The com­pas­sion­ate use poll: A lop­sided vote fa­vors post­ing poli­cies, with plen­ty of fret­ting

We ran a snap poll yes­ter­day on com­pas­sion­ate use poli­cies, and there’s clear­ly plen­ty of pos­i­tive sen­ti­ment among our read­ers back­ing some sort of eas­i­ly ac­ces­si­ble, on­line state­ment ex­plain­ing each biotech’s poli­cies on ex­pand­ed ac­cess to ex­per­i­men­tal drugs.

The vote land­ed 70% for, 30% against post­ing a com­pas­sion­ate use pol­i­cy on­line among the 149 votes we col­lect­ed in the last 24 hours. But maybe that has a lot to do with the fact that a very sim­i­lar sized group al­so said that such pol­i­cy state­ments don’t ap­ply to their com­pa­nies. Among the 49 who said it did ap­ply, 33 said their com­pa­nies didn’t post a pol­i­cy, against 16 who said they did.

That two-to-one ra­tio against, though, is still a far cry from the neg­li­gi­ble 4% of small biotechs tracked by Avalere Health which post­ed such poli­cies.

No, said one poll re­spon­dent: “The de­ci­sion to try an ex­per­i­men­tal med­i­cine is very com­plex (sci­en­tif­i­cal­ly, eth­i­cal­ly and reg­u­la­to­ri­ly) and mak­ing it ‘easy’ is go­ing to open a Pan­do­ra’s box that we’re not ready for.”

The ‘no’ camp al­so ex­pressed con­cerns that a sin­gle ad­verse event from com­pas­sion­ate use could de­rail a pro­gram, as well as wor­ries about the cost of pro­vid­ing drugs for small com­pa­nies years away from mak­ing any mon­ey.

A lot of the com­ments for the “yes” vote echoed this sen­ti­ment:

“Post­ing com­pas­sion­ate use poli­cies on your web­site does not promise that your case will be ac­cept­ed, but at least it pro­vides trans­paren­cy on their pol­i­cy and of­fers the po­ten­tial to be treat­ed, ” said one.

Yes, but. “Com­pas­sion­ate Use should on­ly be of­fered for drugs suc­cess­ful­ly shown to be both ef­fi­ca­cious and safe in Phase IIb/III tri­als. Pri­or to that, it is not ad­vis­able. Re­mem­ber “first do no harm,” says an­oth­er.

In­deed, one read­er al­so steered me to Bio­Marin’s pol­i­cy, say­ing that it’s com­mon for such state­ments to re­strict any com­pas­sion­ate use to drugs which have fin­ished Phase III, when you have sol­id ef­fi­ca­cy and safe­ty da­ta, but be­fore the FDA has act­ed.

Art Ca­plan

That’s a nar­row win­dow. NYU’s not­ed bioethi­cist Art Ca­plan tells me he’s seen com­pas­sion­ate use re­quests for drugs in every­thing from an­i­mal-stage test­ing on up. Some small com­pa­nies will start en­ter­tain­ing re­quests as ear­ly as Phase I. And no biotech should ex­pect the pleas to be re­strict­ed for use in the des­ig­nat­ed dis­eases be­ing stud­ied. It could be for a dif­fer­ent dose as well as a sep­a­rate in­di­ca­tion, which is what caught Chimerix in the mid­dle of an on­line mob as the par­ents of one young boy sought ac­cess for its lead ther­a­py through a pub­lic lob­by­ing ef­fort that sud­den­ly went vi­ral.

Iron­i­cal­ly, I checked out Chimerix’s web site and couldn’t find any men­tion of a com­pas­sion­ate use pol­i­cy. The biotech, which has ex­pe­ri­enced a cou­ple of set­backs with the pro­gram, de­clined com­ment.

Any time you get press cov­er­age of a new drug, Ca­plan tells me, you raise the chances of com­pas­sion­ate use re­quests. And Ca­plan doesn’t sign on with the post-Phase III on­ly camp. The im­por­tant thing is mak­ing sure the drug has a well-es­tab­lished safe­ty pro­file and at least a hint of ef­fi­ca­cy. For Ca­plan, Phase IIb is where you hit the sweet spot for con­sid­er­ing these re­quests.

And he adds that he wasn’t in the least bit sur­prised to hear that few biotechs have clear­ly enun­ci­at­ed poli­cies spelled out on­line.

“If you have a com­pa­ny with one CEO and 20 peo­ple chained to a bench, web­site de­vel­op­ment is not a top pri­or­i­ty,” says Ca­plan. But that doesn’t mean they shouldn’t do it.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,300+ biopharma pros reading Endpoints daily — and it's free.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,300+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Mike Grey. Mirum

In $86M IPO pitch, Mirum spells out plans to turn Shire dis­cards in­to or­phan liv­er drug suc­cess­es

Mike Grey doesn’t have any time to waste. Hav­ing re­gained con­trol of two liv­er dis­ease drugs from Shire and po­si­tioned them for piv­otal stud­ies — five years af­ter first hand­ing them off in a deal to sell Lu­me­na, where he was CEO — Grey is steer­ing Mirum straight in­to an IPO with a $86 mil­lion ask.

Not that Mirum has spent much of its $120 mil­lion Se­ries A cash since launch­ing last No­vem­ber. Ac­cord­ing to the S-1, the Cal­i­forn­ian biotech has burned through $23.3 mil­lion as of March, but ex­pects ex­pens­es to pick up once their clin­i­cal work gath­ers steam.