One of the more than a dozen bid­ders for Dif­fu­sion Phar­ma­ceu­ti­cal­s' spot on Nas­daq has pre­vailed.

Boston biotech EIP Phar­ma will merge with Dif­fu­sion in an all-stock deal, with plans to start a Phase IIb clin­i­cal tri­al in the com­ing months in a com­mon form of de­men­tia with no ap­proved treat­ments. The com­bined com­pa­ny will be re­named Cer­voMed.

The nine-year-old pri­vate­ly-held EIP is work­ing on a for­mer Ver­tex drug that it will test in a 160-per­son Phase IIb in pa­tients with de­men­tia with Lewy bod­ies, or DLB. The Na­tion­al In­sti­tute on Ag­ing is ex­pect­ed to fund that tri­al with a $21 mil­lion grant. With the re­verse merg­er, slat­ed for clos­ing in the mid­dle of this year, EIP will be fund­ed through that read­out in the sec­ond half of 2024. EIP's eq­ui­ty and debt hold­ers will own about 77.25% of the com­bined com­pa­ny.

Dif­fu­sion, a Vir­ginia biotech fo­cused on hy­pox­ia, put up a "for sale" sign last fall, and in De­cem­ber said it had re­ceived more than 15 bids. The com­pa­ny had about $25.9 mil­lion in cash, cash equiv­a­lents and mar­ketable se­cu­ri­ties at the end of last Sep­tem­ber.

EIP CEO John Alam will lead the new com­pa­ny, whose name is the an­gli­cized and short­ened ver­sion of the French word for brain and mind, cerveau, he said.

Alam co-found­ed EIP in 2014 and has raised about $40 mil­lion in eq­ui­ty and a lit­tle more than $10 mil­lion in con­vert­ible debt, he told End­points News. The ex­ec­u­tive pre­vi­ous­ly led re­search of age-re­lat­ed dis­eases at Sanofi and be­fore that spent about a decade at Ver­tex, where he re­tired as chief med­ical of­fi­cer in 2008.

GSK is dish­ing out $90 mil­lion cash to add an an­ti­fun­gal drug to its com­mer­cial port­fo­lio, in a deal spot­light­ing the phar­ma gi­ant’s grow­ing fo­cus on in­fec­tious dis­eases.

The up­front will lock in an ex­clu­sive li­cense to Scynex­is’ Brex­afemme, which was ap­proved in 2021 to treat a yeast in­fec­tion known as vul­vo­vagi­nal can­didi­a­sis, ex­cept in Chi­na and cer­tain oth­er coun­tries where Scynex­is al­ready out-li­censed the drug.

The biotech has been look­ing for a part­ner af­ter it said last Oc­to­ber it was let­ting go 40% of its staff and wind­ing down pro­mo­tion­al ac­tiv­i­ties for the drug, say­ing the drug "re­quires a larg­er or­ga­ni­za­tion with more re­sources, more women’s health ex­pe­ri­ence and a big­ger com­mer­cial foot­print." In the first three quar­ters of 2022, the drug made $3.6 mil­lion, just 4% of what GSK is pay­ing up­front.

But GSK be­lieves the drug could reach peak sales of over half a bil­lion USD, chief com­mer­cial of­fi­cer Luke Miels said dur­ing a me­dia call. GSK would be able to pro­vide the more ro­bust com­mer­cial in­fra­struc­ture for Brex­afemme that Scynex­is "was not able to do be­cause of the scale," he not­ed.

"We can bring a lot more scale to this and a lot more in­vest­ment to this prod­uct to prop­er­ly ed­u­cate physi­cians and al­so make pa­tients aware of it. And that's why we're con­fi­dent that we can change the tra­jec­to­ry of this prod­uct," Miels said.

Bel­gian biotech Con­fo Ther­a­peu­tics has land­ed $183 mil­lion, plus po­ten­tial roy­al­ties, in a drug-dis­cov­ery deal with Dai­ichi Sankyo.

Ear­ly Thurs­day, Con­fo Ther­a­peu­tics put out word of the deal that will be fo­cused on small mol­e­cule an­tag­o­nists to go af­ter an undis­closed tar­get that the com­pa­ny says is as­so­ci­at­ed with CNS dis­eases.

