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.”

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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