PJ Anand, Alcyone Therapeutics CEO

A gene ther­a­py biotech is burst­ing on­to the scene with 12 pro­grams in tow. An ul­tra-rare neu­ro dis­ease is up first

In down­town Low­ell, MA, tucked in­to one of the coun­try’s old­est sur­viv­ing tex­tile mill com­plex­es is a new biotech toil­ing away on next-gen CNS ther­a­pies for se­vere neu­ro­log­i­cal dis­or­ders. And it’s start­ing with Rett syn­drome — a rare neu­rode­vel­op­men­tal con­di­tion that al­most ex­clu­sive­ly af­fects girls.

Kathrin Mey­er

Al­cy­one Ther­a­peu­tics emerged from stealth mode Wednes­day with $23 mil­lion from RTW In­vest­ments and 12 new gene ther­a­py pro­grams in the works. The com­pa­ny was cre­at­ed last sum­mer, short­ly af­ter founder and CEO PJ Anand met Kathrin Mey­er, a prin­ci­pal in­ves­ti­ga­tor in the gene ther­a­py unit at the Abi­gail Wexn­er Re­search In­sti­tute at Na­tion­wide Chil­dren’s Hos­pi­tal.

“We de­cid­ed that there was so much mu­tu­al syn­er­gy,” Anand told End­points News, lead­ing them to ex­e­cute a col­lab­o­ra­tion last year.

The com­pa­ny is in pre-IND en­abling stud­ies for one of its lead can­di­dates, AC­TX-101 — a Rett syn­drome treat­ment us­ing X-re­ac­ti­va­tion tech­nol­o­gy. Its oth­er lead can­di­date, AC­TX-401, is a gene ther­a­py for spinal mus­cu­lar at­ro­phy with res­pi­ra­to­ry dis­tress type 1 (SMARD1), for which it will file an IND this year.

Re­searchers at the Bay­lor Col­lege of Med­i­cine iden­ti­fied mu­ta­tions in a gene called MECP2 as the cause of Rett syn­drome back in 1999. The dis­or­der caus­es se­vere im­pair­ments that af­fect a child’s abil­i­ty to speak, walk, eat and breathe eas­i­ly, a com­mon sign be­ing con­stant repet­i­tive hand move­ments. The chal­lenge with con­ven­tion­al trans­gene re­place­ment, Anand ex­plained, is that over­pro­duc­tion of MECP2 is just as bad as un­der­pro­duc­tion.

“It is one of those Goldilocks in­di­ca­tions where you want to have the right amount of pro­tein,” he said. “The po­ten­tial of this plat­form is to in­crease the ef­fi­cien­cy of gene ther­a­py not by 1 time or 2 times, but by mul­ti­ple times.”

Al­cy­one is cou­pling its pre­cise CNS de­liv­ery plat­form with X-chro­mo­some re­ac­ti­va­tion tech­nol­o­gy, so in­stead of just re­plac­ing the gene in the ac­tive X-chro­mo­some (and risk­ing over­ex­pres­sion of MECP2), they’re se­lec­tive­ly re­ac­ti­vat­ing ar­eas in the in­ac­tive X-chro­mo­some to get MECP2 pro­duc­tion.

Anand has big plans to make this pro­gram a “pipeline in a prod­uct,” where the same gene prod­uct can be ap­plied to oth­er X-chro­mo­some-re­lat­ed syn­dromes. Pri­or to this, he found­ed Anun­cia, a spin­off look­ing to treat hy­dro­cephalus and cere­brospinal flu­id dys­func­tions, and co-found­ed the med­ical de­vice com­pa­ny Arthrome­da.

Al­cy­one’s join­ing a host of oth­er com­pa­nies on the hunt for the first Rett syn­drome ther­a­py, in­clud­ing New York biotech Anavex, which got fast track sta­tus for its blar­came­sine back in 2020. Aca­dia Phar­ma­ceu­ti­cals, mak­er of the Parkin­son’s dis­ease drug Nu­plazid, en­tered its trofine­tide in a piv­otal Phase III study for Rett syn­drome in late 2019. Newron Phar­ma­ceu­ti­cals dropped out of the race last May, culling its Rett syn­drome pro­gram af­ter sari­zotan failed a Phase II/III tri­al.

