FDA ap­proves ex-No­var­tis drug for Cush­ing’s dis­ease

Two months af­ter win­ning Eu­ro­pean ap­proval, a one-time No­var­tis or­phan drug for Cush­ing’s dis­ease has been OK’d by the FDA.

Is­tur­isaknown chem­i­cal­ly as os­ilo­dro­stat, has been ap­proved as a twice-dai­ly pill for those who have not un­der­gone the pi­tu­itary gland surgery of­ten used to treat Cush­ing’s dis­ease, or for those whose symp­toms per­sist af­ter the op­er­a­tion. No­var­tis sold the com­pound along­side two oth­er en­docrine drugs to the Ital­ian phar­ma Recor­dati last Ju­ly for $390 mil­lion, plus Is­tur­isa mile­stones.

A mod­er­ate deal rel­a­tive to No­var­tis’s of­ten high-rolling ta­ble, the terms re­flect­ed the re­al but lim­it­ed mar­ket po­ten­tial for that slice of the com­pa­ny’s en­docrine port­fo­lio. Gen­er­al­ly caused by a non-can­cer­ous tu­mor on the pi­tu­itary gland that caus­es the body to re­lease too much cor­ti­sol, Cush­ing’s af­fects a lit­tle over a mil­lion Amer­i­cans each year. It can cause a range of health prob­lems, in­clud­ing high blood pres­sure, type 2 di­a­betes and change in ap­pear­ance. In ad­di­tion to Is­tur­isa, the deal al­so in­clud­ed al­ready ap­proved Cush­ing’s dis­ease drugs Sig­ni­for and Sig­ni­for LAR.

Most pa­tients are treat­ed with surgery, but not all pa­tients are el­i­gi­ble for surgery, and surgery doesn’t al­ways work. For those pa­tients, doc­tors of­ten pre­scribe drugs that block hu­man growth hor­mone, such as Sig­ni­for, or a drug that blocks an en­zyme in­volved in cor­ti­sol pro­duc­tion, such as metyrapone.

Is­tur­isa works by tar­get­ing a new en­zyme. It blocks 11-be­ta-hy­drox­y­lase, one of the last en­zymes in a chain the body us­es to make cor­ti­sol.

In a Phase III tri­al of 137 adults last year, the drug brought about half of all pa­tients down to nor­mal cor­ti­sol pro­duc­tion dur­ing the 24-week, sin­gle-arm, open-la­bel phase. The 71 pa­tients who tol­er­at­ed and re­spond­ed to the drug were then en­rolled in an 8-week dou­ble-blind phase, test­ing it against place­bo. At the end of the phase, 86% on the drug arm had cor­ti­sol with­in nor­mal ranges, com­pared with 30% of place­bo pa­tients.

Still, the lim­it­ed tri­al left key ques­tions unan­swered, most no­tably how long it will work. Af­ter the En­docrine So­ci­ety meet­ing where the tri­al re­sults were an­nounced, ses­sion co-mod­er­a­tor and as­so­ciate di­rec­tor of the Pi­tu­itary Cen­ter at the Uni­ver­si­ty of Penn­syl­va­nia Ju­lia Khar­lip told Med­scape, “It is like­ly go­ing to work.”

But she cau­tioned that the drug it re­places — metyrapone, which is tak­en four times a day and blocks a dif­fer­ent en­zyme — is known to even­tu­al­ly be­come in­ef­fec­tive as the tu­mor es­sen­tial­ly over­rides the en­zyme block­ade.

Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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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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Noubar Afeyan, Flagship

Step­ping out along­side ARCH's record raise, Flag­ship adds a $1.1B mon­ster fund of its own

ARCH’s unveiling this morning of 2 new funds bulging with $1.5 billion in cash for biotech startups was just the first round of today’s venture news.

Right on its heels we have another monster fund debuting at Flagship Pioneering, another big venture group known for making huge bets on cutting-edge tech — the kind it brews up in its labs.

And this one weighs in at $1.1 billion, which will operate in tandem with the $1.1 billion in funds Flagship rolled out last year.

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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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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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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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Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Mer­ck scores new ad­vance on tu­mor-ag­nos­tic front as Keytru­da beat chemo-based reg­i­mens in a type of col­orec­tal can­cer

Back in 2017, before the term “tumor agnostic” really took hold among cancer drug developers, Merck became the first to secure such an approval for Keytruda as a second-line treatment for patients characterized by a biomarker — rather than where the cancer started in the body. Now it’s looking to break fresh ground with a new slate of data suggesting the drug’s utility in the frontline setting for colorectal cancer.