Look­ing to make a splash in sick­le cell, For­ma Ther­a­peu­tics pre­sent­ed new da­ta they hope can im­prove their shot at an even­tu­al FDA OK.

As part of an on­go­ing Phase I study, For­ma said its FT-4202 pro­gram showed enough he­mo­glo­bin in­creas­es to sup­port the dos­es se­lect­ed in the up­com­ing Phase II/III tri­al, which is cur­rent­ly en­rolling. Da­ta from two co­horts il­lus­trat­ed 10 of 14 pa­tients tak­ing the once-dai­ly ther­a­py saw a he­mo­glo­bin in­crease of at least 1 g/dL from base­line over the two-week treat­ment pe­ri­od.

The re­sults sup­port “the eval­u­a­tion of safe­ty and ef­fi­ca­cy at an up­per range of 400 mg in our Phase II/III tri­al,” CMO Patrick Kel­ly said in a state­ment.

For­ma’s da­ta come from co­horts mea­sur­ing a 300 mg dose and a 600 mg dose. In the Phase I study, six of sev­en in­di­vid­u­als in the 300 mg arm saw the ap­pro­pri­ate he­mo­glo­bin in­crease, while four of sev­en tak­ing a 600 mg dose reached the thresh­old.

The Wa­ter­town, MA-based biotech is con­tin­u­ing to eval­u­ate the ben­e­fit of FT-4202 be­yond 14 days of treat­ment as part of an on­go­ing open-la­bel ex­ten­sion. That pro­gram is mea­sur­ing 400 mg dos­es in pa­tients tak­ing the ex­per­i­men­tal drug for 12 weeks.

Da­ta from Tues­day’s high­er co­hort re­main blind­ed, but For­ma not­ed an ini­tial analy­sis sug­gests a sim­i­lar safe­ty pro­file to the 300 mg dose. Ad­di­tion­al­ly, there were no dose-lim­it­ing or treat­ment-re­lat­ed side ef­fects, For­ma said.

FT-4202 is tak­ing a dif­fer­ent ap­proach to sick­le cell dis­ease than those seen from No­var­tis and Glob­al Blood Ther­a­peu­tics, which saw their drugs ap­proved in the last cou­ple of years. While their com­peti­tors at­tempt to re­lieve the dis­ease’s worst symp­toms or com­pli­ca­tions, For­ma says its pro­gram mod­i­fies the course and un­der­ly­ing bi­ol­o­gy of the dis­ease with­out af­fect­ing the genome. It ac­ti­vates an en­zyme called pyru­vate ki­nase-R that is sup­posed to help pre­vent sick­ling and help he­mo­glo­bin bind to oxy­gen.

For­ma’s pro­gram al­so dif­fers from com­pa­nies that are at­tempt­ing to of­fer a cure for sick­le cell dis­ease through gene ther­a­pies, such as blue­bird bio, Ed­i­tas and Ver­tex/CRISPR Ther­a­peu­tics.

Lian­Bio and Tar­sus part­ner on eye ir­ri­ta­tion treat­ment in Chi­na

Per­cep­tive’s Chi­na play has a new part­ner and some new cash.

Lian­Bio has teamed up with Tar­sus Phar­ma­ceu­ti­cals to de­vel­op a ther­a­py for eye ir­ri­ta­tion caused by De­mod­ex mites, a com­mon ec­topar­a­site found on hu­mans. The agree­ment calls for a $15 mil­lion up­front pay­ment to Tar­sus, with the phar­ma be­ing el­i­gi­ble for up to $185 mil­lion in mile­stones.

In ex­change, Lian­Bio will gain ex­clu­sive rights to com­mer­cial­ize the treat­ment in Chi­na, Hong Kong, Macau and Tai­wan.

The ther­a­py in ques­tion is called TP-03, a 0.25% loti­lan­er oph­thalmic so­lu­tion de­signed to erad­i­cate mites that have in­fest­ed the eye­lid mar­gin. Tar­sus says it has com­plet­ed four Phase II tri­als of TP-03 in De­mod­ex ble­phar­i­tis, all of which met their re­spec­tive end­points with no sig­nif­i­cant side ef­fects pop­ping up.

“With more than 100 mil­lion es­ti­mat­ed De­mod­ex ble­phar­i­tis and Mei­bo­mi­an Gland Dis­ease pa­tients in Chi­na…we’ve se­lect­ed TP-03 to an­chor Lian­Bio’s oph­thal­mol­o­gy port­fo­lio,” Lian­Bio chair­man Kon­stan­tin Poukalov said in a state­ment.