Con­fo CEO Cedric Ververken told End­points News that Dai­ichi orig­i­nal­ly reached out to learn about the biotech’s tech­nol­o­gy. He added that Con­fo, found­ed in 2015, will use its plat­form to drug a GPCR tar­get that Dai­ichi has strug­gled with in­ter­nal­ly.

What Con­fo does is around frag­ment-based GPCR drug dis­cov­ery. Ververken not­ed that in GPCR drug dis­cov­ery, there are two main ways to do it. One, test­ing mil­lions of com­pounds against a par­tic­u­lar re­cep­tor in or­der to find a hit does not al­ways work out be­cause of the shape of the GPCR pock­ets.

“And I al­ways make the par­al­lel to a game of Tetris, for ex­am­ple, where you have pret­ty large Tetris blocks, and you try to make that fit in the pock­et of in­ter­est. And typ­i­cal­ly, it does­n't work re­al­ly well,” the chief ex­ec not­ed.

Rather than this ap­proach, Ververken not­ed that Con­fo es­sen­tial­ly us­es the squares that make up the Tetris blocks — that way, Con­fo can al­ways find some­thing that ini­tial­ly fits. And from there, Con­fo can grow that frag­ment in­to a mol­e­cule that can go af­ter a tar­get.

In the ear­ly 1990s, Math­ai Mam­men was a teach­ing as­sis­tant in Greg Ver­dine’s Sci­ence B46 course at Har­vard. In June, the for­mer R&D head at John­son & John­son will suc­ceed Ver­dine as CEO, pres­i­dent and chair of Fog­Phar­ma, the same month the sev­en-year-old biotech kick­starts its first clin­i­cal tri­al.

Af­ter lead­ing R&D at one of the largest drug­mak­ers in the world, tak­ing the com­pa­ny through more than half a dozen drug ap­provals in the past few years, not to men­tion a Covid-19 vac­cine race, Mam­men de­part­ed J&J last month and will take the helm of a Cam­bridge, MA biotech at­tempt­ing to go af­ter what Ver­dine calls the “true em­per­or of all onco­genes” — be­ta-catenin.

The two kept in touch af­ter Mam­men de­part­ed Har­vard, af­ter which he co-found­ed Ther­a­vance, spend­ing two decades there be­fore tak­ing a brief SVP stint at Mer­ck to lead in­to chief R&D roles at Janssen and its par­ent J&J. Over the years, Fog­Phar­ma pre­sent­ed to Math­ai’s teams at those two Big Phar­mas, Ver­dine told End­points News via email.

“I could have start­ed with some­thing large and then re­ori­ent­ed it; some­thing from scratch, which I con­sid­ered and start­ed pure­ly from a green­field; but Fog was ex­act­ly what I was hop­ing for, in the sense that it was ma­ture, had a very spe­cial plat­form that ap­plied to all parts of med­i­cine, as far as I can tell, across all dis­ease ar­eas,” Mam­men told End­points in a pre­view of Fog­Phar­ma’s an­nounce­ment Thurs­day morn­ing.

Dal­las-based biotech Nanoscope Ther­a­peu­tics un­veiled Phase II re­sults on its gene ther­a­py for a rare eye dis­ease Thurs­day morn­ing.

In the RE­STORE tri­al, 18 pa­tients with re­tini­tis pig­men­tosa got a gene ther­a­py called MCO-010 while nine got place­bo. On a vi­sion test called the MLYMT, the treat­ment group had a one-point greater change over one year in their score com­pared to the place­bo group, the pri­ma­ry end­point of the study. How­ev­er, the p-val­ue was not pro­vid­ed, and the 95% con­fi­dence in­ter­val was 0.0 to 3.0.

All in all, 12 of 18 peo­ple with re­tini­tis pig­men­tosa who got the ther­a­py saw a two lev­el or greater im­prove­ment on the mo­bil­i­ty test for vi­sion called the MLYMT at one year, Nanoscope said. In the place­bo group, 3 of 9 peo­ple saw that lev­el of im­prove­ment.

Nanoscope de­clined to com­ment be­yond the press re­lease.

In re­tini­tis pig­men­tosa, the reti­na breaks down over time and can lead to blind­ness. Nanoscope’s ther­a­py is in­ject­ed di­rect­ly in­to the eye with the goal of restor­ing the eye’s abil­i­ty to de­tect low light lev­els.

