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.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.