Scott Requadt (Talaris)

Ta­laris Ther­a­peu­tics bags an­oth­er $115M to curb im­muno­sup­pres­sion in or­gan trans­plant pa­tients

Just over a year af­ter se­cur­ing a $100 mil­lion Se­ries A round, Ta­laris Ther­a­peu­tics is back with a $115 mil­lion Se­ries B to fu­el its mis­sion of mak­ing or­gan trans­plants safer.

CEO Scott Re­quadt said he’s seen “dra­mat­ic progress” since the Se­ries A. In the last 18 months, the biotech has crossed the Phase III start­ing line with its lead drug, FCR001, in liv­ing donor kid­ney trans­plant re­cip­i­ents. Plus, it “laid the ground­work” for two Phase II stud­ies, he not­ed.

Trans­plant pa­tients are of­ten put on im­muno­sup­pres­sion reg­i­mens to pre­vent re­jec­tion of the do­nat­ed or­gan, which ex­pos­es them to in­fec­tions.

“Covid has changed every­thing for all of us. But in par­tic­u­lar, it’s a per­fect storm for or­gan trans­plant re­cip­i­ents be­cause they’re on chron­ic im­muno­sup­pres­sion,” Re­quadt said. The goal, he added, is to re­store im­mune tol­er­ance with­out the body re­ject­ing the or­gan.

Suzanne Ild­stad

To do so, the com­pa­ny’s pro­cess­ing bone mar­row. The ap­proach is the brain­child of Suzanne Ild­stad, di­rec­tor of the Uni­ver­si­ty of Louisville’s In­sti­tute for Cel­lu­lar Ther­a­peu­tics, who launched Re­generex in 2002 to build out the idea. No­var­tis helped up­grade man­u­fac­tur­ing and shape a Phase II pro­to­col be­fore shut­ting down its cell and gene ther­a­pies unit in 2016.

When fund­ing ran dry, Ild­stad turned to Clarus (now Black­stone Life Sci­ences), which led a $100 mil­lion Se­ries A to pro­pel her work to Phase III. Re­quadt, who spent 13 years as Clarus’ man­ag­ing di­rec­tor, be­came CEO of the nom­i­nal­ly new com­pa­ny, Ta­laris.

“It was one of the most unique things that I’ve seen as an in­vestor in 13 years,” he told End­points News. “And so I de­cid­ed to put up my hands and be­come a first-time CEO.”

Re­quadt plans on dump­ing Ta­laris’ re­cent Se­ries B in­to its on­go­ing Phase III tri­al in liv­ing donor re­cip­i­ents, as well as prospec­tive Phase II tri­als in dif­fuse sys­temic scle­ro­sis and pa­tients who have pre­vi­ous­ly re­ceived a liv­ing donor kid­ney trans­plant. The Phase III will en­roll up to 120 donor-re­cip­i­ent pairs at 15 sites in the US, Re­quadt said. He de­clined to com­ment on the time­line of the tri­al, which is still in ear­ly stages. Both Phase II tri­als are ex­pect­ed to be­gin next year.

In a Phase II study, 70% of 37 liv­ing donor kid­ney trans­plant pa­tients giv­en FCR001 were weaned off all of their im­muno­sup­pres­sion treat­ments, ac­cord­ing to Ta­laris. And out of 7 pa­tients with a pre-ex­ist­ing au­toim­mune dis­ease who quit im­muno­sup­pres­sion, none had a re­cur­rence, ac­cord­ing to the com­pa­ny.

In the up­com­ing Phase II de­layed tol­er­ance tri­al, re­searchers will test whether they can re­cruit pa­tients who re­ceived trans­plants in the last 12 months, take some cells from their donors, and make a prod­uct to in­duce im­mune tol­er­ance.

“Ini­tial­ly, we’re go­ing to fo­cus on pa­tients who are with­in 12 months of a trans­plant, but we may well ex­plore… longer de­lays down the line,” Re­quadt said.

The Se­ries B was led by Sur­vey­or Cap­i­tal and Viking Glob­al In­vestors. Black­stone chipped in again this round, as well as Lon­gi­tude Cap­i­tal and Qim­ing Ven­ture Part­ners USA. Oth­er new in­vestors in­clude Cor­morant As­set Man­age­ment, In­vus, funds and ac­counts man­aged by Black­Rock, Even­tide As­set Man­age­ment, Lo­gos Cap­i­tal, Ais­ling Cap­i­tal and Pamo­ja Cap­i­tal.

“Im­muno­sup­pres­sive agents are tox­ic to the kid­ney, and as a re­sult you typ­i­cal­ly need to have kid­ney trans­plant every 15 years or so. And if we’re able to dis­con­tin­ue the drugs that are tox­ic to the kid­ney, you know, the po­ten­tial is that peo­ple would ben­e­fit from a sin­gle trans­plant for much longer pe­ri­ods of time,” Re­quadt said. “Maybe even one kid­ney for life.”

Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.