Key data read outs and patent hearings in the first half of 2012

Analysis

Results from late-stage studies are often make-or-break events for smaller drug makers, and the first half of 2012 is likely to yield a number of big read outs. Notable phase III studies due to report include Actelion’s pulmonary arterial hypertension drug, macitentan; ImmunoGen’s Herceptin follow-on trastuzumab-DM1, licensed to Roche, and Stimuvax, a novel lung cancer vaccine developed by Merck KGaA and Oncothyreon.

Disappointing results from Theravance and GlaxoSmithKline’s Relovair earlier this month show a big backer willing to fund an extensive pivotal programme is no guarantee of success; the companies below will be hoping for a more unequivocal win (Glaxo's Relovair struggles to impress,January 9, 2012). Meanwhile, the outcomes of a couple of big patent cases are also likely to be closely watched in the coming months, with Momenta Pharmaceuticals awaiting two verdicts, in cases over its generic versions of Sanofi’s Lovenox and Teva’s Copaxone (see tables).

Data read outs

Actelion continues to await results from the Seraphin trial with macitentan. The company’s biggest potential growth driver with a product NPV of $1.7bn, the study needs to prove the drug helps patients live longer if it is to compete with incumbents Tracleer and Gilead’s Letairis (Event - Actelion nears do-or-die news on macitentan, January 11, 2012).

ImmunoGen’s antibody drug conjugate trastuzumab-DM1 is currently in a phase III trial as a second-line treatment of advanced HER2 positive breast cancer, named the Emilia study. Interim results are expected in mid 2012 and will form the basis of regulatory filings; a strong result is needed to overcome the disappointment of 2010, when the FDA refused to grant the drug accelerated approval (Roche and ImmunoGen sent reeling by FDA roadblock, August 27, 2010). Results in a first-line setting are not due until 2016.

The first quarter should see interim phase III data from the Start trial, conducted with non-small cell lung cancer vaccine Stimuvax. Despite the relative successes of the prostate cancer treatment Provenge, cancer vaccines have had a chequered past; these results could give an important confidence boost to “off-the-shelf” cancer vaccines, or cast another shadow over this field of research (Event – Oncothyreon awaits pivotal cancer vaccine data, December 19, 2011).

Targacept and partner AstraZeneca eagerly await top line phase III results for the depression drug TC-5214 in two fixed dose trials and a long term study. Hopes are low for a positive outcome, however, following disappointing results from ealier studies in the programme (Pipeline setbacks hurt AstraZeneca, December 20, 2011).

Finally, KRX-0401 or perifosine, an anti cancer drug that inhibits Akt activation in the PI3K pathway, is expected to report phase III results in advanced refractory colorectal cancer. The study is being conducted under a special protocol assessment and the drug already has fast track designation. As Keryx’s biggest growth driver the company will be hoping for a strong read out, as will partner AEterna Zentaris which has bought ex-US rights. Further phase III results are expected towards the end of 2012 in the treatment of multiple myeloma.

Drawn from EvaluatePharma's Calendar of Events, the tables below highlight some of the biggest data read outs and patent decisions facing the industry, outside of big pharma.

Data read outs
Product Pharmacological Class Company Event Date Product  NPV ($m) NPV as % of Market Cap Market Cap ($m) Trial IDs
Macitentan Endothelin receptor antagonist Actelion Phase III results H1 1,775 +36% 4,968 NCT00660179
Trastuzumab-DM1 Anti-HER2 (ErbB-2) MAb-DM1 maytansinoid conjugate ImmunoGen Phase III results H1 1,081 +113% 956 NCT00829166
Stimuvax Anti-MUC1 vaccine Merck KGaA Phase III results H1 415 +2% 22,920 NCT00409188
Stimuvax Anti-MUC1 vaccine Oncothyreon Phase III results H1 727 +271% 268 NCT00409188
TC-5214 Alpha 4 & beta 2 nicotinic agonist Targacept Phase III results H1 576 +285% 202 NCT01153347
                                    NCT01197508
                                    NCT01152554
Toctino Retinoid Basilea Pharmaceutica Top line phase III results Q1 358 +86% 417 NCT00817063
KRX-0401 Akt kinase/PI3K inhibitor Æterna Zentaris Phase III results Q1 309 +174% 178 NCT01097018
KRX-0401 Akt kinase/PI3K inhibitor Keryx Biopharmaceuticals Phase III results Q1 307 +142% 216 NCT01097018
Varisolve Capillary protectant BTG Phase III results H1 262 +16% 1,631 NCT01197833
                                    NCT01072877
                                    NCT01231373 
TZP-101 Ghrelin agonist Tranzyme Top line phase III results Q2 245 +307% 80 NCT01285570
                                    NCT01296620
Amigal Alpha galactosidase chaperone Amicus Therapeutics Phase III results Q2 218 +103% 211 NCT01218659
PA-32540 NSAID & proton pump inhibitor (PPI) POZEN Phase III results Q2 76 +59% 129 NCT00960869
                                    NCT00961350
QVA-149 Anti-cholinergics Vectura  Phase III results H1 74 +22% 329 NCT01294787
                                    NCT01315249
                                    NCT01202188
                                    NCT01120717
                                    NCT01120691
Reolysin Oncolytic virus - Ras activated Oncolytics Biotech Interim phase III/OS data Q1/H1 n/a - 320 NCT01166542

Patent decisions

On January 24 a court is due to hear Amphastar’s appeal on the injunction on its generic version of Lovenox, which a court late last year ruled should be removed from the market because it infringed Momenta’s patents. The case is important because Momenta earns bigger royalties from partner Sandoz as long as it remains the sole provider of generic enoxaparin.

Momenta, along with Teva, is also awaiting the outcome of legal tussles over generic versions of the Israeli giant's MS therapy, Copaxone (Event – Teva needs Copaxone patent victory, August 22, 2011).

Meanwhile Endo’s case against Watson, which is trying to launch a generic version of Lidoderm, goes to trial on February 6. Watson’s 30-month stay expires on July 15. The case is testing the strength of patent ‘529, which expires in October 2015; another trial testing patent ‘510, which expires in March 2014, is also pending but not confirmed.

Analysts currently model sales of the pain patch peaking in 2013 at $844m then falling back to $655m the following year, suggesting they believe a settlement between the two companies will be reached to allow Watson to launch in 2014 . With the court case nearing, a settlement could come any time. 

Patent decisions
Product Pharmacological Class Company Event Date Product  NPV ($m) NPV as % of Market Cap Market Cap ($m)
M356 MS agent Momenta Pharmaceuticals Patent update H1 1,164 +117% 991
Lidoderm Local anaesthetic Endo Pharmaceuticals Patent trial Feb 6 785 +18% 4,347
Lidocaine Local anaesthetic Watson Pharmaceuticals Patent update H1 313 +4% 7,663
Enoxaparin sodium Heparin, low molecular weight (LMWH) Momenta Pharmaceuticals Patent update H1 267 +27% 991
Lexapro SSRI Forest Laboratories Generic Lexapro launch Mar 14 130 +2% 8,610
Lovaza Omega-3 fatty acid Pronova BioPharma Patent update H1 97 +27% 356

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