Am­i­cus Ther­a­peu­tics be­gan March with word that the FDA has sched­uled a pre-ap­proval in­spec­tion of its po­ten­tial treat­ment for Pompe dis­ease. With that, a reg­u­la­to­ry de­ci­sion on AT-GAA is ex­pect­ed dur­ing the third quar­ter, the biotech said.

Am­i­cus said last Oc­to­ber the FDA wasn’t able to do the re­quired in­spec­tion of its man­u­fac­tur­ing part­ner, WuXi Bi­o­log­ics in Chi­na, dur­ing the re­view time­frame be­cause of “re­stric­tions on trav­el re­lat­ed” to the pan­dem­ic. AT-GAA con­sists of bi­o­log­ic cipaglu­cosi­dase al­fa and miglu­s­tat, a sta­bi­liz­er of the bi­o­log­ic. The ap­proval re­quest in­cludes both a BLA and NDA.

AT-GAA has break­through ther­a­py des­ig­na­tion for late-on­set Pompe dis­ease. Reg­u­la­to­ry re­views are on track in the EU and UK, Am­i­cus said, with ap­proval de­ci­sions al­so ex­pect­ed in the third quar­ter. — Kyle LaHu­cik

2sev­en­ty looks to raise $125M in pub­lic of­fer­ing for R&D

2sev­en­ty bio has priced more shares to work on R&D along with its Bris­tol My­ers-part­nered ther­a­py.

The blue­bird bio on­col­o­gy spin­out said late Tues­day night that it will be of­fer­ing more than 10.8 mil­lion shares at $11.50 each, seek­ing to raise ap­prox­i­mate­ly $125 mil­lion. The com­pa­ny said that un­der­writ­ers would al­so get ac­cess to pur­chase an ad­di­tion­al 1.6 mil­lion shares as a 30-day op­tion.

2sev­en­ty said in its an­nounce­ment that it plans to close the of­fer­ing on or around March 3.

Shares of $TVST closed at $13.49 Tues­day af­ter­noon.

2sev­en­ty wrote in an SEC fil­ing that it plans to use the mon­ey raised for R&D to ad­vance prod­uct can­di­dates and ex­pand oth­er de­vel­op­ment pro­grams. Ad­di­tion­al­ly, the biotech said it would be in­vest­ing to ad­vance Kar­M­MA tri­als in a bid to test Abec­ma in ear­li­er lines of ther­a­py — along with in­vest­ing in­to Abec­ma’s com­mer­cial ac­tiv­i­ties as part of 2sev­en­ty’s cost-shar­ing agree­ment with Bris­tol My­ers Squibb.

The pair rolled out ear­li­er-line da­ta in Abec­ma last month, show­ing the tri­al met the pri­ma­ry end­point of pro­gres­sion-free sur­vival in pa­tients with sec­ond to fourth line mul­ti­ple myelo­ma. Abec­ma is cur­rent­ly on­ly in­di­cat­ed in the fifth-line and lat­er set­ting. — Paul Schloess­er

Tra­vere takes ap­proval to fundrais­ing route in bid for $200M

Af­ter the FDA green­lit Tra­vere Ther­a­peu­tics’ IgAN treat­ment sparsen­tan less than two weeks ago, the biotech is go­ing for cash to get things mov­ing.

The biotech said Tues­day night that it is of­fer­ing 8,275,000 shares at $21 a share — to raise a to­tal of $200 mil­lion in a pub­lic of­fer­ing. Tra­vere wrote in SEC doc­u­ments that the funds are planned to go to­wards sparsen­tan’s com­mer­cial launch, along with oth­er “gen­er­al cor­po­rate pur­pos­es” such as clin­i­cal tri­als and man­u­fac­tur­ing.

Brand­ed as Filspari, the can­di­date was ap­proved with a warn­ing of liv­er and fe­tal tox­i­c­i­ty.

The FDA is al­so re­quir­ing Tra­vere to run REMS, a risk eval­u­a­tion and mit­i­ga­tion strat­e­gy, in con­junc­tion with the ap­proval — re­quir­ing doc­tors to get cer­tain kinds of train­ing and hav­ing pa­tients en­tered in­to a mon­i­tor­ing pro­gram.

Shares of $TVTX closed at $22.16 Tues­day af­ter­noon. — Paul Schloess­er

Atea stops work on Dengue to ex­tend cash run­way

Atea said the costs and time as­so­ci­at­ed with run­ning Phase II stud­ies are too much for the biotech. It will fo­cus its mon­ey and time on Covid-19 and HCV, the Boston drug de­vel­op­er said Tues­day af­ter the mar­ket closed.

“We be­lieve AT-752 holds promise for the treat­ment of dengue as an oral di­rect act­ing an­tivi­ral. How­ev­er, it has be­come clear that im­proved di­ag­nos­tics are need­ed to bet­ter iden­ti­fy pa­tients ear­li­er in the course of the dis­ease,” CEO Jean-Pierre Som­ma­dos­si said in a state­ment. “In ad­di­tion, giv­en the high vari­abil­i­ty in both treat­ment and pro­phy­lax­is set­tings, sub­stan­tial­ly larg­er pa­tient sam­ple sizes would be re­quired for fu­ture Phase 2 stud­ies.”

The biotech has run in­to hur­dles be­fore with its Covid-19 pro­gram, lead­ing for­mer part­ner Roche to walk away from an an­tivi­ral. The com­pa­ny moved for­ward with­out the Big Phar­ma, work­ing on a Phase III tri­al test­ing be­m­ni­fos­bu­vir for treat­ment of Covid-19 in pa­tients at high-risk. An in­ter­im analy­sis is due in the sec­ond half of this year, Atea said.

Mean­while, a Phase II of be­m­ni­fos­bu­vir in com­bi­na­tion with ruzasvir will start en­roll­ment this quar­ter and have ini­tial da­ta in the last few months of 2023, Atea said, as it in­ves­ti­gates the pair as a treat­ment for HCV. — Kyle LaHu­cik

Ver­tex lines up ADC deal with Im­muno­gen 

An­ti­body-drug con­ju­gate biotech Im­muno­Gen is let­ting Ver­tex use its ADC tech to find new tar­get­ed con­di­tion­ing agents to be paired with gene edit­ing.

Af­ter a re­search pe­ri­od, Ver­tex can snag the world­wide, ex­clu­sive li­cense for R&D and po­ten­tial com­mer­cial­iza­tion of the con­di­tion­ing agents. Im­muno­Gen gets $15 mil­lion in ex­change to start. An­oth­er $337 mil­lion could come its way via op­tion fees, de­vel­op­ment mile­stones and com­mer­cial biobucks. Tiered roy­al­ties, too.

Ver­tex cell and gene ther­a­pies SVP Mike Cooke said the tech will be in­ves­ti­gat­ed with the bio­phar­ma’s exa-cel, which is be­ing test­ed for treat­ing sick­le cell and trans­fu­sion-de­pen­dent be­ta tha­lassemia. Ver­tex and part­ner CRISPR Ther­a­peu­tics are ask­ing the FDA and oth­er agen­cies to ap­prove the gene-edit­ed ther­a­py. — Kyle LaHu­cik