Voy­ager's gene ther­a­py for Parkin­son's shows promise in ear­ly da­ta from small Phase Ib tri­al

Voy­ager Ther­a­peu­tics $VY­GR re­port­ed some good news from the front lines of its gene ther­a­py clin­i­cal tri­al in Parkin­son’s, show­ing the com­pa­ny’s drug might one day res­cue pa­tients from the in­evitable de­cline as­so­ci­at­ed with their re­sponse to lev­odopa.

Parkin­son’s, char­ac­ter­ized by low dopamine lev­els in the brain, pro­gress­es over time with the pa­tient ex­pe­ri­enc­ing a de­bil­i­tat­ing loss of mo­tor and non-mo­tor func­tions – with symp­toms like trem­bling, slow­ing of move­ments, and im­paired bal­ance. As the dis­ease pro­gress­es, these symp­toms be­come more se­vere, re­sult­ing in pe­ri­ods of de­creased func­tion in the pa­tient, al­so called “off” time. Thanks to ex­ces­sive dos­es of lev­odopa aimed at treat­ing the “off” time, some pa­tients ex­pe­ri­ence in­vol­un­tary move­ments, or dysk­i­ne­sia.

Voy­ager’s ther­a­py, called VY-AADC, is cur­rent­ly in a Phase Ib tri­al that on­ly in­cludes 15 to­tal pa­tients, with 5 in each co­hort. In its best-per­form­ing co­hort, VY-AADC showed a mean in­crease of five hours a day of “on” time — with­out any dysk­i­ne­sia — in Parkin­son’s pa­tients. The ear­ly da­ta have Voy­ager up­beat, as it helped the com­pa­ny nar­row down the dose that will be used in Phase II and III pro­grams.

Bernard Rav­ina

“Giv­en the im­prove­ments in mo­tor func­tion and wider spec­trum to titrate oral lev­odopa with our Co­hort 2 dose, we are ex­cit­ed to con­sid­er this as our like­ly dose in the piv­otal pro­gram while still plan­ning to re­view the six-month re­sults from the Phase 1 pos­te­ri­or tra­jec­to­ry tri­al next quar­ter,” said Bernard Rav­ina, chief med­ical of­fi­cer of Voy­ager Ther­a­peu­tics, in a state­ment. “We look for­ward to re­view­ing these re­sults from the Phase 1b with the FDA as part of a planned Type C meet­ing and we con­tin­ue to ex­pect to dose the first pa­tient in the piv­otal Phase 2-3 pro­gram in mid-2018.”

The Cam­bridge, MA-based biotech re­port­ed a 2.1-hour im­prove­ment in “on” time from base­line to three years for pa­tients in Co­hort 1, a 3.5-hour im­prove­ment from base­line to 18 months in Co­hort 2, and an im­prove­ment from base­line to six months of 1.5 hours that plateaued from six to 12 months in Co­hort 3.

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.

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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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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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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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.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

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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