Ac­cord­ing to the press re­lease, the ther­a­py came with “no se­ri­ous or se­vere ad­verse events,” though Nanoscope pro­vid­ed few de­tails on safe­ty out­side of that.

Roche's Lux­tur­na is al­ready ap­proved cer­tain sub­types of the eye dis­ease caused by ge­net­ic mu­ta­tions. A num­ber of oth­ers are al­so work­ing on rare eye dis­ease gene ther­a­pies — Janssen and MeiraGTx are test­ing a ther­a­py for X-linked re­tini­tis pig­men­tosa in a Phase III tri­al set to com­plete in March of next year, ac­cord­ing to a fed­er­al clin­i­cal tri­als data­base.

When Vig­il Neu­ro­science filed its IPO pa­pers in late 2021, the biotech re­vealed that the FDA had just cleared its Phase I tri­al — but with a par­tial clin­i­cal hold that lim­it­ed dos­ing to un­der a cer­tain lev­el.

More than a year lat­er, the FDA has lift­ed the hold.

Vig­il is now free to dose VGL101, an an­ti­body tar­get­ing TREM2, at lev­els high­er than 20 mg/kg in its on­go­ing and fu­ture clin­i­cal tri­als in pa­tients with adult-on­set leukoen­cephalopa­thy with ax­on­al spher­oids and pig­ment­ed glia (AL­SP), an in­her­it­ed con­di­tion that af­fects the brain and spinal cord.

“Al­though we be­lieve that 20 mg/kg is a clin­i­cal­ly rel­e­vant dose in AL­SP, we are very pleased that the hold has been lift­ed as we be­lieve it’s im­por­tant to main­tain op­tion­al­i­ty to de­vel­op treat­ments that sup­port pa­tients suf­fer­ing from both rare and com­mon neu­rode­gen­er­a­tive in­di­ca­tions,” pres­i­dent and CEO Ivana Magov­če­vić-Liebisch said in a state­ment.

The biotech has test­ed the drug among both healthy vol­un­teers and pa­tients with AL­SP in tri­als con­duct­ed in the US and Aus­tralia, where it’s been ex­plor­ing dos­es above the 20 mg/kg thresh­old, up to 60 mg/kg. Da­ta from the on­go­ing Phase I tri­al helped con­vince the FDA, ac­cord­ing to the com­pa­ny.

Stifel an­a­lyst Paul Mat­teis not­ed that ul­ti­mate­ly, the high­er dos­es may not be nec­es­sary, but it’s hard to tell un­til Vig­il tests it.

“That said, for mon­o­clon­al an­ti­bod­ies in the brain, more (up to a cer­tain point) is of­ten bet­ter, and giv­en the safe­ty so far, it seems rea­son­able that Vig­il would con­tin­ue to dose es­ca­late fur­ther as tri­als in AL­SP/Alzheimer's progress,” he wrote.

A month af­ter re­veal­ing plans to con­cen­trate on its late-stage im­muno-neu­rol­o­gy pipeline, Alec­tor is trim­ming its head­count by 11%.

The lay­offs will im­pact around 30 em­ploy­ees across the or­ga­ni­za­tion, the com­pa­ny dis­closed in an SEC fil­ing, adding that the plan will “bet­ter align the com­pa­ny’s re­sources” with the new strat­e­gy. With $712.9 mil­lion in cash, cash equiv­a­lents and in­vest­ments as of the end of 2022, Alec­tor be­lieves the re­serves will now get it through 2025.

Like many biotechs, Alec­tor is weath­er­ing a harsh fi­nanc­ing win­ter. Its stock has wilt­ed over the past year, los­ing more than half of its val­ue, and the cur­rent mar­ket cap of about $524 mil­lion is just a frac­tion of the $1.3 bil­lion uni­corn val­u­a­tion it boast­ed in its 2019 IPO.

Alec­tor is now bank­ing on its lead pro­grams to yield pos­i­tive clin­i­cal da­ta be­fore mon­ey runs out — and prove out its the­o­ry that it can go af­ter neu­rode­gen­er­a­tive dis­eases by restor­ing the im­mune bal­ance.

