Av­enue Ther­a­peu­tics lands an FDA ad­comm for trou­bled flag­ship drug; FDA ap­proves eye in­jec­tion treat­ment for flu­id buildup

In­tra­venous tra­madol-fo­cused Av­enue Ther­a­peu­tics may not be out of the game just yet.

Af­ter re­ceiv­ing two CRLs for their drug, Av­enue an­nounced to­day that it has re­ceived a writ­ten in­ter­im re­sponse from the FDA’s Of­fice of New Drugs that reg­u­la­tors will hold an ad­comm to reach a de­ci­sion on a for­mal dis­pute res­o­lu­tion re­quest filed by Av­enue back in Ju­ly.

This oc­curred af­ter the FDA sent Av­enue the sec­ond CRL for their drug, an in­tra­venous form of syn­thet­ic opi­oid tra­madol.

The biotech said back in June that the FDA had de­nied its ap­pli­ca­tion as the “de­layed and un­pre­dictable on­set” of pain re­duc­tion from tra­madol didn’t sug­gest a ben­e­fit as a monother­a­py. And at the time, there was not enough in­for­ma­tion to know whether it worked in com­bi­na­tion with oth­er ther­a­pies.

Av­enue dis­agreed at the time, and said they would keep push­ing for reg­u­la­to­ry ap­proval.

The FDA hasn’t yet an­nounced when the ad­comm will con­vene.  The OND will re­spond to the FDRR with­in 30 days af­ter meet­ing.

FDA ap­proves Xipere for flu­id buildup in reti­na

Bausch + Lomb and Clear­side Bio­med­ical an­nounced this morn­ing that the FDA ap­proved Xipere, a tri­am­ci­nolone ace­tonide in­jectable sus­pen­sion, for supra­choroidal use to treat mac­u­lar ede­ma as­so­ci­at­ed with uveitis, a form of eye in­flam­ma­tion.

Mac­u­lar ede­ma is the buildup of flu­id in the mac­u­la, lo­cat­ed in the reti­na at the back of the eye. This buildup can cause reti­nal swelling and dis­tort­ed vi­sion — and can lead to per­ma­nent vi­sion loss if left un­treat­ed, ac­cord­ing to Bausch + Lomb.

The method of choice is unique. The treat­ment us­es a mi­croin­jec­tor — de­vel­oped by Clear­side — to in­ject Xipere in­to the supra­choroidal space of the eye. That space, which is be­tween the scle­ra and choroid, may turn out to be a more ef­fec­tive method to im­prove tar­get­ed drug de­liv­ery.

Ac­cord­ing to Bausch Health chair­man and CEO Joseph Pa­pa, the com­pa­ny ex­pects to make Xipere avail­able in Q1 of next year.

“With this ap­proval, we be­gin a new era in de­liv­er­ing ther­a­pies to the back of the eye,” said Clear­side’s pres­i­dent and CEO George Lasezkay. “Xipere is the first com­mer­cial prod­uct de­vel­oped by Clear­side, the first prod­uct ap­proved for in­jec­tion in­to the supra­choroidal space and the first ther­a­py ap­proved for mac­u­lar ede­ma as­so­ci­at­ed with uveitis.”

Cedil­la Ther­a­peu­tics in­creas­es Se­ries B size with fresh $25 mil­lion

Third-Rock backed Cedil­la Ther­a­peu­tics ex­pand­ed its Se­ries B from last year with an ad­di­tion­al $25 mil­lion, bring­ing the to­tal amount raised to $82.6 mil­lion.

The Se­ries B ex­pan­sion an­nounced to­day in­cludes new in­vestors RA Cap­i­tal Man­age­ment, Janus Hen­der­son In­vestors, Wood­line Part­ners and Lo­gos Cap­i­tal, along­side Third Rock.

The funds will sup­port Cedil­la’s con­tin­u­ing de­vel­op­ment of its two lead pro­grams: a TEAD in­hibitor for sol­id tu­mor treat­ment such as mesothe­lioma and cer­tain squa­mous cell car­ci­no­mas, along with a high­ly se­lec­tive CDK2/Cy­clin E in­hibitor for mul­ti­ple tu­mor types, in­clud­ing CDK4/6-re­sis­tant breast can­cer.

The com­pa­ny plans to ini­ti­ate IND-en­abling stud­ies for its TEAD pro­gram by next June and for the CDK2 pro­gram by the end of 2022.

“With this fi­nanc­ing, we plan to ac­cel­er­ate and ex­pand our de­vel­op­ment ef­forts by pro­gress­ing our most ad­vanced pro­grams to­ward the clin­ic while con­tin­u­ing to in­vest in on­go­ing dis­cov­ery ef­forts against ad­di­tion­al high val­ue can­cer tar­gets,” said Cedil­la pres­i­dent and CEO San­dra Glucks­mann.

Jake Sim­son, a part­ner with RA Cap­i­tal Man­age­ment, will join Cedil­la’s Board of Di­rec­tors as part of the fi­nanc­ing.

Ju­ven­tas Cell Ther­a­py rais­es $63 mil­lion in Se­ries C

Chi­nese biotech Ju­ven­tas Cell Ther­a­py com­plet­ed a Se­ries C fi­nanc­ing round, bring­ing in close to $63 mil­lion.

Ac­cord­ing to a state­ment, Ju­ven­tas will use the funds to pur­sue an NDA in Chi­na and launch im­ple­men­ta­tion of a com­mer­cial­iza­tion and in­ter­na­tion­al re­search and de­vel­op­ment plan for part­ner CASI’s CAR-T treat­ment CNCT19.

CASI shares glob­al co-com­mer­cial and prof­it-shar­ing rights with Ju­ven­tas, and in­di­rect­ly owns just over 12% of the com­pa­ny’s shares fol­low­ing com­ple­tion of the Se­ries C round.

Ju­ven­tas’ fi­nanc­ing was led by CI­CC Cap­i­tal.

“The col­lab­o­ra­tion be­tween CASI and Ju­ven­tas will speed up the adop­tion of CAR-T ther­a­py in Chi­na,” CASI CEO and chair­man Wei-Wu He said 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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