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. 

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Ken Greenberg, SonoThera CEO

Gene ther­a­py goes acoustic as ARCH-backed biotech launch­es with ul­tra­sound gene de­liv­ery plat­form

After co-founding two biotechs off virus-based therapies, one for pain and one for cancer, Ken Greenberg decided to go in a different direction for his newest biotech, SonoThera.

Based out of San Francisco, SonoThera announced Monday morning that it raised $60.75 million to develop new gene therapies — but delivered by ultrasound, which Greenberg says can address the major challenges facing more conventional viral gene therapies.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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