Roche adds a stel­lar round of piv­otal da­ta on Hem­li­bra, look­ing to ex­pand its mar­ket for a po­ten­tial mega-block­buster

Roche just rolled out a slate of new da­ta that once again show why Hem­li­bra has roiled the fu­ture of one of Shire’s big ther­a­peu­tic fran­chis­es.

In­ves­ti­ga­tors have col­lect­ed new late-stage da­ta on the drug demon­strat­ing a dra­mat­ic re­duc­tion in bleed­ing for a swath of he­mo­phil­ia pa­tients, amp­ing up an­a­lysts’ ex­pec­ta­tions for the drug to be­come the next stan­dard of care in he­mo­phil­ia.

The da­ta came from two sep­a­rate Phase III tri­als that are in­tend­ed to eval­u­ate the drug in non-in­hibitor pa­tients — those who haven’t de­vel­oped re­sis­tance to fac­tor re­place­ment ther­a­py — and on a month­ly dos­ing sched­ule, re­spec­tive­ly. In HAVEN 3, when com­pared to pa­tients who re­ceived no pro­phy­lax­is, those who got Hem­li­bra every week or every two weeks showed a 96% and 97% re­duc­tion in treat­ed bleeds (p<0.0001 for both fig­ures). And through an in­tra-pa­tient com­par­i­son among a sub­set of pa­tients who pre­vi­ous­ly re­ceived fac­tor VI­II pro­phy­lax­is, Roche not­ed a 68% re­duc­tion ((p<0.0001) in treat­ment bleeds.

John­ny Mahlangu

An ap­proval in this in­di­ca­tion would mark a ma­jor mar­ket op­por­tu­ni­ty for Hem­li­bra, which first got ap­proved for pa­tients with in­hibitors. But Roche has not been shy about its am­bi­tions to go af­ter Shire’s prized fac­tor re­place­ment fran­chise, and an­a­lysts have pegged peak sales es­ti­mates of the block­buster-to-be at $5 bil­lion.

Take­da, though, hasn’t hes­i­tat­ed in of­fer­ing a big pre­mi­um to com­plete its planned buy­out of Shire $SH­PG.

“Even with cur­rent pro­phy­lac­tic treat­ments, many peo­ple with he­mo­phil­ia A con­tin­ue to have bleeds that can lead to long-term joint dam­age, and there is a need for more treat­ment op­tions,” said John­ny Mahlangu of the Uni­ver­si­ty of the Wit­wa­ter­srand and NHLS, Jo­han­nes­burg, South Africa.

In a lengthy state­ment, Roche al­so tout­ed ze­ro treat­ed bleeds for 55.6% of pa­tients who re­ceived week­ly in­jec­tions and 60% of those treat­ed every two weeks. In the group treat­ed with no pro­phy­lax­is, the rate was 0%.

San­dra Horn­ing

Haven 4 of­fered a look at Hem­li­bra’s ef­fi­ca­cy as a month­ly treat­ment, re­gard­less of pa­tient sta­tus vis-a-vis fac­tor VI­II in­hibitors. In the sin­gle-arm study — which en­rolled 48 pa­tients aged 12 or old­er and gave them Hem­li­bra every four weeks — 56.1% ex­pe­ri­enced ze­ro treat­ed bleeds and 90.2% had three or few­er.

“These new piv­otal da­ta show that Hem­li­bra con­trolled bleeds in peo­ple with he­mo­phil­ia A, while of­fer­ing the flex­i­bil­i­ty of less fre­quent sub­cu­ta­neous dos­ing op­tions,” said San­dra Horn­ing, CMO and head of glob­al prod­uct de­vel­op­ment. “With this da­ta, we now have pos­i­tive re­sults from all four of our Phase III tri­als that re­in­force the over­all ef­fi­ca­cy and safe­ty of Hem­li­bra and its po­ten­tial to im­prove care for all peo­ple with he­mo­phil­ia A.”

Key ques­tions about safe­ty linger, which could af­fect pa­tients and physi­cians al­ready on a dif­fer­ent treat­ment re­gard­ing whether to switch to Hem­li­bra. Yet, re­cent re­ports about deaths among pa­tients tak­ing Hem­li­bra (which Roche says were all un­re­lat­ed to the drug) have not stopped the FDA from hand­ing it break­through ther­a­py sta­tus as a treat­ment for pa­tients with­out fac­tor VI­II in­hibitors, which trans­lates to speedy re­view by the reg­u­la­tors.

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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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.

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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.

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.

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.

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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