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. 

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Geoffrey Porges, new Schrödinger CFO

Long­time an­a­lyst Ge­of­frey Porges de­parts SVB to lead fi­nances at a drug dis­cov­ery shop

Geoffrey Porges has ended his two-decade run as a biotech analyst, as the former SVB Securities vice chair began as CFO of Schrödinger on Thursday.

The long-running analyst, who previously headed up vaccines marketing at Merck before the turn of the millennium, will lead the financial operations of the 700-employee company as Schrödinger broadens its focus from a drug discovery partner to also building out an in-house pipeline, with clinical trial No. 1 set to begin next quarter.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

Joel Dudley, new partner at Innovation Endeavors (Bosch Health Campus)

For­mer Google CEO’s VC is mak­ing a big­ger push in­to the biotech world, hir­ing promi­nent Ther­a­nos skep­tic

Venture capital firm Innovation Endeavors has mainly had its focus on investments across the tech space, but it has been slowly turning its attention to the biotech world. Now, a new partner is coming into the fold showing that its interest in biotech is likely to grow further.

The Silicon Valley-based company, which is headed up by former Google CEO Eric Schmidt, has brought on Joel Dudley as a partner. According to Dudley’s LinkedIn page, he is joining Innovation Endeavors after serving as the chief science officer of biotech startup Tempus Labs since 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”