Kevin Boyle, Kuur

Months af­ter a ma­jor re­brand­ing around CAR-NKT cells, Ku­ur Ther­a­peu­tics is of­fer­ing a peek at ear­ly re­sults

Sev­er­al months af­ter a name change and ma­jor re­struc­tur­ing, Ku­ur Ther­a­peu­tics has of­fered an ear­ly glimpse at re­sults from a small num­ber of pa­tients treat­ed with their CAR-NKT cell ther­a­pies de­vel­oped with the Bay­lor Col­lege of Med­i­cine.

Out of 10 evalu­able neu­rob­las­toma pa­tients dosed with Ku­ur’s lead can­di­date, an au­tol­o­gous GD2-tar­get­ing CAR-NKT ther­a­py, re­searchers not­ed one com­plete re­sponse, one par­tial re­sponse, and three pa­tients with sta­ble dis­ease, ac­cord­ing to the Hous­ton-based biotech.

The pa­tient with a com­plete re­sponse had pre­vi­ous­ly achieved a par­tial re­sponse be­fore re­ceiv­ing a sub­se­quent dose of the drug. The drug ap­peared safe, ac­cord­ing to Ku­ur, with one case of Grade 2 cy­tokine re­lease syn­drome (CRS) oc­cur­ring in the Phase I tri­al, dubbed GI­NAKIT2.

“We have some very good ear­ly signs of NKT cells be­ing safe, hom­ing to the site of tu­mor, which is very im­por­tant go­ing af­ter sol­id tu­mors … and now we’re show­ing signs of ef­fi­ca­cy as well,” CEO Kevin Boyle told End­points News. “So, based on that, we want the com­pa­ny to be fo­cused on just this plat­form.”

The drug con­sists of nat­ur­al killer T cells en­gi­neered with a CAR tar­get­ing GD2, a ma­jor gan­glio­side ex­pressed on many neu­rob­las­toma tu­mors. It’s de­signed to se­crete IL-15, which has been shown to bol­ster the ef­fi­ca­cy and per­sis­tence of CAR-NKT cells in the im­muno­sup­pres­sive tu­mor mi­croen­vi­ron­ment.

NKT cells are rare lym­pho­cytes that aren’t fre­quent­ly found in the pe­riph­er­al blood, and as a re­sult, re­searchers tend not to work with them, Boyle ex­plained. The sci­en­tist lead­ing Bay­lor’s re­la­tion­ship with Ku­ur has been work­ing with the cells for two decades, he said.

Kurt Gunter

“One rea­son that we like this cell type is it homes nat­u­ral­ly to the tis­sue in sol­id tu­mors… It al­so has an in­vari­ant T-cell re­cep­tor… and that means it’s the same in every­one so it won’t cause graft-ver­sus-host dis­ease when we use it in an­oth­er per­son as off-the-shelf ther­a­py. And that means we don’t need to gene ed­it the TCR and that puts us in a fun­da­men­tal­ly dif­fer­ent po­si­tion from the peo­ple work­ing with CAR-T cells,” CMO Kurt Gunter said.

Ku­ur’s sec­ond can­di­date, an off-the-shelf CD19-di­rect­ed CAR-NKT ther­a­py, achieved a PR and CR in the two pa­tients with hema­to­log­i­cal ma­lig­nan­cies who were evalu­able in the Phase I study, AN­CHOR. The re­searchers saw no CRS cas­es, and no ev­i­dence of graft ver­sus host dis­ease (GvHD) — a con­di­tion where donor cells at­tack their host, the com­pa­ny said.

Boyle, who took the helm last March dur­ing a ma­jor re­struc­tur­ing, ex­pects to read out full da­ta from both the Phase I stud­ies next year. And if all goes well, he’s plan­ning to launch a Phase II ex­pan­sion tri­al of the AN­CHOR study in 2021.

Un­til last March, Ku­ur went by the name Cell Med­ica. The Lon­don-based up­start had lo­ca­tions across two con­ti­nents and three coun­tries, Boyle said. But in­stead of work­ing on a va­ri­ety of cell ther­a­pies, the com­pa­ny chose to laser-fo­cus on the CAR-NKT pro­gram un­der an ex­clu­sive li­cense with Bay­lor. It set­tled on one lo­ca­tion in Hous­ton, right next door to their col­lab­o­ra­tors, and for­mer CEO Chris Now­ers was re­placed by Boyle, who had pre­vi­ous­ly served as CFO.

The com­pa­ny hopes to close a pri­vate fi­nanc­ing round this quar­ter, Boyle hint­ed, some of which will be used to trans­form the sin­gle-cen­ter stud­ies in­to mul­ti-cen­ter stud­ies.

“The sit­u­a­tion with neu­rob­las­toma is still kind of bleak es­pe­cial­ly for these high risk re­lapsed re­frac­to­ry pa­tients,” Gunter said. “They have gen­er­al sur­vivals of less than one year, once they’ve re­lapsed,” he con­tin­ued, adding that he be­lieves the com­pa­ny can bring a sig­nif­i­cant ben­e­fit to pa­tients.

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.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.