The 35-per­son team at Al­cy­one is al­so de­vel­op­ing can­di­dates us­ing con­ven­tion­al trans­gene re­place­ment, vec­tor­ized ex­on skip­ping and pro­mo­tor mod­u­la­tion.

“Our next-gen­er­a­tion pre­ci­sion de­liv­ery plat­form shows strong po­ten­tial in over­com­ing the fun­da­men­tal chal­lenge in CNS ther­a­py de­vel­op­ment,” Anand said.

Cor­rec­tion: Al­cy­one plans on fil­ing an IND this year for AC­TX-401, not AC­TX-101. 

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Days af­ter con­tro­ver­sy greet­ed Bio­gen's block­buster Alzheimer's OK, the big biotech con­cedes a set­back on the tau front

Just days after triggering a maelstrom of controversy with their decision to launch an unproven Alzheimer’s drug with a $56,000 price, Biogen $BIIB is back with the latest data on its mid-stage tau drug.

And it’s not good.

The big biotech says that gosuranemab — targeted at tau, the second leading drug target in Alzheimer’s — flat failed its Phase II and will now be taken out and dumped in the mass grave for all but one other Alzheimer’s drug in the past generation.

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Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Richard Mason, Apollo Therapeutics CEO

A UK biotech looks to bring al­liance be­tween GSK, J&J, As­traZeneca, and top uni­ver­si­ties state­side with its first megaround

Richard Mason was working at the J&J Innovation Center in London about six years ago when the company linked arms with two other pharmas and three of the UK’s leading universities to form Apollo Therapeutics — a translational medicine group aimed at spinning out new therapies. Now he’s running the show at Apollo, and he’s pulled in $145 million to drive those programs into the clinic.

Mason unveiled what he calls Apollo’s “first institutional financing” early Thursday morning, which he plans on using to beef up operations both in the UK and the US, and advance a portfolio of more than 15 programs.

As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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Franz-Werner Haas, CureVac CEO (Christoph Schmidt/picture-alliance/dpa/AP Images)

UP­DAT­ED: Cure­Vac blames vari­ants as a close­ly-watched Covid vac­cine goes down in flames, fail­ing piv­otal study with woe­ful da­ta

CureVac was widely expected to come in with a late but likely late-stage winner in the race to develop new vaccines for the Covid-19 pandemic. Instead, late Monday, the German biotech says their mRNA candidate CVnCoV flat failed a pivotal trial — quashing any hopes for a quick entry in the blockbuster field and gutting their share price.

CVnCoV demonstrated an interim vaccine efficacy of 47% against COVID-19 disease of any severity and did not meet prespecified statistical success criteria. Initial analyses suggest age and strain dependent efficacy.

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Yaron Pereg, KAHR CEO

An Is­raeli biotech is look­ing to make its name in the jam-packed CD47 space. Is bi­func­tion­al­i­ty the an­swer?

Jerusalem’s KAHR Medical had been working on fusion proteins for autoimmune disorders for about 10 years, before “issues in development” spurred then-new CEO Yaron Pereg to change gears in 2017.

“We had done our gap analysis and thought, ‘This is a very cool technology and very interesting platform, why won’t we start from scratch, do things different and also select more novel targets?’” he said.

Samantha Du, Zai Lab

Chi­nese on­col­o­gy spe­cial­ist Zai Lab bro­kers a deal with Macro­Gen­ics for up to 4 bis­pecifics with a mod­est down pay­ment

Samantha Du’s Zai Lab has earned its reputation as a Chinese oncology partner of choice with an aggressive licensing strategy to tap that growing market. Now, a West Coast bispecifics player with a lead collaboration molecule already identified will add its name to Zai Lab’s growing rolodex.

Zai Lab will shell out $55 million upfront — $25 million in cash and $30 million in equity — for a mix of Asian and global rights to four of San Francisco-based MacroGenics’ bispecific antibodies, with one lead molecule already in development, the partners said Wednesday.

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