Lil­ly en­lists a long-in­cu­bat­ing RNA up­start in new $1.25B part­ner­ship

For near­ly a decade af­ter Im­pe­r­i­al Col­lege Lon­don pro­fes­sor Nagy Habib launched Mi­NA Ther­a­peu­tics around a new kind of RNA in 2008, the com­pa­ny made vir­tu­al­ly no an­nounce­ment: no ven­ture cap­i­tal raised, no col­lab­o­ra­tions signed. Now, it’s like a fire hose.

Robert Habib

On Tues­day, Mi­NA an­nounced a re­search col­lab­o­ra­tion with Eli Lil­ly worth up to $1.25 bil­lion: $25 mil­lion up­front and $245 mil­lion in mile­stones for each of five po­ten­tial tar­gets. The col­lab­o­ra­tors didn’t di­vulge what those tar­gets would be, but said they would come in Lil­ly’s “key ther­a­peu­tic ar­eas,” such as meta­bol­ic dis­eases and dis­eases of the cen­tral ner­vous sys­tem.

“It’s noth­ing odd they haven’t been work­ing on,” CEO Robert Habib, Nagy’s son, told End­points News.

The deal is the third and largest phar­ma col­lab­o­ra­tion Mi­NA has signed since the start of 2020, af­ter a re­search deal with As­traZeneca for an undis­closed sum and an up to $266 mil­lion neu­rol­o­gy-fo­cused part­ner­ship with Servi­er. They al­so grabbed their first ven­ture round in that span, rais­ing $30 mil­lion from the Is­raeli VC aMoon and oth­ers.

Nagy Habib

The flur­ry rep­re­sents the pay­off of over a decade of work on a tech­nol­o­gy Nagy Habib deemed small-ac­ti­vat­ing RNA. These strands func­tion sim­i­lar­ly to the small in­ter­fer­ing RNA that has turned Al­ny­lam in­to a $15 bil­lion com­pa­ny but in re­verse: In­stead of hi­jack­ing cel­lu­lar ma­chin­ery to turn the vol­ume down on a gene, they hi­jack it to turn the vol­ume way up.

The biotech first used saR­NAs for liv­er can­cer, Nagy Habib’s fo­cus as a re­searcher. They used saR­NA to turn up ex­pres­sion of cer­tain genes on the myeloid cells that clus­ter around and of­ten shield a tu­mor from the im­mune sys­tem. The idea was to re­pro­gram the cells from pro-tu­mor myeloid cells to tu­mor-eat­ing myeloid cells and boost the ef­fi­ca­cy of check­point ther­a­pies like Keytru­da. They showed (ear­ly) proof-of-con­cept da­ta at AS­CO last year.

Mi­NA thinks that saR­NA, like mR­NA and RNAi, can be used across a range of dis­eases and, with their new­found part­ners, they’re now work­ing to ex­pand the list. There’s al­so an old­er col­lab­o­ra­tion with Boehringer In­gel­heim on fi­brot­ic liv­er dis­eases, such as NASH.

In the Lil­ly deal, which they first be­gan ne­go­ti­at­ing at JPM 2020 be­fore the pan­dem­ic side­tracked every­thing, the In­di­anapo­lis phar­ma will se­lect the genes they want to am­pli­fy and Mi­NA will de­sign the RNA strands need­ed to do the trick.

“Where we start­ed is im­muno-on­col­o­gy but clear­ly we be­lieve this tech can be ap­plied across dif­fer­ent dis­ease ar­eas,” Robert Habib said. “It’s not lim­it­ed to any cell type.”

Cash from their part­ners has giv­en Mi­NA a good run­way, Habib said. Still, he added, they will look to raise an­oth­er ven­ture round in the next year.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Scott Struthers, Crinetics CEO

Cri­net­ics spins out ra­dio­phar­ma ef­forts in­to a new com­pa­ny, high­light­ing the grow­ing field­'s al­lure

Largely known for its nonpeptide small molecule research, Crinetics has been keeping its radiopharma work comparatively under wraps. But that changed Monday afternoon as the California biotech spun out a new company focused solely on the burgeoning field.

Crinetics launched Radionetics after the closing bell Monday, the company announced, seeding the new entity with $30 million raised from 5AM Ventures and Frazier Healthcare Partners. Radionetics will start with its own radiopharma-centric platform and a pipeline of 10 programs aimed at solid tumors.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.