Dave Greenwald (Deerfield)

Deer­field-backed biotech emerges from stealth with gene ther­a­py de­liv­ery plat­forms from Har­vard and MIT

As a PhD stu­dent at Tufts, Dave Green­wald stud­ied the use of ade­no-as­so­ci­at­ed virus (AAV) vec­tors to de­liv­er gene ther­a­pies to peo­ple with re­tini­tis pig­men­tosa, a group of rare ge­net­ic eye dis­eases that caus­es the reti­na to break down over time.

While he lat­er left lab life, he now sits at the helm of Aper­tu­ra Gene Ther­a­py, a biotech that hopes to cus­tomize AAV vec­tors that are used wide­ly in de­liv­er­ing most gene ther­a­pies. Aper­tu­ra an­nounced its launch from stealth with $67 mil­lion in Se­ries A fi­nanc­ing from Deer­field, where Green­wald is al­so a vice pres­i­dent of busi­ness de­vel­op­ment.

Ben De­v­er­man

The com­pa­ny ac­quired its AAV tech­nol­o­gy from Ben De­v­er­man, a Broad In­sti­tute sci­en­tist who has spent near­ly 15 years study­ing AAV vec­tors.

“When de­vel­op­ing a gene ther­a­py, it has been com­mon to use nat­u­ral­ly oc­cur­ring serotype AAV cap­sids,” De­v­er­man said in a press re­lease.

On the oth­er hand, his lab’s tech­nol­o­gy de­signs “cus­tom AAV cap­sids that have the cho­sen char­ac­ter­is­tics for treat­ing spe­cif­ic dis­eases,” he said, “and we be­lieve this ap­proach will re­sult in new and ef­fec­tive gene ther­a­pies.”

When asked which spe­cif­ic dis­eases Aper­tu­ra plans on go­ing af­ter with the cus­tom AAV cap­sids, Green­wald said, “The nice thing about these plat­forms is that they are ag­nos­tic to cell type and or­gan, so we ac­tu­al­ly use them in pret­ty much any dis­ease.”

How­ev­er, he told End­points News, “we’re not dis­clos­ing spe­cif­ic ther­a­peu­tic ar­eas or in­di­ca­tions at this time.”

Kristi­na Wang

Giv­ing slight­ly more in­sight, Aper­tu­ra’s di­rec­tor of cor­po­rate de­vel­op­ment Kristi­na Wang said, “Nat­ur­al serotype AAVs don’t reach cer­tain tis­sues very well, such as CNS, kid­ney, and a bunch of oth­er tis­sues. We are en­gi­neer­ing AAVs to get to those dif­fi­cult-to-ac­cess tis­sues.”

Aper­tu­ra al­so plans to in­cor­po­rate an­oth­er gene ther­a­py tech­nol­o­gy, known as PESCA, that it ac­quired from a dif­fer­ent Cam­bridge-area sci­en­tist — Michael Green­berg, the chair of Har­vard’s neu­ro­bi­ol­o­gy de­part­ment.

While the AAV tech­nol­o­gy is about what goes on around the cap­sid, “the tech­nol­o­gy from Har­vard is in­side the cap­sid, so it’s more about the reg­u­la­tion,” Green­wald said. “So once we get the gene ther­a­py to the right place, do we turn it on in the right cells at the right amount?”

PESCA can fine-tune the ge­net­ic pay­load of a ther­a­py, “po­ten­tial­ly avoid­ing off-tar­get tox­i­c­i­ties, and com­bined with our AAV cap­sids, can po­ten­tial­ly avoid some of the high sys­temic dosage re­quire­ments as in some of the cur­rent gene ther­a­pies,” added Wang, who worked in Green­berg’s lab while she at­tend­ed grad school at Har­vard.

The tox­i­c­i­ty from high dos­es of AAV-de­liv­ered gene ther­a­pies has been an on­go­ing safe­ty con­cern. In a re­port pub­lished last year, FDA reg­u­la­tors not­ed that 35% of near­ly 150 tri­als with AAV gene ther­a­pies re­port­ed se­ri­ous ad­verse events, in­clud­ing deaths, re­lat­ed to the AAV-de­liv­ered treat­ments.

Aper­tu­ra hopes to present its pre­clin­i­cal da­ta lat­er this year, Green­wald said.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

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Athena Countouriotis, Avenzo Therapeutics CEO (website via Nasdaq)

Ex-Turn­ing Point ex­ecs plan to have their next bet, Aven­zo, on the Nas­daq next sum­mer

The crew at Turning Point Therapeutics is back together for a new biotech that wants to acquire early-stage oncology small molecules, including antibody drug conjugates, and potentially form partnerships with China-based drug developers for ex-China rights as it eyes a speedy leap onto the Nasdaq around this time next year, CEO Athena Countouriotis told Endpoints News.

After selling Turning Point to Bristol Myers Squibb, announced at the onset of last year’s ASCO confab, she and colleague Mohammad Hirmand founded Avenzo Therapeutics. The CEO and CMO already have approximately $200 million in seed and Series A financing from five big-name investors to evaluate which drugs to bring into its pipeline. That includes SR One, OrbiMed, Foresite Capital, Citadel’s Surveyor Capital and Lilly Asia Ventures. Bidding wars for assets have led Avenzo to miss out on some deals in recent months, but the biotech has three active term sheets and hopes to bring in its first asset in the third quarter, Countouriotis said in a Friday morning interview.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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Car­mot rais­es an­oth­er $150M for obe­si­ty drugs, though race by com­peti­tors is well un­der­way

Carmot Therapeutics announced a $150 million Series E round that it plans to use to fund several trials of its obesity drugs.

Its lead candidate is a GLP-1/GIP dual receptor modulator heading into testing for obesity and diabetes. The latest round brings the startup’s total raised to around $370 million, according to the company.

Similar to Eli Lilly’s Mounjaro, Carmot’s lead drugs target hormones that together can impact insulin production and appetite. Its lead compound is CT-388, a once-weekly injection it is taking into Phase II. It also has a daily injection with the same mechanism and an oral GLP-1; a Phase I trial is in the works.

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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