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.”

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three ma­jor buy­outs an­nounced: Take­da/Shire; Bris­tol-My­ers/Cel­gene and now Ab­b­Vie/Al­ler­gan. And with this lat­est deal it’s in­creas­ing­ly clear that the sharp fall from grace suf­fered by high-pro­file play­ers which have seen their share prices blast­ed has cre­at­ed an open­ing for the growth play­ers in big phar­ma to up their game — in sharp con­trast to the pop­u­lar bolt-on deals that have been dri­ving the growth strat­e­gy at No­var­tis, Mer­ck, Roche and oth­ers.

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Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

The top 15 mega-deals in bio­phar­ma: Ab­b­Vie and Bris­tol-My­ers ac­qui­si­tions stir fresh de­bate over what's too big to buy

The debate over what’s too big to buy in biotech is back. A number of top analysts went right after AbbVie’s rationale for the Allergan deal today, just as Bristol-Myers Squibb stirred immediate debate over the worth and wisdom of acquiring Celgene.

To help provide some added context to this discussion, we asked DealForma chief Chris Dokomajilar to look over the past decade of major M&A in biopharma to decipher the top 15 plays.

The new numbers, unadjusted for inflation, harken back to the days of the Pfizer-Wyeth buyout and Merck’s decision to absorb Schering-Plough — both triggered in 2009. The heat over those acquisitions made the big pharma mega-deal highly unpopular for most everyone — except Pfizer — as industry leaders swore off almost all but the handy bolt-on acquisition.

Until recently.

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Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.