Is bio­phar­ma’s first $1.5M ther­a­py now in Phase III?

Key­ing off of Spark Ther­a­peu­tics’ roll­out of Lux­tur­na as the first ap­proved gene ther­a­py in the US, the an­a­lysts at Leerink have been crunch­ing the num­bers on what the first he­mo­phil­ia gene ther­a­pies could fetch. And by rais­ing their es­ti­mate to $1.5 mil­lion, they may well be tempt­ing fate at a time the de­bate over drug pric­ing is reach­ing the boil­ing point in the US.

Spark sat­is­fied most an­a­lysts with its $425,000 price per eye for Lux­tur­na. And Leerink says that the pos­i­tive re­sponse by Har­vard Pil­grim to a 30-month dura­bil­i­ty start­ing point for a gene ther­a­py pric­ing agree­ment set the stage for many of the rest of the field to fol­low suit. And that will jus­ti­fy a $1.5 mil­lion-plus price for a ther­a­py that could re­place cur­rent stan­dards in he­mo­phil­ia with a sim­i­lar track record on dura­bil­i­ty.

Af­ter check­ing with some KOLs in the field, Leerink of­fered:

As a re­sult of this work, we are rais­ing our as­sumed price for he­mo­phil­ia gene ther­a­pies in our cov­er­age uni­verse to $1.5M per pa­tient in the US ($1M in the EU), an in­crease from pri­or as­sump­tions of $350,000-$500,000 (he­mo­phil­ia A) and $500,000 (he­mo­phil­ia B) per pa­tient. Our checks in­di­cate that there may be some head­room above this price, po­ten­tial­ly up to $2M per pa­tient on a gross ba­sis, but giv­en the un­cer­tain­ty about re­spon­sive­ness, re­bates and oth­er fac­tors, we be­lieve $1.5M is a jus­ti­fi­able es­ti­mate for net price per pa­tient at this junc­ture….

We be­lieve the man­u­fac­tur­ers are test­ing the wa­ters cur­rent­ly as they em­bark on Phase 3 tri­als in or­der to de­ter­mine where pay­ors push back sig­nif­i­cant­ly. Based on our checks, a price some­where in the range of $1-2M per pa­tient seems most like­ly.

The big rea­son for that 7-fig­ure price tag is based on pay­ers’ will­ing­ness to cov­er the $580,000 to $800,000 price per pa­tient for fac­tor re­place­ment ther­a­py, giv­ing them some up­side even with a $1 mil­lion-plus stick­er for a once-and-done gene ther­a­py. And that could have a pro­found ef­fect on Spark $ONCE, Bio­Marin $BM­RN and uniQure $QURE, which are all en­gaged in de­vel­op­ment ef­forts.

As long as the av­er­age pa­tient can re­ly on fac­tor re­place­ment ther­a­py, Leerink adds, the low hang­ing fruit for the de­vel­op­ers ear­ly on will be among the most se­vere pa­tients, and stick­ing with that small pa­tient pop­u­la­tion al­so helps jus­ti­fy the big num­ber.

At this stage of the game, the biotechs lead­ing this field will like­ly hedge their bets, steer­ing clear of of­fer­ing any num­bers they may have in mind as they test the wa­ters on the fi­nal price tag. But Leerink just gave them a big op­por­tu­ni­ty to con­tin­ue their pri­vate pay­er talks with some big num­bers in mind.

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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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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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.

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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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.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

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.