T cell ex­perts at Kiadis bag Cy­toSen's NK cell tech, plot­ting a dual can­cer killing ap­proach with tips from Carl June

In an ef­fort to mar­ry T cell and nat­ur­al killer cell tech­nolo­gies for the treat­ment of blood can­cer, Dutch biotech Kiadis has inked an all-stock deal to ac­quire US-based Cy­toSen Ther­a­peu­tics.

Arthur Lahr

Both com­pa­nies have had the same goal: to boost the chances of can­cer pa­tients un­der­go­ing hematopoi­et­ic stem cell trans­plants by su­per­charg­ing their im­mune sys­tem while their bone mar­row re­cov­ers from the pro­ce­dure. Kiadis does this with en­gi­neered T cells; Cy­toSen chose NK cells, de­signed to at­tack any re­main­ing ma­lig­nant cells that could lead to a re­lapse. Both tech­nolo­gies take cells from a fam­i­ly mem­ber of the pa­tient so that the im­mune cells would be par­tial­ly matched.

Aside from a new tech plat­form, Kiadis al­so scored a net­work of promi­nent col­lab­o­ra­tors on Cy­toSen’s found­ing team, in­clud­ing Dean Lee of Na­tion­wide Chil­dren’s Hos­pi­tal, Ste­fan Ciurea of the MD An­der­son Can­cer Cen­ter and Robert Igarashi from the Uni­ver­si­ty of Cen­tral Flori­da. Carl June, the pi­o­neer­ing CAR-T ex­pert, is al­so join­ing its sci­en­tif­ic ad­vi­so­ry board.

UCF first came up with the Cy­toSen method, which de­rives nanopar­ti­cles from anti­gen pre­sent­ing cells to stim­u­late pro­lif­er­a­tion of NK cells. Na­tion­wide fur­ther re­fined it, and the first hu­man stud­ies were con­duct­ed at MD An­der­son.

Carl June

The com­pa­ny, which is list­ed on Eu­ronext Am­s­ter­dam and Brus­sels ($KDS) now ex­pects to take Cy­toSen’s lead pro­gram, CS­DT002-NK, in­to the clin­ic in 2020, tar­get­ing high-risk acute myeloid leukemia pa­tients. Ear­li­er proof-of-con­cept da­ta from 25 pa­tients had demon­strat­ed a re­lapse rate of 8% and pro­gres­sion-free sur­vival of 66%, ac­cord­ing to a press re­lease.

Mean­while, Kiadis’ ATIR101 is un­der re­view in Eu­rope for a mar­ket­ing ap­proval, with a po­ten­tial launch planned for this year.

“The ATIR T-cell and CS­DT002-NK-cell pro­grams each have the po­ten­tial to make trans­plants safer and more ef­fec­tive,” CEO Arthur Lahr said. “In com­bi­na­tion, they have the po­ten­tial to rev­o­lu­tion­ize HSCT, mak­ing it suit­able for an even wider group of pa­tients.”

Per the deal, Cy­toSen in­vestors — who have re­mained un­named — will gain a 7.4% stake in Kiadis, or 1.94 mil­lion shares. They are el­i­gi­ble to re­ceive up to 5.82 mil­lion more shares should de­vel­op­ment and reg­u­la­to­ry mile­stones be reached.

The com­bined en­ti­ty will have $55.4 mil­lion (€ 49 mil­lion) from Kiadis and $6 mil­lion from Cy­toSen to work with.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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New play­ers are jump­ing in­to an old vac­cine game as pan­dem­ic pan­ic spreads fast — putting their tech to the test

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Roche cracks Chi­na's ADC mar­ket open as Kad­cy­la scores its first breast can­cer OK in the coun­try

Roche’s Kadcyla has become the first antibody-drug conjugate to enter the Chinese market, marking a dramatic advance for both the Swiss pharma giant and the therapeutic class.

The local arm of Roche announced the approval late Tuesday, which covers the therapy’s use in the adjuvant setting in patients with early HER-2 positive breast cancer who still have residual invasive disease after receiving paclitaxel and Herceptin as neoadjuvant treatment.

Pascal Soriot, Getty

Pas­cal So­ri­ot and As­traZeneca com­mit to car­bon neu­tral­i­ty by 2025. Where's the rest of Phar­ma?

Pascal Soriot has spent more than 20 years at the top of an industry recently found to emit more carbon than the automotive industry.

He called himself a “global citizen,” and traveled often across three-plus continents. While CEO of AstraZeneca, he commissioned a flight service — media-dubbed AstraZeneca airlines — from Cambridge to the company’s other European hub in Gothenburg. He made few, if any, public statements on the environment or his companies’ impact on it.

Fresh tri­al da­ta for­ti­fy po­si­tion of Roche's oral ther­a­py in spinal mus­cu­lar at­ro­phy bat­tle­ground

With an FDA decision date looming, Roche on Wednesday unveiled positive pivotal data on its blockbuster-bound oral spinal muscular atrophy (SMA) drug in patients with the most severe form of the muscle-wasting disease.

The FDA is set to make its decision on the therapy, risdiplam, by May 24. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.

Partnered with PTC Therapeutics, the Roche drug was tested in 41 patients aged 1-7 months with type 1 SMA, a rare genetic muscle-wasting disease. The trial, dubbed FIREFISH, measured efficacy via the proportion of infants sitting without support after 12 months of treatment, and longer.

Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.