Chas­ing a 'mas­ter mod­u­la­tor' in the en­do­cannabi­noid sys­tem, biotech up­start promis­es to shake up neu­ropsy­chi­atric treat­ments

For two years in the ear­ly 2000s, new dis­cov­er­ies and bud­ding en­thu­si­asm around the en­doge­nous cannabi­noid sys­tem — so named be­cause it’s re­spon­si­ble for the ef­fects of cannabis in the hu­man body — cul­mi­nat­ed in a mar­ket­ed drug: Sanofi’s Acom­plia.

An­drea Chic­ca

The first drug to specif­i­cal­ly tar­get the cannabi­noid re­cep­tor CB1, it was ul­ti­mate­ly with­drawn from the EU and Brazil due to find­ings that it may cause sui­ci­dal thoughts de­spite be­ing ef­fec­tive in treat­ing obe­si­ty. The FDA nev­er ap­proved it.

“For some years the com­pa­nies be­came a bit re­luc­tant to touch the re­cep­tor be­cause of this po­ten­tial ef­fect,” said An­drea Chic­ca, then a post­doc study­ing can­cer bi­ol­o­gy.

But the pen­du­lum has swung back again, ac­cord­ing to Chic­ca — whose biotech start­up, Synen­dos, has just gath­ered $21.85 mil­lion (CHF$20 mil­lion) to prove its suite of se­lec­tive en­do­cannabi­noid re­up­take in­hibitors can of­fer a brand new way to treat neu­ropsy­chi­atric dis­or­ders.

Chic­ca, a long­time re­searcher at the Uni­ver­si­ty of Bern, is CEO, CSO and, for now, the on­ly full-time em­ploy­ee of the biotech start­up.

Jürg Gertsch

The idea draws from a decade of ba­sic sci­ence work by him­self and Jürg Gertsch, chas­ing pro­teins known as en­do­cannabi­noid trans­porters. Even­tu­al­ly, they re­al­ized that the com­pounds they were gen­er­at­ing to block cannabi­noid re­up­take for ex­per­i­men­tal use could have po­ten­tial as hu­man med­i­cines, and de­vised a sec­ond gen­er­a­tion of what they called SERIs.

With­out giv­ing away the pre­cise tar­get, Chic­ca not­ed that what they’re go­ing af­ter sits up­stream to the clas­sic neu­ro­trans­mit­ters: sero­tonin, glu­ta­mate, GA­BA and oth­ers.

In oth­er words, it’s a “mas­ter mod­u­la­tor” that be­comes dys­reg­u­lat­ed in CNS dis­or­ders and trig­gers a cas­cade to the down­stream sig­nal­ing.

While Chic­ca ac­knowl­edges the op­ti­mism around psy­che­del­ic drugs, their in­trin­sic risks mean pa­tients must be treat­ed in very spe­cif­ic hos­pi­tal set­tings un­der tight con­trol of doc­tors.

“So they can­not re­al­ly be­come pills to be giv­en to a pa­tient that they can take at home for months,” he said.

Si­mon Rus­sell

Even cannabis, which is much less risky, is as­so­ci­at­ed with side ef­fects like cog­ni­tive, learn­ing and mem­o­ry is­sues. Synen­dos’ small mol­e­cules are de­signed to side­step those ex­act prob­lems while re­tain­ing the an­ti-stress func­tion. They can al­so pen­e­trate the brain, at least in an­i­mal mod­els — a nice plus for a com­pound Synen­dos is po­si­tion­ing for large in­di­ca­tions like post-trau­mat­ic stress dis­or­der, anx­i­ety, com­pul­sive be­hav­ior and even de­pres­sion.

“We aim not to ac­ti­vate the sys­tem from out­side, but re­store the en­doge­nous cannabi­noid sys­tem,” Chic­ca said. “So by in­creas­ing the lev­el of the en­doge­nous mol­e­cules that are al­ready present in our body, we don’t need to add any­thing.”

If suc­cess­ful — and this is no guar­an­tee in an area akin to a mine­field for drug de­vel­op­ers — Chic­ca sees Synen­dos ex­pand­ing to oth­er ar­eas such as neu­roin­flam­ma­tion.

The Se­ries A should grow the team and take the com­pa­ny through ini­tial, healthy vol­un­teers test­ing fol­lowed by a proof-of-con­cept in a yet-to-be-an­nounced in­di­ca­tion that links anx­i­ety, stress and mood re­lat­ed dis­or­ders. It’s al­so the first fi­nanc­ing Synen­dos has raised since spin­ning out of the Uni­ver­si­ty of Bern and the re­search con­sor­tium NC­CR Tran­sCure, as Chic­ca and Gertsch had been re­ly­ing on non-di­lu­tive fund­ing while work­ing with Base­Launch.

Si­mon Rus­sell, an en­tre­pre­neur-in-res­i­dence at the Basel-based ac­cel­er­a­tor and in­cu­ba­tor who has a full time job as CBO of Ome­icos Ther­a­peu­tics, joined the duo as a co-founder and board mem­ber.

Kur­ma Part­ners and Sun­stone Life Sci­ence Ven­tures led the round, with par­tic­i­pa­tion from BERN­I­NA BioIn­vest, Schroder Ad­veq, High-Tech Grün­der­fonds, Licht­stein­er Foun­da­tion, Es­sen­tial In­vest­ments, Zürcher Kan­ton­al­bank and pri­vate in­vestors.

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

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