Van­da's PhII da­ta of­fer hope for gas­tro­pare­sis pa­tients who have seen no new treat­ments in decades

As one of the hand­ful of drugs-in-de­vel­op­ment for gas­tro­pare­sis, Van­da Phar­ma­ceu­ti­cals’ $VN­DA tradip­i­tant has set it­self apart for the con­di­tion that af­fects about 6 mil­lion in the Unit­ed States and has not seen an ap­proval in near­ly four decades. On Mon­day, the drug­mak­er re­leased mid-stage da­ta that im­pressed, show­ing the drug con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in nau­sea symp­toms and nau­sea-free days.

Gas­tro­pare­sis is a chron­ic con­di­tion with few treat­ment op­tions that af­fects the nor­mal spon­ta­neous move­ment of the mus­cles in the stom­ach, which usu­al­ly pro­pel food through the di­ges­tive tract. In pa­tients af­flict­ed with the con­di­tion, these mus­cles work poor­ly or not at all, de­lay­ing or pre­vent­ing the stom­ach from emp­ty­ing, caus­ing nau­sea, vom­it­ing and is­sues with blood sug­ar lev­els and nu­tri­tion. The eti­ol­o­gy of gas­tro­pare­sis is un­clear, but the con­di­tion some­times presents as a com­pli­ca­tion of di­a­betes or surgery. The one FDA-ap­proved drug specif­i­cal­ly sanc­tioned for use in gas­tro­pare­sis is meto­clo­pramide, which car­ries a black box warn­ing that cau­tions against pre­scrib­ing for longer than 12 weeks.

Van­da’s drug, a neu­rokinin-1 re­cep­tor (NK-1R) an­tag­o­nist that was li­censed from Lil­ly $LLY in 2012, was test­ed against a place­bo in id­io­path­ic and di­a­bet­ic gas­tro­pare­sis pa­tients in the month long Phase II study. The drug met the main goal, as well as a num­ber of key sec­ondary end­points, and was well tol­er­at­ed with a safe­ty pro­file sim­i­lar to that of the place­bo.

“Tradip­i­tant could of­fer a much more tol­er­a­ble, chron­ic treat­ment, and giv­en the pauci­ty of pro­grams in de­vel­op­ment for this in­di­ca­tion (we count ~4 oth­ers), we be­lieve this as­set could gar­ner in­ter­est form po­ten­tial part­ners. We think this point will be­come a de­bate among in­vestors on whether VN­DA should part­ner this pro­gram based on the Phase 2 da­ta or pro­ceed in­to Phase 3, with the lat­ter be­ing the strat­e­gy cur­rent­ly be­ing pur­sued by the com­pa­ny,” Stifel an­a­lysts wrote in a note.

The tri­al hit the pri­ma­ry end­point of nau­sea im­prove­ment as mea­sured by pa­tient di­aries that rat­ed their symp­toms on a 0-5 scale. Pa­tients on tradip­i­tant re­port­ed a drop of 1.2 ver­sus a fall of 0.7 on place­bo (p=0.0099). For nau­sea-free days, an­oth­er crit­i­cal end­point, tradip­i­tant brought about a rough­ly 29% in­crease, com­pared to about 15% for pa­tients re­ceiv­ing place­bo. Im­prove­ments were al­so seen in most of the core gas­tro­pare­sis symp­toms in­clud­ing vom­it­ing, bloat­ing, and full­ness af­ter meals — most ef­fects were ap­par­ent by the sec­ond week of treat­ment al­though im­prove­ments con­tin­ued through the fourth and last week of treat­ment in the tradip­i­tant group, the com­pa­ny re­port­ed.

“Based on our pre­vi­ous KOL checks, a ≥1 im­prove­ment on nau­sea and ≥20% im­prove­ment on nau­sea free days was viewed as clin­i­cal­ly mean­ing­ful, and we be­lieve tradip­i­tant has met this hur­dle in the more se­vere pa­tients, which made up the ma­jor­i­ty of the study pop­u­la­tion. Im­por­tant­ly, there were im­prove­ments/no wors­en­ing on the oth­er core symp­toms of gas­tro­pare­sis, which is im­por­tant from an FDA per­spec­tive based on the agency’s 2015 draft guid­ance,” Stifel an­a­lysts added.

Tradip­i­tant is ex­pect­ed to rake in peak sales of 898 mil­lion, es­ti­mat­ed Jef­feries an­a­lysts. Mean­while, an­a­lysts at Stifel said they had raised their peak sales ex­pec­ta­tions from $535 mil­lion to $800 mil­lion, not­ing that their up­dat­ed fore­cast could still be con­ser­v­a­tive, “giv­en the size of the mar­ket and the fact over ~3-4 mil­lion scripts are writ­ten each year for meto­clo­pramide”.

The drug is cur­rent­ly al­so be­ing test­ed in a late-stage study in atopic der­mati­tis as­so­ci­at­ed chron­ic pru­ri­tus, af­ter a mid-stage study yield­ed mixed da­ta in the con­di­tion.

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.

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.

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.

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.

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:

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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.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

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.

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