Stag­gered by a Duchenne pa­tient’s death, Akashi fi­nal­ly gets a green light to get back in­to the clin­ic

Marc Blaustein, CEO

Akashi Ther­a­peu­tics was put in lim­bo more than a year ago af­ter the FDA forced the lit­tle biotech to slam the brakes on its de­vel­op­ment ef­fort for a new drug to treat Duchenne mus­cu­lar dy­s­tro­phy fol­low­ing the death of a pa­tient in an ear­ly-stage study. But 13 months lat­er Akashi says it now has a green light from reg­u­la­tors to get back in­to the clin­ic with HT-100.

Akashi is slash­ing the dose to 150 µg/day af­ter test­ing a range that ex­tend­ed from 300 µg/day to 1500 µg/day. And at the time Akashi said that the pa­tient who died was on a high dose of the drug HT-100. The biotech is al­so end­ing the use of antiemet­ic ther­a­pies.

HT-100 (de­layed-re­lease halofug­i­none) is a small mol­e­cule de­signed to tamp down on in­flam­ma­tion and spur mus­cle growth, an ap­proach that CEO Marc Blaustein has said could work as part of a fu­ture cock­tail of reme­dies for DMD pa­tients. The drug is a syn­thet­ic ver­sion of an in­gre­di­ent found in plants which in­ves­ti­ga­tors say blocks pro-in­flam­ma­to­ry Th17 cells.

Blaustein told Xcon­o­my that the pa­tient’s blood pres­sure fell dan­ger­ous­ly low af­ter — not un­nat­u­ral­ly — build­ing up lev­els of halofug­i­none. The symp­toms weren’t caught though be­cause he was tak­ing an an­ti-nau­sea med­i­cine that masks symp­toms, which is why they’re re­quir­ing pa­tients to stop us­ing those ther­a­pies.

With some cru­cial sup­port from non­prof­its like Charley’s Fund, the Mus­cu­lar Dy­s­tro­phy As­so­ci­a­tion and the Nash Av­ery Foun­da­tion, lit­tle Akashi was able to take its ther­a­py in­to a Phase Ib/IIa study while strik­ing a $100 mil­lion col­lab­o­ra­tion with Ger­many’s Grü­nen­thal Group.

Now Akashi says it plans to get back in the clin­ic as soon as pos­si­ble while pur­su­ing talks with in­vestors and pos­si­ble part­ners. The tim­ing on that re­mains up in the air.

The Duchenne MD field is dom­i­nat­ed by seem­ing­ly un­end­ing con­tro­ver­sy. The lat­est de­vel­op­ment to roil R&D came with the FDA’s con­tro­ver­sial ap­proval of an old gener­ic steroid — de­flaza­cort — as an or­phan ther­a­py for Duchenne. But af­ter run­ning in­to a buzz saw of con­tro­ver­sy when it priced the ther­a­py at $89,000 a year, Marathon sold it to PTC Ther­a­peu­tics for $140 mil­lion up front.

PTC, mean­while, has been grap­pling with the FDA’s re­fusal to re­view ataluren, which has now failed three straight tri­als, but re­mains on the mar­ket in Eu­rope.

Sarep­ta won an ap­proval for its drug eteplirsen, but now has a la­bel that states there’s no clear ev­i­dence that it works.

Hope springs eter­nal, though, and Akashi plans to push ahead. The com­pa­ny is al­so work­ing on DT-200 (se­lec­tive an­dro­gen re­cep­tor mod­u­la­tor) and AT-300 (cation chan­nel mod­u­la­tor).

“Our goal con­tin­ues to be im­prov­ing the lives of pa­tients with DMD and oth­er mus­cle func­tion dis­eases,” sais Blaustein in a pre­pared state­ment. “We are pleased that the FDA has agreed with our con­clu­sion that it is ap­pro­pri­ate to re­sume de­vel­op­ment of HT-100 and look for­ward to mov­ing ahead with the tri­al as quick­ly as pos­si­ble.”

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.

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.

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.

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.

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.

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.

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.