A fa­ther's plea: The FDA needs to make sure that Juno's lethal dis­as­ter is­n't re­peat­ed

The FDA nev­er has pub­licly ex­plained just why it de­cid­ed to lift a clin­i­cal hold on Juno’s lead CAR-T drug JCAR015 just days af­ter the stun­ning news that the tri­al was be­ing halt­ed af­ter three pa­tients tak­ing the cell ther­a­py died of a cere­bral ede­ma. But then, it nev­er does, cit­ing se­cre­cy rules that fre­quent­ly keep reg­u­la­tors silent.

Now the fa­ther of one of those vic­tims has joined in a pe­ti­tion from the Cen­ter for Re­spon­si­ble Sci­ence seek­ing some re­al trans­paren­cy at the FDA on the way it han­dles pa­tient deaths — with ma­jor im­pli­ca­tions for the peo­ple who par­tic­i­pate in clin­i­cal tri­als.

Al­most a year af­ter Michael Vokhgelt’s 24-year-old son Max died in Juno’s ROCK­ET study, and five months af­ter telling STAT that “he died for greed,” Vokhgelt is still look­ing for an­swers. Max didn’t die from leukemia, says his fa­ther. He was killed by the drug, which has since been scrapped by Juno.

Not on­ly did Juno not an­nounce Max Vokhgelt’s death, a week lat­er it put out a re­lease cit­ing the “en­cour­ag­ing” and “im­pres­sive” re­sults it was see­ing.

In the fa­ther’s words:

It wasn’t un­til two more tri­al vol­un­teers died and FDA is­sued a clin­i­cal hold that on Ju­ly 7, 2016, Juno an­nounced the deaths. Juno blamed the deaths on flu­dara­bine, a chemother­a­py pre­con­di­tion­ing treat­ment in con­junc­tion with the CAR-T ther­a­py. FDA ac­cept­ed Juno’s ex­pla­na­tion and al­lowed Juno to re­sume the tri­al with­out Flu­dara­bine. I was dev­as­tat­ed when I learned that two more tri­al par­tic­i­pants died in No­vem­ber from cere­bral ede­ma. How can this hap­pen? I don’t even know the ra­tio­nale be­hind FDA’s de­ci­sion to lift the clin­i­cal hold af­ter my son and two oth­ers died, be­cause that is con­sid­ered “pro­pri­etary”.

I have read the Cit­i­zen Pe­ti­tion from the Cen­ter for Re­spon­si­ble Sci­ence and sup­port the re­quest­ed reg­u­la­to­ry amend­ments to al­low for the use of the pre­clin­i­cal test that is best to pre­dict what will hap­pen to clin­i­cal tri­al par­tic­i­pants. If tra­di­tion­al tests don’t al­ways pre­dict dead­ly tox­i­c­i­ties, drug spon­sors must be al­lowed to use more pre­dic­tive tests that bet­ter pre­dict what hap­pens in hu­mans.

It is FDA’s re­spon­si­bil­i­ty to pro­tect hu­man health and pro­tect the pub­lic from dan­ger­ous drugs. To achieve that man­date, all avail­able tools to pre­dict safe­ty must be used. I don’t want an­oth­er fam­i­ly to go through what my fam­i­ly went through.

In a let­ter sent last April, and first re­port­ed by RAPS’ Zachary Bren­nan, the Cen­ter for Re­spon­si­ble Sci­ence not­ed 19 treat­ment-re­lat­ed deaths in clin­i­cal tri­als from Ju­ly 2016 through April 2017.  Three of those were in a study of a ri­val CAR-T by Kite, which re­cent­ly was hit with the death of its first pa­tient from a case of cere­bral ede­ma.

The CRS wants things to change be­fore more pa­tients die.

“We ar­gue that every­thing that can be done to pro­tect clin­i­cal tri­al vol­un­teers must be done. Rather than re­sume a tri­al with­out know­ing the ac­tu­al cause of the dead­ly tox­i­c­i­ty, spon­sors should have made the drug avail­able so that it could be test­ed in a hu­man-rel­e­vant plat­form.”

The FDA says it’s still un­der re­view. Let’s hope they do bet­ter by tri­al vol­un­teers.

UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid 19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $200 million financing illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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Dai­ichi Sankyo sinks $200M in­to new gene ther­a­py tech from Ul­tragenyx

In a leap to the gene therapy space, Daiichi Sankyo has dropped $200 million to access Ultragenyx’s manufacturing technology, providing the rare disease biotech with plenty of cash and a stock boost amid a general cash crunch.

For $125 million in cash and a $75 million equity investment, Daiichi Sankyo has bought a non-exclusive license to the IP around two platforms with which it plans to develop AAV-based gene therapy products. The Japanese pharma is purchasing the stock $RARE at $60 per share, more than a third above its current price of $44.43.

It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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