Rigel’s 2nd PhI­II for fos­ta­ma­tinib scut­tled by a sin­gle place­bo re­sponse, shares crater

Rigel Phar­ma­ceu­ti­cals con­tin­ues to be plagued by a low re­sponse rate in its big Phase III pro­gram for fos­ta­ma­tinib. But un­like its first late-stage da­ta drop, which scored a hit on the pri­ma­ry end­point, the biotech now has to ex­plain why its sec­ond con­fir­ma­to­ry study failed.

Raul Ro­driguez, Rigel CEO

It’s not an en­vi­able task. At the end of Au­gust the South San Fran­cis­co-based biotech’s share price $RIGL went on a roller coast­er ride with a pos­i­tive but unim­pres­sive 18% re­sponse rate for im­mune throm­bo­cy­tope­nia pa­tients who achieved a sta­ble platelet re­sponse. That was ex­act­ly the same rate seen in the sec­ond Phase III, on­ly this time one pa­tient on a place­bo al­so achieved a sta­ble platelet re­sponse, and that scut­tled any shot at hit­ting the pri­ma­ry end­point. For an­a­lysts who had been look­ing for a re­sponse rate at least in the low 20s with clear signs of ef­fi­ca­cy, this stum­ble will not play well.

In­vestors didn’t care for news, ei­ther. Rigel’s shares plunged 33% in ear­ly trad­ing Thurs­day.

Rigel CEO Raul Ro­driguez, though, says if you add all the da­ta from both Phase III stud­ies to­geth­er, you still come up with a pos­i­tive out­come. And that’s what they want to take to the FDA.

“We be­lieve that the to­tal­i­ty and con­sis­ten­cy of da­ta from the FIT Phase III pro­gram, which in­clud­ed two Phase III stud­ies and one long-term ex­ten­sion study, strong­ly sup­ports a clear treat­ment ef­fect, with a sus­tained clin­i­cal ben­e­fit of fos­ta­ma­tinib,” said Ro­driguez in a state­ment.  “We are en­cour­aged by these re­sults and be­lieve that the risk/ben­e­fit ra­tio for fos­ta­ma­tinib is pos­i­tive for pa­tients with chron­ic/per­sis­tent ITP, a pop­u­la­tion with a se­ri­ous un­met med­ical need.  As a re­sult, we will con­tin­ue to pur­sue this op­por­tu­ni­ty. Our next step is to seek feed­back from the FDA.”

The ther­a­py is a spleen ty­ro­sine ki­nase (Syk) in­hibitor, which should help stop the bleed­ing that oc­curs when some­one has a low platelet count. But in a small proof-of-con­cept study for throm­bo­cy­tope­nia, the ther­a­py scored a 50% re­sponse rate, which had whet­ted ap­petites for some­thing sol­id in Phase III.

There’s no turn­ing back now, though. Rigel laid off close to half of its staff last month as it cleared the decks for an FDA ap­pli­ca­tion and launch plans.

Back in 2013 fos­ta­ma­tinib flopped in a Phase III study of rheuma­toid arthri­tis, forc­ing As­traZeneca to wash its hands of their part­ner­ship and leav­ing Rigel to un­der­go an over­haul, shed­ding staff and re­or­ga­niz­ing around dry eye dis­eases (R348) and lu­pus (R333). They both quick­ly failed Phase II. An­oth­er drug, R118, had to be aban­doned af­ter in­ves­ti­ga­tors tracked some se­ri­ous side ef­fects.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.