Sofinno­va leads $32.4 mil­lion round for Italy's Ery­Del; Cel­gene ex­pands col­lab­o­ra­tion with Zymeworks

→ Hav­ing fol­lowed Mi­lan-based Ery­Del and its CEO for years, Sofinno­va Part­ners is jump­ing in for the ride, lead­ing its lat­est €26.5 mil­lion ($32.4 mil­lion) round. The funds will cov­er the reg­is­tra­tional Phase III clin­i­cal tri­al of Ery­Del’s lead prod­uct, Ery­Dex, which us­es the biotech’s red-blood-cell-based drug de­liv­ery tech­nol­o­gy to treat atax­ia telang­iec­ta­sia. The tri­al, dubbed AT­TeST, is cur­rent­ly en­rolling in Eu­rope, Asia, Aus­tralia and the US. Sofinno­va is joined by ex­ist­ing share­hold­er Genex­tra SpA and Inno­gest SGR, all chip­ping in to push the com­pa­ny along in its in­ter­na­tion­al de­vel­op­ment. “We are thrilled to wel­come Sofinno­va Part­ners as our lead in­vestor,” CEO Lu­ca Be­nat­ti said in a state­ment. “Their unique in­ter­na­tion­al reach and rec­og­nized ex­pe­ri­ence for build­ing glob­al lead­ers rep­re­sent a huge as­set at a time when Ery­Del is about to ac­cel­er­ate its growth plan.”

→ More than three years in­to its part­ner­ship with Zymeworks $ZYME, Cel­gene $CELG is ex­er­cis­ing an op­tion to ex­pand its use of the Azy­met­ric plat­form. The big biotech now has rights to de­vel­op and com­mer­cial­ize 10 bis­pe­cif­ic an­ti­body can­di­dates com­ing out of that plat­form, up from eight. For Van­cou­ver-based Zymeworks, that means an un­spec­i­fied ex­pan­sion as well as an ex­tra po­ten­tial $328 mil­lion in mile­stones ($164 mil­lion for each prod­uct, as es­tab­lished in orig­i­nal agree­ment). The re­search pro­gram term has al­so been ex­tend­ed by two years.

→ Al­ny­lam’s lead drug patisir­an just got one step clos­er to ap­proval with pos­i­tive da­ta re­port­ed out this morn­ing from a Phase III tri­al. In a new post-hoc analy­sis, patisir­an re­duced com­pos­ite rate of all-cause hos­pi­tal­iza­tion and mor­tal­i­ty by about 50% rel­a­tive to place­bo. The drug al­so demon­strat­ed an im­prove­ment in mul­ti­ple qual­i­ty of life mea­sure­ments when com­pared to place­bo. The Cam­bridge/Boston RNAi spe­cial­ist has ex­pressed much con­fi­dence in patisir­an for hered­i­tary AT­TR amy­loi­do­sis, hav­ing re­struc­tured its deal with Sanofi to re-gain glob­al rights just ahead of the JP Mor­gan health care con­fer­ence in Jan­u­ary. An ap­proval — by the PDU­FA date of Au­gust 11 — is wide­ly ex­pect­ed. “We be­lieve these re­sults, along with pre­vi­ous­ly pre­sent­ed APOL­LO da­ta that show halt­ing or re­ver­sal of neu­ropa­thy pro­gres­sion in a ma­jor­i­ty of pa­tients treat­ed with patisir­an, strength­en the body of ev­i­dence demon­strat­ing that patisir­an, if ap­proved, has the po­ten­tial to be a trans­for­ma­tive treat­ment for pa­tients with all forms of hered­i­tary AT­TR amy­loi­do­sis,” said Er­ic Green, VP and gen­er­al man­ag­er of the TTR pro­gram at Al­ny­lam in a state­ment. “We con­tin­ue to work col­lab­o­ra­tive­ly with the FDA and EMA through patisir­an’s re­view process, with the goal of mak­ing this med­i­cine avail­able to pa­tients as quick­ly as pos­si­ble, up­on ap­proval.”

UK-based Or­chard Ther­a­peu­tics is open­ing up a Boston of­fice in the Sea­port Dis­trict, fast on the heels of its ac­qui­si­tion of GSK’s rare dis­ease unit. The new US of­fice for the gene ther­a­py com­pa­ny will be for “com­mer­cial op­er­a­tions and sev­er­al key fi­nan­cial and ad­min­is­tra­tive func­tions.”

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.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

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.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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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.

Mike Grey. Mirum

In $86M IPO pitch, Mirum spells out plans to turn Shire dis­cards in­to or­phan liv­er drug suc­cess­es

Mike Grey doesn’t have any time to waste. Hav­ing re­gained con­trol of two liv­er dis­ease drugs from Shire and po­si­tioned them for piv­otal stud­ies — five years af­ter first hand­ing them off in a deal to sell Lu­me­na, where he was CEO — Grey is steer­ing Mirum straight in­to an IPO with a $86 mil­lion ask.

Not that Mirum has spent much of its $120 mil­lion Se­ries A cash since launch­ing last No­vem­ber. Ac­cord­ing to the S-1, the Cal­i­forn­ian biotech has burned through $23.3 mil­lion as of March, but ex­pects ex­pens­es to pick up once their clin­i­cal work gath­ers steam.