PTC shares rock­et up as Eu­ro­pean reg­u­la­tors of­fer a thumbs-up on Duchenne drug mar­ket­ing

PTC Ther­a­peu­tics CEO Stu­art Peltz

A few days af­ter warn­ing in­vestors of the ris­ing un­cer­tain­ty it faced in the on­go­ing Eu­ro­pean re­view of its con­di­tion­al ap­proval for Translar­na, shares of PTC Ther­a­peu­tics $PTCT sky­rock­et­ed this morn­ing af­ter reg­u­la­tors gave it a thumbs up on keep­ing the con­tro­ver­sial Duchenne drug on the mar­ket.

There is a con­di­tion at­tached to the rec­om­men­da­tion from the Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use. The CHMP wants PTC to con­duct a place­bo-con­trolled, 18-month study of their drug fol­lowed by an 18-month ex­ten­sion study, with da­ta ex­pect­ed in ear­ly 2021. That would give them a clear mar­ket­ing run­way of close to 5 more years, if the EMA for­mer­ly ac­cepts the rec­om­men­da­tion.

PTC’s shares, beat­en down by the skep­ti­cism is faced, shot up more than 100% on the news.

PTC skep­tics abound. Its drug has re­peat­ed­ly failed clin­i­cal tri­als and a num­ber of an­a­lysts have spec­u­lat­ed that EU reg­u­la­tors would jerk the drug, which was giv­en a con­di­tion­al ap­proval back in 2014, ahead of a failed Phase III study. The FDA re­cent­ly re­fused to even con­sid­er its ap­pli­ca­tion, say­ing PTC had too weak a case to mer­it a thor­ough re­view.

PTC is ap­peal­ing that de­ci­sion, look­ing to get its drug and its pa­tient ad­vo­cates in front of the same reg­u­la­tors that gave Sarep­ta an ap­proval for Ex­ondys 51, even though in­sid­ers at the agency waged a hard fought cam­paign in­sist­ing that the drug had nev­er come close to meet­ing the FDA’s stan­dards for an ap­proval.

But Duchenne mus­cu­lar dy­s­tro­phy is a field un­to it­self, where the hard log­ic and high reg­u­la­to­ry bar that of­ten trig­gers drug re­jec­tions have been blunt­ed by pa­tient ad­vo­cates des­per­ate to have some drug avail­able that might slow a dis­ease that grad­u­al­ly kills its young vic­tims. That de­sire to have some­thing, any­thing, avail­able has been a boon to PTC and Sarep­ta, even as crit­ics shake their heads at the idea of ex­per­i­men­tal drugs with lit­tle ev­i­dence of ef­fi­ca­cy be­ing sold to pa­tients for six-fig­ure prices.

Bar­clays fol­lowed up with a dis­cus­sion with PTC ex­ecs and came away with this:

In speak­ing with man­age­ment, it is clear that there was sig­nif­i­cant dis­cus­sion and en­gage­ment with EMA over the past year. CHMP de­ter­mined that the drug does slow pro­gres­sion and has a fa­vor­able risk ben­e­fit pro­file. The com­pa­ny has not yet dis­closed oth­er as­pects of the tri­al, name­ly end­points and in­clu­sion/ ex­clu­sion cri­te­ria, but not­ed that the tri­al will be sim­i­lar in size to ACT DMD (230 en­roll­ment). Man­age­ment em­pha­sized that this up­date on­ly per­tains to EMA, as FDA dis­cus­sions are still on­go­ing. While the com­pa­ny would like to align tri­al ef­forts be­tween the EU and US, they not­ed that they hope that Ata­lau­ren would be avail­able in the US well be­fore the 2021 read out of this new con­fir­ma­to­ry tri­al. Giv­en the high lev­el of un­cer­tain­ty around nmD­MD in the US we con­tin­ue to as­sign no val­ue in our NPV analy­sis.

“We are pleased with this out­come which took in­to ac­count all avail­able da­ta for Translar­na,” said Stu­art W. Peltz, PhD, Chief Ex­ec­u­tive Of­fi­cer, PTC Ther­a­peu­tics, Inc. “This de­ci­sion re­flects the ben­e­fit that Translar­na is hav­ing for pa­tients suf­fer­ing from non­sense mu­ta­tion Duchenne mus­cu­lar dy­s­tro­phy.”

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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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.

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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