Michael Ehlers. Limelight Bio

Michael Ehlers teas­es plan to build the next 'sig­na­ture' ge­net­ic med­i­cine com­pa­ny with $75M from Ap­ple Tree Part­ners

When Michael Ehlers jumped ship at Bio­gen to be­come a ven­ture part­ner and CSO at Ap­ple Tree, he al­so got a new job as CEO of a gene ther­a­py start­up named Lime­light Bio. The stealthy biotech is now ready to shed some — though not much — of its veils.

Seth Har­ri­son

As ATP man­ag­ing part­ner Seth Har­ri­son tells it, the new ven­ture grew out of a strong re­la­tion­ship with the co-founders, Jean Ben­nett and Phil John­son. Ben­nett, who steered the ear­ly re­search on the land­mark gene ther­a­py even­tu­al­ly known as Lux­tur­na along­side Spark Ther­a­peu­tics CSO Kathy High, now leads Penn’s Cen­ter for Ad­vanced Reti­nal and Oc­u­lar Ther­a­pies. Mean­while, John­son left his role at the Chil­dren’s Hos­pi­tal of Philadel­phia to be­come Lime­light’s CSO.

“Ini­tial­ly we were fo­cused on back­ing Jean and CAROT and some reti­nal dy­s­tro­phy gene ther­a­py projects us­ing the stan­dard AAV ap­proach­es,” he said, “but as we got to know the trove of tech­nolo­gies that were pos­si­ble to ac­cess through Jean and Phil, some from Penn, some not, we’ve been very ex­cit­ed about the po­ten­tial of cre­at­ing a ma­jor gene ther­a­py com­pa­ny.”

Jean Ben­nett (via Drex­el)

Click on the im­age to see the full-sized ver­sion

Lime­light is pur­su­ing gene ther­a­py in its broad­est sense. In ad­di­tion to stan­dard gene re­place­ment de­liv­ered by ade­no-as­so­ci­at­ed virus, or AAV, the biotech will al­so ex­plore non-vi­ral ap­proach­es and what they call mul­ti-ex­on gene cor­rec­tion.

The broad spec­trum of tar­gets and modal­i­ties Lime­light is go­ing af­ter makes it unique, said Ben­nett, who’s al­so co-found­ed oph­thal­mol­o­gy gene ther­a­py com­pa­ny Gen­Sight. Some of the ul­tra-rare dis­eases they want to treat would have been dis­missed by most com­pa­nies, she told End­points News.

The po­ten­tial of this suite of tech­nolo­gies to move the nee­dle on ge­net­ic med­i­cine and scale it up across new dis­eases and ge­o­gra­phies was al­so what drew Ehlers to the op­por­tu­ni­ty in the first place. ATP’s promise of sub­stan­tial cap­i­tal to de­ploy in the space prob­a­bly didn’t hurt, ei­ther.

“The de­gree of ef­fi­ca­cy you can get on these ther­a­pies is sub­stan­tial, it’s not just in­cre­men­tal ef­fi­ca­cy, you squint your eyes and you find a p-val­ue,” he said.

Phil John­son

Click on the im­age to see the full-sized ver­sion

With the well-known gene re­place­ment tech, Lime­light boasts of re­fined meth­ods that al­low them to slip in larg­er genes than pre­vi­ous­ly pos­si­ble and bet­ter con­trol gene ex­pres­sion. As for gene cor­rec­tion, Ehlers is care­ful to dis­tin­guish what they’re do­ing from the “old clas­sic type­writer” ef­fect typ­i­cal­ly as­so­ci­at­ed with gene edit­ing.

“We are ex­plic­it­ly us­ing the term ‘cor­rec­tion’ be­cause we’re able to do much more than just one lit­tle point in the gene at a time,” he said. “We can do big por­tions at a time, at en­doge­nous lev­els — and not re­quir­ing ex­oge­nous en­zymes or co-fac­tors” such as Cas9.

He al­so sug­gest­ed their tech doesn’t come with risk of per­ma­nent ge­nom­ic al­ter­ation, hint­ing at RNA or re­versible DNA changes.

Just ex­act­ly how Lime­light will ac­com­plish those feats, or how many staffers are work­ing on the tech plat­forms, or what man­u­fac­tur­ing ops they have in place, Ehlers is not quite ready to dis­close, say­ing mul­ti­ple times to “stay tuned.”

He is, how­ev­er, ea­ger to pre­view that Lime­light ex­pects to be a clin­i­cal com­pa­ny in one to two years, ush­er­ing at least two pro­grams in reti­nal dis­eases. That will be fol­lowed by pro­grams in hema­to­log­ic/meta­bol­ic, neu­ro­log­ic and hear­ing dis­or­ders — each on in­di­ca­tions that they have iden­ti­fied with the quick­est path to clin­i­cal and reg­u­la­to­ry val­i­da­tion.

As Lime­light takes the pub­lic stage on the same day Ehlers’ for­mer col­leagues at Bio­gen present one of biotech’s most an­tic­i­pat­ed datasets of the year at an Alzheimer’s con­fer­ence, will he com­ment on a ques­tion that at least one an­a­lyst has won­dered out loud: How in the world would he have left Bio­gen if he be­lieved in ad­u­canum­ab?

He won’t.

“My main fo­cus is re­al­ly how ex­cit­ed I am about Lime­light and the promise of re­al­ly build­ing a sig­na­ture gene ther­a­py com­pa­ny,” Ehlers said.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

UP­DAT­ED: Eli Lil­ly toss­es a mar­quee pain drug and hits the gas on Alzheimer’s — as Bio­gen’s suf­fer­ing opens mar­ket to ri­vals

The furious chorus of critics that brought sales of Biogen’s ultra controversial Alzheimer’s drug aducanumab (sold as Aduhelm) to a near halt is opening up some big opportunities for a major league rival that has long sought the lead role in this largely untapped megamarket.

In its Q3 update today, Eli Lilly — noted for its dogged persistence in attempting for years to get solanezumab across the FDA finish line — said that it has begun a rolling submission of its rival Alzheimer’s drug donanemab in search of an accelerated approval. Anne White, senior VP of Lilly’s neuroscience unit, acknowledged during the investor call the challenges Biogen has faced with uptake and noted Lilly may face similar hurdles.

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Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

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