Welcome to your weekly digest of approaching regulatory and clinical readouts.
All information is sourced to analyst notes and the EvaluatePharma Calendar of Events tool.
On March 18 the Australian company Pharmaxis should hear final word from the FDA on its cystic fibrosis (CF) project Bronchitol. Hopes are low since an advisory panel voted against approval at the beginning of the year, citing concerns over the drug's safety and efficacy. Upon that news, Pharmaxis shares plummeted nearly 46%, wiping $183m off the company’s market cap.
Analysts at Deutsche bank note that US launch could be pushed back to 2015 with the expectation of lengthy delays and possible requirement for further clinical trials following the FDA rejection.
Bronchitol was approved in Europe after an initial negative CHMP decision, and is the leading growth driver for the company, with worldwide sales in CF forecast by EvaluatePharma to reach $322m by 2018. The silver lining could be use of Bronchitol in non-CF bronchiectasis (a build-up of mucus due to damaged bronchi in the lung), with analysts at Edison Research noting that this use could be eight times the size of the CF market in Europe and the rest of the world.
Phase III results in bronchiectasis are expected in the second quarter of this year, and analysts at Credit Suisse note that this is the main valuation driver. However, with $85m in cash operations are only supported until the end of 2014. To keep the project afloat the company badly needs toimprove on its dire share performance and raise more cash.
Phase I/II data on BioMarin’s BMN-701 are expected at the end of the first quarter. The orphan drug is being tested in Pompe’s disease and all eyes will be on how it stacks up against the current standard of care, Sanofi’s Myozyme (BioMarin needs to follow cash call with clinical successes, June 1, 2012).
The key efficacy endpoints are the six-minute walk test (6MWT), the percentage of super-responders in the 6MWT and pulmonary function. Greater than 15% improvement versus Myozyme’s late-onset treatment study data in one of the key criteria, or a greater than 10% improvement in two or more of the criteria, will see the project move forward, BioMarin says.
Analysts at JP Morgan note that a phase III trial could begin in the fourth quarter of this year.
BMN-701 is the fifth-largest growth driver for the California company, with an NPV of $60m, 1% of BioMarin’s market cap. Myozyme is the current market leader and will reach blockbuster status by 2018, according to EvaluatePharma. Analysts at Cowen note, however, that Myozyme is far from a cure, and a modestly better offering could take share. All BioMarin needs is the evidence to prove it.
American Academy of Neurology conference 16-23:
Finally, AAN is taking place from 16-23 March in San Diego. Some of the presentations taking place are highlighted below:
Biogen Idec/Abbvie: Daclizumab
While Biogen will be concentrating on Tecfidera’s upcoming PDUFA, data are expected on March 19 from daclizumab, its injectable multiple sclerosis antibody being developed with Abbvie. An update is expected from the Selection trial, primary results of which were initially reported at Ectrims last year.
While efficacy was sustained in the trial, there was one patient death due to autoimmune hepatitis. Safety was also an issue in the first year of an earlier trial, called Select (Daclizumab dazzles on efficacy but sours on safety, August 10, 2011).
With murky safety results there are obvious risks to commercial success. Despite this, Leerink Swann analysts note that once-monthly therapy could take significant market share from all treatments except orals. Pivotal Decide and Observe trial readouts are expected in 2014.
Biogen Idec/Isis Pharmaceuticals: Isis-SMNRx
On March 20 single-dose phase I data are expected from Biogen/Isis’s antisense drug Isis-SMNRx, which is designed to treat spinal muscular atrophy. The abstract states that no serious adverse events or dose-limiting toxicities were reported.
Isis management has indicated that improvement was seen in some patients after just one dose and expects efficacy to increase with more frequent dosing. However, with such early data Leerink Swann analysts believe that it is too early to draw meaningful conclusions (Isis signs another antisense deal but more success needed, January 5, 2012).
Early results are also due on March 20 from Xenoport’s XP23829. Preliminary phase I data were announced last October. The "BG-12-like" compound achieved expected levels of monomethyl fumarate in the blood with the possibility of once-a-day dosing.
Leerink Swann analysts note that pharmacokinetic details in the full dataset at AAN should create a base on which to compare the drug to Biogen Idec's BG-12. Meanwhile phase I data from the multiple ascending dose trial are expected mid-2013.
|Product||Company||Study name||Trial ID|
(late-onset treatment study)
|ISIS-SMNRx||Biogen Idec/Isis Pharmaceuticals||SMNRx||NCT01494701|