Tiny hep B drug com­bo study touts po­ten­tial of a ‘func­tion­al cure’ at lit­tle-known Replicor

A small, large­ly un­known biotech in Cana­da is re­port­ing some re­mark­able re­sults from a tiny proof-of-con­cept study for a new hep B com­bo.

Re­port­ing at the Amer­i­can As­so­ci­a­tion for the Study of Liv­er Dis­ease meet­ing in Wash­ing­ton, DC, in­ves­ti­ga­tors for the Mon­tre­al-based Replicor say that they tracked a per­sis­tent func­tion­al cure for 4 of the hand­ful of pa­tients be­ing treat­ed with a com­bi­na­tion ther­a­py. And 8 of 10 pa­tients have achieved what they term “func­tion­al con­trol” over the dis­ease.

An­drew Vail­lant, Replicor

The com­bo match­es an in-house drug dubbed REP 2139 with teno­fovir diso­prox­il fu­marate and pe­gy­lat­ed in­ter­fer­on al­pha 2a. With­out the pro­pri­etary drug, the re­searchers say that pa­tients can ex­pect a 10% re­sponse. And they’re now set­ting the stage for a big­ger tri­al to put their drug to a more am­bi­tious test.

Their de­f­i­n­i­tion of func­tion­al con­trol was the re­duc­tion of he­pati­tis B sur­face anti­gens to less than one IU/mL and vi­ral DNA lev­els of less than 10 IU/mL. This was an open-la­bel study with no con­trol arm.

Not much has been re­port­ed about this pri­vate biotech com­pa­ny, in­clud­ing any de­tails on how it’s been fi­nanced since it was found­ed in 2009.

Replicor CSO An­drew Vail­lant had this to say in a pre­pared state­ment:

The ad­di­tion of REP 2139’s unique abil­i­ty to clear cir­cu­lat­ing HB­sAg im­proves this out­come in a strik­ing fash­ion, achiev­ing func­tion­al con­trol in 80% of pa­tients not on­ly dur­ing treat­ment but per­sist­ing af­ter treat­ment is with­drawn. Equal­ly im­por­tant is the nor­mal­iza­tion of liv­er func­tion dur­ing fol­low-up in all these pa­tients, even those with sig­nif­i­cant­ly el­e­vat­ed liv­er en­zymes at the start of ther­a­py.

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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