Per­cep­tive launched Lian­Bio as its first in-house start­up last Au­gust, fol­low­ing up two months lat­er with a $310 mil­lion raise. Among a host of oth­er mar­quee VCs, the syn­di­cate al­so in­clud­ed Pfiz­er, which in No­vem­ber chipped in an­oth­er $70 mil­lion to co-de­vel­op pipeline pro­grams.

FDA agrees to re­view Ab­b­Vie’s mi­graine pre­ven­tion pitch

The mi­graine com­pe­ti­tion be­tween Ab­b­Vie and Bio­haven con­tin­ues, as the FDA ac­cept­ed Ab­b­vie’s NDA for pre­ven­ta­tive treat­ment on Tues­day.

Ab­b­Vie’s Ubrelvy was ap­proved for the treat­ment of mi­graines back in 2019, but now the com­pa­ny is look­ing to ex­pand that in­di­ca­tion in­to pre­ven­tion. The FDA is like­ly to make its de­ci­sion in the late third quar­ter of 2021, Ab­b­Vie said.

In or­der to gain the ex­pan­sion, Ab­b­Vie test­ed Ubrelvy in about 2,500 pa­tients with four to 14 mi­graine days per month. The da­ta come from at least three clin­i­cal stud­ies, a Phase III tri­al, a Phase IIb/III study and a Phase III long-term safe­ty study.

Ubrelvy, al­so known as ato­gepant, has gone up against Bio­haven’s rimegepant, sold as Nurtec. Both drugs are oral CGRP re­cep­tor an­tag­o­nists, with Nurtec pub­lish­ing its most re­cent Phase III da­ta back in De­cem­ber, and both are look­ing to add mi­graine pre­ven­tion to their la­bels.

Eli Lil­ly al­so has a mi­graine drug on the mar­ket in Reyvow, ap­proved in Oc­to­ber 2019, but it tar­gets the 5-HT1F re­cep­tor.

For­tis forges ahead with $40M Se­ries A to ad­vance mul­ti­ple myelo­ma pro­gram

About four and a half years since de­but­ing with an $18 mil­lion start­up round, For­tis Ther­a­peu­tics has re­turned to the ven­ture well. The San Diego-based biotech pulled in a new $40 mil­lion Se­ries A on Mon­day, as it looks to con­tin­ue de­vel­op­ment of an an­ti­body drug con­ju­gate against CD46.

The pro­gram, called FOR46, binds a spe­cif­ic con­for­ma­tion­al epi­tope of CD46, For­tis says. CD46 acts as part of the body’s im­mune de­fense sys­tem and ap­pears to be spe­cif­ic to tu­mor cells, the com­pa­ny added.

For­tis says it’s com­plet­ed sin­gle pa­tient co­horts and is now en­rolling an ex­pan­sion co­hort for the treat­ment of re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma.

Ex­ist­ing in­vestors such as Aval­on, Bregua, Lil­ly Asia Ven­tures, Os­age Uni­ver­si­ty Part­ners, and Vi­vo Cap­i­tal all par­tic­i­pat­ed. New in­vestors al­so joined in — the Myelo­ma In­vest­ment Fund, and Ful­crum 2020.

Ox­ford Nanopore pre­pares Lon­don IPO

DNA se­quenc­ing biotech Ox­ford Nanopore is prep­ping an IPO — for the Lon­don stock ex­change. The com­pa­ny sent in­vestors a no­tice Tues­day morn­ing stat­ing its in­tent to go pub­lic, but did not dis­close fur­ther de­tails.

Ox­ford Nanopore’s move will like­ly be seen as a big win for UK Prime Min­is­ter Boris John­son’s gov­ern­ment, Bloomberg News re­port­ed, as it looks to at­tract life sci­ences in­vest­ment in a post-Brex­it cli­mate. John­son re­port­ed­ly met with the biotech last De­cem­ber in an ef­fort to con­vince them to list in Lon­don.

The biotech, spun out of Ox­ford Uni­ver­si­ty, boasts “scal­able” DNA and RNA se­quenc­ing tech that can range from bench-top de­vices to pock­et-sized ma­chines. It had pre­vi­ous­ly been pri­vate­ly val­ued at al­most $2 bil­lion.



Source link