The biotech is en­rolling pa­tients in a piv­otal Phase III tri­al for la­tozinemab (AL001) in at-risk and symp­to­matic pa­tients with fron­totem­po­ral de­men­tia due to a pro­gran­ulin gene mu­ta­tion (FTD-GRN). Alec­tor said in its re­cent quar­ter­ly up­date that it’s prepar­ing to meet with reg­u­la­to­ry agen­cies lat­er this year to see if it can tweak the tri­al de­sign so that it can wrap up the study soon­er, with the goal of get­ting to a read­out in ear­ly 2025.

La­tozinemab is one of two an­ti­bod­ies GSK paid $700 mil­lion up­front back in 2021 to part­ner on, both of which are de­signed to el­e­vate lev­els of pro­gran­ulin — a key reg­u­la­tor of im­mune ac­tiv­i­ty in the brain, ac­cord­ing to the com­pa­ny.

Paul McKen­zie took over as CEO of Aus­tralian phar­ma gi­ant CSL this month, fol­low­ing in the foot­steps of long-time CSL vet Paul Per­reault.

With an eye on mR­NA, and quick­ly com­mer­cial­iz­ing its new, $3.5 mil­lion-per-shot gene ther­a­py for he­mo­phil­ia B, McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte an­swered some ques­tions from End­points News this af­ter­noon about where McKen­zie is go­ing to take the com­pa­ny and what ad­vances may be com­ing to mar­ket from CSL's pipeline. Be­low is a light­ly edit­ed tran­script.

End­points: You took over this month as CEO from long-time CSL vet­er­an Paul Per­reault — in what ways are you go­ing to move the com­pa­ny in­to new ar­eas, and what have you learned from what Mr. Per­reault as he led CSL over the last decade?

McKen­zie: It's a re­al priv­i­lege to be able to be giv­en this op­por­tu­ni­ty to work with this fan­tas­tic man­age­ment team. That ded­i­ca­tion and promise we have to pa­tients and from my first in­ter­ac­tion with CSL, that was crit­i­cal­ly im­por­tant. From Paul, that pas­sion, he knows some of the pa­tients in he­mo­phil­ia by first name, and that's some­thing that won't change. What I al­so learned was that as the or­ga­ni­za­tion grows larg­er, is to re­al­ly fo­cus. It's easy to do lots of things, and hard­er to do a few things very well.

Mi­cro­cap biotech See­los Ther­a­peu­tics is halt­ing en­roll­ment of its study in spin­ocere­bel­lar atax­ia type 3 (al­so known as Macha­do-Joseph dis­ease) be­cause of “fi­nan­cial con­sid­er­a­tions,” and in or­der to fo­cus on oth­er stud­ies, the com­pa­ny said to­day, adding that the pause would be tem­po­rary.

The study will con­tin­ue with the pa­tients who have al­ready en­rolled, and the da­ta from them will be used to de­cide whether to con­tin­ue en­rolling oth­ers in the fu­ture.

See­los SEEL is turn­ing its fo­cus and re­sources in­stead to its study of in­tranasal racemic ke­t­a­mine for acute sui­ci­dal ideation and be­hav­ior in pa­tients with ma­jor de­pres­sive dis­or­der, plan­ning for a da­ta read­out in the third quar­ter of this year. The com­pa­ny is al­so con­tin­u­ing with its Phase II/III study us­ing an in­ves­ti­ga­tion­al mol­e­cule called tre­halose in amy­otroph­ic lat­er­al scle­ro­sis (ALS) with plans for a da­ta re­veal at the end of 2023.

Once the fi­nal re­sults of non-clin­i­cal tox­i­col­o­gy are in, the com­pa­ny al­so plans to start dos­ing its can­di­date sub­lin­gual ke­t­a­mine in a pro­gram for com­plex re­gion­al pain syn­drome (CRPS) while putting non-es­sen­tial pre­clin­i­cal work on hold, CEO Raj Mehra added.

"Our cor­po­rate struc­ture and out­sourced mod­el al­lows us to be nim­ble and make these strate­gic de­ci­sions, en­abling us to ex­tend our cash run­way through da­ta read­out,” Mehra said in a state­ment.

An at­tempt to ex­tend its cash run­way comes just two weeks af­ter See­los put up mil­lions of its com­mon stock for sale, pre­dict­ing gross pro­ceeds of ap­prox­i­mate­ly $11.24 mil­lion be­fore fees and oth­er ex­pens­es. See­los said it would use the net pro­ceeds for “gen­er­al cor­po­rate pur­pos­es” and ad­vance de­vel­op­ment of its can­di­dates. The of­fer­ing was ex­pect­ed to close about March 14.

