For­ma sees da­ta 'sup­port­ing' sick­le cell ther­a­py dos­ing lev­els; Lian­Bio teams with Tar­sus for eye ir­ri­ta­tion in Chi­na

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 for ato­gepant on Tues­day. The FDA is like­ly to make its de­ci­sion in the late third quar­ter of 2021, Ab­b­Vie said.

Ato­gepant was de­vel­oped specif­i­cal­ly for the pre­ven­tion of mi­graines. As part of its pitch, Ab­b­Vie test­ed ato­gepant 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.

Ab­b­Vie has an­oth­er mi­graine drug in Ubrelvy, which ap­proved for the treat­ment of acute mi­graines with or with­out au­ra back in 2019.

The mi­graine space has seen hefty com­pe­ti­tion in re­cent years. Ubrelvy, al­so known as ubro­gepant, 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. Nurtec is look­ing to add mi­graine pre­ven­tion to its la­bel.

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.

Cor­rec­tion: An ear­li­er ver­sion this piece in­cor­rect­ly re­ferred to Ab­b­Vie’s Ubrelvy mi­graine drug as ato­gepant. It is ubro­gepant. 

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.