing with mul­ti­ple scle­ro­sis need ad­di­tion­al ther­a­pies and we are pleased that oral ozan­i­mod showed mean­ing­ful im­prove­ments across pri­ma­ry and mea­sured sec­ondary end­points in this study,” said Scott Smith, pres­i­dent of Cel­gene In­flam­ma­tion and Im­munol­o­gy. “We look for­ward to da­ta from the con­fir­ma­to­ry phase III RA­DI­ANCE tri­al in the sec­ond quar­ter as we ad­vance to­ward planned reg­u­la­to­ry sub­mis­sions by year-end.”

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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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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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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US to stop sup­ply­ing Lil­ly's mAb for Covid-19 this month as com­mer­cial mar­ket awaits

Federal officials said yesterday that shipments of Eli Lilly’s bebtelovimab — one of the final two remaining mAb treatments for Covid-19 — would halt later this month, setting up a commercial market where the government no longer pays for the doses and hospitals and other clinics will have to purchase supplies.

According to ASPR, the arm of HHS that ships Covid-19 drugs, states have ordered 627,536 bebtelovimab courses, and 383,515 courses have been administered as of July 31. The US has paid Lilly a total of about $1.27 billion for all of the courses so far, amounting to about $2,100 per course to start and then receiving a discounted $1,833 ASP for the later part of the deal. According to the Wall Street Journal, Lilly’s list price for bebtelovimab is $2,100 per dose.

Stanley Erck, Novavax CEO (Photographer: Kevin Dietsch/UPI/Bloomberg via Getty Images)

No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room: