Af­ter Kite Phar­ma stuns an­a­lysts with news of a death in lead CAR-T study, safe­ty ques­tions sour launch fore­cast

There may nev­er have been a right way for Kite Phar­ma to an­nounce that a pa­tient in one of its CAR-T stud­ies died from cere­bral ede­ma — but wait­ing more than a week and then bring­ing it up on the quar­ter­ly call with an­a­lysts def­i­nite­ly wasn’t the way to go. That route seems to have on­ly bright­ened the glar­ing spot­light that im­me­di­ate­ly swiveled over to com­pa­ny ex­ecs as they tried to ex­plain, and down­play, the death.

David Nieren­garten, Wed­bush an­a­lyst

To­day, in a note to in­vestors, Wed­bush an­a­lyst David Nieren­garten high­light­ed the mine field that Kite — which saw its shares plunge 13% yes­ter­day — is try­ing to nav­i­gate on this top­ic.

While Kite CMO David Chang re­peat­ed­ly em­pha­sized in the call just how sick this pa­tient was be­fore he died, Nieren­garten sees some dis­turb­ing sim­i­lar­i­ties with Juno’s old lead drug, JCAR015, which im­plod­ed in Phase III af­ter it killed 5 peo­ple in two dif­fer­ent groups — be­fore and af­ter a clin­i­cal hold by the FDA.

In his note to in­vestors this morn­ing, Nieren­garten said:

We al­so see the death as ad­di­tion­al ev­i­dence against the use of CD28 co-stim­u­la­to­ry do­mains in CAR-T de­signs — re­call, JUNO scrapped its CD28-con­tain­ing CAR-T JCAR015 pro­gram that caused mul­ti­ple fa­tal­i­ties due to cere­bral ede­ma in the ROCK­ET study of adult ALL pa­tients. We see high risk for safe­ty prob­lems in axi-cel’s on­go­ing stud­ies in adult and pe­di­atric ALL pa­tients, as well as for pipeline CAR-Ts KITE-585 (BC­MA) and KITE-796 (CLL-1/CLEC12A) that both uti­lize a CD28 co-stim­u­la­to­ry do­main.

David Chang, Kite

While Kite ex­ecs, led by CEO Arie Bellde­grun, want to fo­cus on near-term op­por­tu­ni­ties for a lead CAR-T that’s neck-and-neck with No­var­tis in seek­ing a pi­o­neer­ing FDA OK, Nieren­garten sees in­creased risk — even with an ex­pect­ed ap­proval. And ri­vals may well com­pli­cate the pic­ture even more at the up­com­ing AS­CO meet­ing.

“We see adop­tion as be­ing lim­it­ed by physi­cian aware­ness and com­fort re­gard­ing risk/ben­e­fit pro­file,” notes Nieren­garten. “We see lit­tle push­back from pay­ers, giv­en the lim­it­ed ini­tial pa­tient pop­u­la­tion and one-time na­ture of the treat­ment, and as­sume the price re­ceived by KITE from each pro­ce­dure to be $325k. We await da­ta from Juno’s (JUNO, NEU­TRAL) JCAR017 at AS­CO, as well as NVS’s CAR-T CTL019 Juli­et study at ICML in June to gain fur­ther in­sight in­to mar­ket dy­nam­ics go­ing for­ward.”

Arie Bellde­grun

Juno nev­er did ful­ly ex­plain just what went wrong with JCAR015. It’s first ex­pla­na­tion, try­ing to pin the blame on flu­dara­bine, was clear­ly off base. Long time ob­servers nev­er thought that was cred­i­ble. And Juno wound up shelv­ing the whole thing and go­ing the next-wave ap­proach with JCAR017.

It didn’t help that two top Kite ex­ecs, in­clud­ing Chang, cashed in some stock op­tions be­tween the time they learned of the death and the time it was an­nounced. Those sales may have been pro­grammed in well ahead of time, but the op­tics are al­ways bad.

Kite now has lit­tle room for er­ror. The risk of an­oth­er lethal cere­bral ede­ma case pop­ping up is cre­at­ing doubts just when Kite needs to ex­ude con­fi­dence and cer­tain­ty.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.

Per­cep­tive fields SPAC #3 as an­oth­er group of biotechs scoops up $364M in lat­est Nas­daq romp

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

Alex­ion cre­ates new post for chief di­ver­si­ty of­fi­cer; Bar­ry Greene step­ping down at Al­ny­lam, Yvonne Green­street named as suc­ces­sor

Alexion has carved out a new position for chief diversity officer and filled it with an inside promotion.

Uzair Qadeer will now be responsible for their “diversity, inclusion and belonging” strategy, looking to reshape the biotech’s corporate culture. A veteran of Deloitte and Bristol Myers Squibb, Qadeer was working on executive coaching and helping create the diversity program he now leads.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,700+ biopharma pros reading Endpoints daily — and it's free.

President Trump (AP Images)

UP­DAT­ED: FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as phar­ma warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.