FDA ac­cepts In­tel­li­a's IND for CRISPR and TCR-T cell ther­a­py; San­té clos­es Fund IV at $260M

Rid­ing the coat­tails of a mas­sive $600 mil­lion cash raise in June, In­tel­lia an­nounced that the FDA ac­cept­ed their IND ap­pli­ca­tion for their gene edit­ing treat­ment NT­LA-5001, built as a treat­ment for acute myeloid leukemia.

The Cam­bridge, MA biotech said that they have plans to start pa­tient screen­ing in a Phase I/IIa study by the end of 2021. The study will eval­u­ate the ef­fects of a sin­gle dose of the treat­ment in adults who have de­tectable AML af­ter hav­ing re­ceived stan­dard first-line ther­a­py. The study will con­tain a dose es­ca­la­tion and ex­pan­sion phase, with up to 54 par­tic­i­pants.

“The FDA’s ac­cep­tance of our IND for NT­LA-5001 is an im­por­tant mile­stone in our pur­suit of de­vel­op­ing ad­vanced cell ther­a­pies uti­liz­ing In­tel­lia’s pro­pri­etary en­gi­neer­ing plat­form to treat pa­tients with can­cer,” said In­tel­lia pres­i­dent and CEO John Leonard.

Out­side of the US, In­tel­lia al­so sub­mit­ted a reg­u­la­to­ry ap­pli­ca­tion to the UK for NT­LA-5001.

San­té clos­es over­sub­scribed Fund IV af­ter rais­ing $260 mil­lion

Texas health­care in­vest­ment firm San­té fi­nal­ly closed its over­sub­scribed Fund IV. The $260 mil­lion fund will make in­vest­ments across ear­ly-stage biotech, healthtech and medtech com­pa­nies, ac­cord­ing to a com­pa­ny state­ment.

Fund IV se­cured com­mit­ments from more than two-dozen part­ners, in­clud­ing new in­sti­tu­tion­al in­vestors in Asia and Latin Amer­i­ca. Re­turn­ing part­ners ac­count­ed for near­ly 90% of the raised funds.

San­té co-founder and man­ag­ing di­rec­tor Kevin La­lande said in a state­ment: “The con­fi­dence and com­mit­ment of our in­vestors led to the quick­est fundraise since in­cep­tion. We are grate­ful for their part­ner­ship as we de­ploy this next fund to cap­i­tal­ize on sig­nif­i­cant health­care op­por­tu­ni­ties and nur­ture cut­ting-edge tech­nolo­gies.”

Zai Lab ex­pands US op­er­a­tions with of­fice in Cam­bridge

Zai Lab is ex­pand­ing its pres­ence in the Unit­ed States — by open­ing an of­fice in biotech hub Cam­bridge. The new of­fice will be the head­quar­ters of dif­fer­ent busi­ness op­er­a­tions such as al­liance man­age­ment, along with le­gal and gov­er­nance func­tions.

“We are ex­cit­ed to ex­pand our pres­ence in the U.S. by open­ing a new Zai Lab of­fice in Cam­bridge,” Zai Lab CEO and chair Saman­tha Du said in a state­ment. “This is an im­por­tant step in Zai Lab’s jour­ney to be­come a glob­al bio­phar­ma­ceu­ti­cal com­pa­ny, a part­ner of choice and a place where our em­ploy­ees can do their best work.”

This an­nounce­ment comes al­most im­me­di­ate­ly af­ter Zai Lab an­nounced on Tues­day that they re­ceived BTD for be­mar­ituzum­ab from the NM­PA. They are now look­ing to ad­vance the drug in­to reg­is­tra­tional tri­als.

Bio­gen re­leas­es re­sults from Phase II study on neu­ropa­thy pain drug

Cam­bridge, MA biotech Bio­gen an­nounced pos­i­tive topline re­sults from its Phase II study of vixot­rig­ine, a non-opi­oid oral pain drug be­ing eval­u­at­ed for the treat­ment of small fiber neu­ropa­thy (SFN).

The CON­VEY study had a dou­ble-blind pe­ri­od, where pa­tients were ad­min­is­tered 200 mg of vixot­rig­ine twice dai­ly. That arm met its pri­ma­ry end­point of change from base­line to week 12 of the dou­ble-blind pe­ri­od in mean av­er­age dai­ly pain (ADP) score.

