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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Pfiz­er and Sang­amo re-open PhI­II of he­mo­phil­ia A gene ther­a­py; AEON head­ed to PhI­II

A year after voluntarily pausing the Phase III study of their hemophilia A gene therapy program, and four months after FDA lifted the clinical hold, Pfizer and Sangamo Therapeutics say they will resume dosing next month.

The move pushes back the original data readout by two years. At the time of the first patient dosing in October 2020, a Pfizer spokesperson had told Endpoints News that a pivotal readout would come in early 2022. Following the hurdles caused by the clinical hold, Pfizer said Friday morning the new anticipated timeline is the first half of 2024.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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