With key test still loom­ing, Cel­gene and Ac­celeron nab FDA okay for be­ta-tha­lassemia med

While pric­ing con­cerns con­tin­ue to cloud the fu­ture of blue­bird bio’s gene ther­a­py fix, the FDA has giv­en its okay for Cel­gene and Ac­celeron’s be­ta-tha­lassemia drug Re­blozyl — to treat ane­mia in adults who re­quire reg­u­lar trans­fu­sions. An ap­proval for myelodys­plas­tic syn­dromes (MDS) — which will like­ly de­ter­mine if the drug is a ma­jor com­mer­cial suc­cess — is still un­der re­view.

The drug will be priced at $3,441 per 25 mg vial, Ac­celeron said.

A fu­sion pro­tein, Re­blozyl (lus­pa­ter­cept-aamt) boosts red blood cell growth in ane­mic pa­tients by tar­get­ting TGF-be­ta pro­teins that play a role in these cells’ mat­u­ra­tion. The drug, which had been grant­ed break­through sta­tus, was ap­proved on the ba­sis of a 336-per­son Phase III tri­al.  The tri­al showed slight­ly over a fifth of the be­ta-tha­lassemia pa­tients who took the drug saw a 33% or greater re­duc­tion in trans­fu­sion bur­den.

Richard Paz­dur FDA

“When pa­tients re­ceive mul­ti­ple blood trans­fu­sions, there is a risk for iron over­load, which can af­fect many or­gans,” said FDA on­col­o­gy chief Richard Paz­dur. “To­day’s ap­proval pro­vides pa­tients with a ther­a­py that, for the first time, will help de­crease the num­ber of blood trans­fu­sions.”

Cel­gene ac­quired the drug in 2011 for $25 mil­lion up­front and a mod­est $217 mil­lion in mile­stones. It’s a key part of the late-stage pipeline Bris­tol-My­ers Squibb bet on in their $74 bil­lion buy­out; an­a­lysts have pegged the drug as a po­ten­tial block­buster worth up to $2 bil­lion per year.

Two thirds of that pro­ject­ed rev­enue, though, come from MDS pa­tients.

For the be­ta-tha­lassemia pa­tients, a dif­fer­ent so­lu­tion emerged in the time since Cel­gene’s deal as blue­bird bio de­vel­oped a gene ther­a­py that could of­fer a com­plete rem­e­dy for the dis­ease. But that ther­a­py, Zyn­te­glo, has yet to be ap­proved in the US (al­though an FDA okay could come as soon as next year), and in Eu­rope, where the EMA has giv­en its bless­ing, the promise of a one-shot fix for a life­long dis­ease has been com­pro­mised by a $1.8 mil­lion price tag — twice what an­a­lysts had pro­ject­ed for the drug on the con­ti­nent.

The FDA is due to rule on the MDS in­di­ca­tion by April 4, 2020. Cel­gene ap­plied on the ba­sis of late-stage da­ta that showed 38% of pa­tients went 8 weeks with­out need­ing a blood trans­fu­sion and 28% man­aged 12 weeks with­out one.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected 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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,100+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.