Oton­o­my may be shut­ting down, but the lessons learned there will live on at an­oth­er biotech work­ing on new treat­ments for hear­ing loss.

San Fran­cis­co-based Spi­ral Ther­a­peu­tics has bought cer­tain as­sets re­lat­ed to three of Oton­o­my’s pro­grams, rang­ing from da­ta, patent rights, and know-how to in­ven­to­ry. That in­cludes da­ta around Oton­o­my’s twice-failed lead pro­gram, OTO-104 (Otividex), a sus­tained-ex­po­sure for­mu­la­tion of dex­am­etha­sone.

In De­cem­ber, Oton­o­my an­nounced it’s let­ting all of its em­ploy­ees go af­ter the board ap­proved a dis­so­lu­tion plan, cap­ping a fruit­less search for strate­gic al­ter­na­tives. But as part of the liq­ui­da­tion process, it ex­plored sell­ing the as­sets still in its pipeline — with pro­ceeds from any sales to be dis­trib­uted back to share­hold­ers.

Spi­ral said it “plans to lever­age valu­able da­ta and in­sights gained from Oton­o­my's 15 years of ex­pe­ri­ence in the field of in­ner ear dis­or­ders" to ac­cel­er­ate its own lead drug’s path to late-stage clin­i­cal tri­als.

For the two as­sets oth­er than OTO-104, "the plan is to re­for­mu­late in­to our nov­el drug de­liv­ery plat­form," Spi­ral CEO Hugo Peris wrote in an email to End­points News.

"The ac­quired da­ta will help us de­fine the tar­get prod­uct pro­file and clin­i­cal study de­sign as we build these in­to new as­sets in our pipeline," he wrote.

The com­pa­ny raised $8.25 mil­lion ear­li­er this year to fund its work on ther­a­pies for in­ner ear dis­or­ders.

In a first, Mer­ck has se­cured a full ap­proval for Keytru­da in a tu­mor ag­nos­tic set­ting — as a treat­ment for any un­re­sectable or metasta­t­ic sol­id tu­mors that are clas­si­fied as mi­crosatel­lite in­sta­bil­i­ty-high (MSI-H) or mis­match re­pair de­fi­cient (dMMR).

The FDA grant­ed Keytru­da ac­cel­er­at­ed ap­proval in this in­di­ca­tion in 2017, and GSK's Jem­per­li fol­lowed suit in 2021. But now it’s con­vert­ed to a full ap­proval for Keytru­da. Be­fore pre­scrib­ing, doc­tors would have to make sure pa­tients car­ry this bio­mark­er, us­ing an FDA-ap­proved test.

Mer­ck pooled da­ta — main­ly over­all re­sponse rates — from three open-la­bel tri­als to sup­port this fil­ing. The first en­rolled 124 pa­tients with ad­vanced MSI-H/dMMR col­orec­tal can­cer that pro­gressed af­ter chemo; the sec­ond en­rolled 373 pa­tients with ad­vanced MSI-H/dMMR non-col­orec­tal can­cers who pro­gressed fol­low­ing pri­or ther­a­py; and the third com­prised sev­en pe­di­atric pa­tients with MSI-H/dMMR can­cers.

By Mer­ck’s analy­sis, Keytru­da showed an ORR of 33.3% across these three tri­als, in­clud­ing a com­plete re­sponse rate of 10.3%, at a me­di­an fol­low-up time of 20.1 months. — Am­ber Tong

Can­del Ther­a­peu­tics is paus­ing en­roll­ment in a Phase II tri­al of its lead vi­ral im­munother­a­py can­di­date, a de­ci­sion it at­trib­ut­es to “cost man­age­ment and dy­nam­ic port­fo­lio man­age­ment ini­tia­tives.”

The tri­al was test­ing CAN-2409, which con­sists of a con­struct en­cod­ing the thymi­dine ki­nase car­ried in an ade­n­ovirus vec­tor, in bor­der­line re­sectable pan­cre­at­ic ade­no­car­ci­no­ma. But ad­di­tion­al fund­ing would be re­quired to keep push­ing that tri­al for­ward, Can­del sug­gest­ed.

De­spite the pause, the biotech ex­pects to present ini­tial da­ta from the tri­al by the end of the year. — Am­ber Tong