Upcoming events: Contrave up for US approval again and the EHA meeting

Welcome to your weekly digest of approaching regulatory and clinical readouts. Second time could be the charm for Orexigen’s obesity treatment Contrave, with the FDA due to decide on approval by June 11. Now with positive CV data under its belt Contrave could make headway but needs the marketing push to deliver.

Meanwhile two members of the IPO class of 2013, Acceleron Pharma and bluebird bio, are due to release early data at the European Hematology Association (EHA) congress, taking place June 12-15. And following its successful Asco, TG Therapeutics will also be presenting the first combination data on its anti-CD20 MAb TG1101.

Orexigen Therapeutics/Takeda: Contrave

Obesity drug Contrave – which Orexigen has referred to as NB32 since February, suggesting a brand name change is on the way – was originally given a mixed but largely positive US panel vote back in December 2010. However, two months later a CRL was issued with the FDA requesting a new trial to judge the risk of major cardiovascular events (FDA wipes obesity slate clean with rejection of Orexigen's Contrave, February 1, 2011).

The Light study measuring CV outcomes in over 10,000 patients has since produced positive results at an interim look, although few details were released at the time (Orexigen sees Light at the end of the tunnel, November 25, 2013).

In terms of the competition both Arena’s Belviq and Vivus’s Qsymia were eventually waved through although both have struggled to gain market traction. Arena has signed up Eisai to help sell Belviq in the US while Qsymia has suffered from reimbursement issues and lack of a marketing partner (Vivus attracts dubious bid interest as Orexigen awaits verdict, May 30, 2014).

Neither Arena nor Vivus will have CV data for years, and Orexigen now stands as the only obesity company with a large CV trial under its belt, which could help it make headway in Europe. Takeda has co-promotion rights in US, Canada and Mexico and Orexigen will be hoping it manages a more impressive launch than its competitors.

EvaluatePharma consensus shows that analysts covering Takeda have forecast sales of $390m by 2020.

19th Congress of the European Hematology Association

Taking place in Milan, Italy, Acceleron will present data on sotatercept and ACE-536 in β-thalassaemia; ACE-536 also has data in myelodysplastic syndrome (MDS).

Beta-thalassaemia is an inherited blood disorder that reduces the production of haemoglobin, while MDS is characterised by the inability of the bone marrow to produce mature red and white blood cells as well as platelets. Both can lead to severe anaemia.

A small phase II trial of ACE-536 aimed to evaluate its effect on anaemia in patients with transfusion-dependent or non-transfusion dependent low-intermediate risk MDS. ACE-536 was shown to increase haemoglobin levels. Four out of 10 transfusion-dependent patients experienced a ≥50% reduction in transfused units during an eight week treatment interval versus the eight weeks prior to treatment.

In the beta-thalassemia trial ACE-536 also showed reductions in transfusion requirements. Further efficacy details of dose groups are expected at the meeting. Similarly with sotatercept, data from the higher dose and follow-up from existing cohorts are expected.

Expectations

Acceleron completed its $84m IPO last year and to date shares are up 51%. Although the data are encouraging it is still early stage and currently there are no forecasts for either drug. Leerink analysts note the probability of success for MDS at 30% and 40% for β-thalassemia.

Another of last year’s IPO class, bluebird bio will present data on its gene therapy LentiGlobin. Hitting $101m raised, it was the 8th biggest IPO last year but the stock has fallen back 16% to date (Fast times in biotech push IPO count to new highs, January 6, 2014).

LentiGlobin works by inserting a fully functional human β-globin gene. Data from the small phase I/II European study is expected with the first look at the transplant outcomes in two β-thalassemia patients with up to six months of follow-up. Data in sickle cell disease patients is expected later this year, while US trials in both indications are going ahead.

Finally TG Therapeutics will present early data on their anti-CD20 monoclonal antibody TG1101, known generically as ublituximab, and the PI3K inhibitor TGR-1202. Initial data on the individual drugs at Asco caused its shares to rally (Asco Event Analyzer – 2014’s winners and losers, June 5, 2014).

Investors are keen to see combination data of TG1101 and ibrutinib. The first set of response data in four CLL and two MCL patients will be released at the EHA meeting, with the abstract noting that treatment was well tolerated with early clinical activity observed.

Product Company Trial Name Trial ID
ACE-536 Acceleron - NCT01749514
NCT01749540
sotatercept Acceleron - NCT01571635
LentiGlobin bluebird bio HGB-205 (EU trial in β-thalassemia and sickle cell disease) NCT02151526 
LentiGlobin bluebird bio HGB-204/Northstar (US trial in β-thalassemia) NCT01745120
LentiGlobin bluebird bio HGB-206 (US trial in sickle cell disease) Expected to start this year
TG1101 TG Therapeutics - NCT02013128 
TGR-1202 TG Therapeutics - NCT01767766

To contact the writer of this story email Joanne Fagg in London at joannef@epvantage.com or follow @JoEPVantage on Twitter

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