In the same study, there was an open-la­bel pe­ri­od where all par­tic­i­pants who en­rolled re­ceived a high­er dose of the drug — 350 mg twice dai­ly. This pre­ced­ed the dou­ble-blind por­tion of the study.

While the 350 mg twice dai­ly arm did not meet the pri­ma­ry end­point, it met sta­tis­ti­cal sig­nif­i­cance in the Pa­tient Glob­al Im­pres­sion of Change (PG­IC) at week 12. Da­ta from the study will in­form po­ten­tial dos­es for study in fu­ture Phase III clin­i­cal tri­als.

“We are en­cour­aged by the over­all re­sults of the CON­VEY study,” said Bio­gen SVP and head of the ther­a­peu­tics de­vel­op­ment unit Kather­ine Daw­son. “We are grate­ful to all the par­tic­i­pants, in­ves­ti­ga­tors and study staff who con­tributed to this study and al­lowed us to eval­u­ate vixot­rig­ine as a non-opi­oid treat­ment op­tion.”

Niger­ian start­up 54gene rais­es $25 mil­lion in Se­ries B — Re­port

Ge­nomics start­up 54gene re­cent­ly raised $25 mil­lion in a Se­ries B round to ad­vance its pre­ci­sion med­i­cine ca­pa­bil­i­ties.

Ac­cord­ing to TechCrunch, the round comes a year af­ter the com­pa­ny, found­ed by Abasi Ene-Obong in 2019, raised $15 mil­lion in a Se­ries A and two years af­ter it closed a $4.5 mil­lion seed round.

The com­pa­ny launched a ge­net­ic se­quenc­ing and mi­croar­ray lab in La­gos, Nige­ria last Sep­tem­ber through a part­ner­ship with Il­lu­mi­na.

Ac­cord­ing to a press re­lease is­sued by Il­lu­mi­na last year, the fa­cil­i­ty will look at ge­net­ic in­for­ma­tion for health re­search and drug de­vel­op­ment — and Ene-Obong fur­ther said that the lab will do whole-genome se­quenc­ing and whole-ex­ome se­quenc­ing as part of the lab’s work.

New York biotech Ban­tam com­pletes $25 mil­lion in seed fund­ing

Ban­tam Phar­ma­ceu­ti­cal com­plet­ed a $25 mil­lion seed fund­ing round to fi­nal­ize ac­tiv­i­ties for its lead pro­gram BTM-3566. The funds are in­tend­ed to fin­ish prepa­ra­tions so the com­pa­ny can file its IND, which it plans to do in Feb­ru­ary — along with mak­ing prepa­ra­tions for first-in-hu­man clin­i­cal tri­als.

The fund­ing was co-led by Ban­tam’s cur­rent in­vestors.

Ban­tam is look­ing to start Phase I clin­i­cal stud­ies in pa­tients with B-cell hema­to­log­i­cal ma­lig­nan­cies in Q2 of 2022. Ban­tam has es­tab­lished its op­er­a­tions in the North Car­oli­na Re­search Tri­an­gle.

“This fund­ing en­ables Ban­tam Phar­ma­ceu­ti­cal to be ful­ly pre­pared to en­ter the clin­ic for first in hu­man stud­ies in ear­ly 2022, rep­re­sent­ing a ma­jor mile­stone. We are ex­cit­ed about mov­ing to the next stage of de­vel­op­ment,” said co-found­ing in­vestor and board mem­ber Li­onel Gold­frank in a state­ment.

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.

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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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.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

FDA ad­comm to de­cide on mol­nupi­ravir EUA; Can­cer at­las un­veils new po­ten­tial drug tar­get

The FDA has another adcomm coming down the pipeline — this time on Covid-19 oral antiviral molnupiravir.

The federal agency’s advisory committee will meet on November 30th to go over Merck and Ridgeback’s EUA request for their investigational antiviral drug, and discuss the available data supporting its use in Covid-19 patients.

This comes two weeks after Merck claimed that their antiviral pill reduced the chance that newly diagnosed Covid-19 patients would be hospitalized or die by 50%. The pharma made the announcement after interim data on 775 patients in their clinical trial showed the antiviral’s potential.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Rahul Singhvi, Resilience CEO

A Bob Nelsen start­up turns to Har­vard to help sharp­en its tech, in­spir­ing first spin­out

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.