Welcome to your weekly digest of approaching regulatory and clinical readouts. Topline phase III results are expected shortly for Kythera’s ATX-101 for the reduction of unwanted submental fat (under the chin). If approved the injectable treatment would be the first non-surgical option for this facial area.
Bayer, which has rights outside the US and Canada, reported positive results from a European trial last year, and Kythera has previously shown efficacy two years after treatment; it could well attract interest from bigger aesthetic players. Elsewhere, the European Society of Cardiology's annual congress starts on August 31 in Amsterdam, where several presentations will be closely watched – one of the most keenly awaited is full safety data from the Onglyza outcomes trial, run by the diabetes drug's collaborators, Bristol-Myers Squibb and AstraZeneca.
Kythera Biopharmaceuticals/Bayer: ATX-101
ATX-101 is a synthetic formulation of sodium deoxycholate, which Kythera states is a well-characterised endogenous compound that promotes natural breakdown of dietary fat.
Results from two trials called Refine 1 and 2 are expected in the late third or early fourth quarter. The studies recruited 1,000 patients from the US and Canada, and tested subcutaneous 2mg/cm
Primary outcomes included rating scales from clinicians and participants, while an MRI was used to measure the thickness of the fat as a secondary outcome.
In two phase III studies that recruited 723 patients, Bayer said the project was well tolerated and demonstrated a statistically significant reduction of moderate to severe submental fat compared with placebo, as assessed by clinician and subject satisfaction rating scales. Improvements were also confirmed by the use of calipers to measure fat thickness.
Kythera received $43m up front from the Bayer deal back in 2010, with milestones set at $330m. Following the positive European results Kythera received a $33m milestone payment.
Analysts covering Kythera have forecast sales of $361m by 2018, according to consensus data from EvaluatePharma, which gives an NPV of $940m, more than double the company’s market cap. Leerink Swann estimates US peak sales potential of more than $500m. Analysts covering Bayer are less optimistic about the product's potential – sales are seen hitting $8m in 2016.
Analysts at JP Morgan estimate that ATX-101 could initially target 2 million US patients who currently receive other facial aesthetic treatments such as botulinum toxin or dermal fillers. Analysts also highlight potential off-label use such as in the back and knee area.
A US filing is expected in the first half of 2014 with European filing around the end of this year. Typical treatment for fat under the chin includes liposuction and surgical neck lifts. If approved ATX-101 would become the first in a new class of aesthetic drugs to reduce this fat without the need for surgery.
Kythera completed its $70m IPO last October, and ATX-101 is its only project; the California company has funding for at least the next 12 months.
The hope is that ATX-101 might attract interest from existing aesthetic players, and with many of Kythera's executive team having worked at Allergan, the aesthetic giant could be waiting in the wings.
European Society of Cardiology Congress 2013 August 31 – September 4, Amsterdam
Among the most closely watched data to emerge at the ESC Congress this year will be detailed safety results from AstraZeneca and Bristol-Myer Squibb’s Onglyza outcomes trial. Topline results revealed that the study found no cardiovascular benefit – but importantly no harm.
However, this drug class is still haunted by lingering concerns about pancreatic cancer, and the full results of this 16,500-patient trial will be scrutinised for safety (Regulators clear incretin mimetics – for now, August 2, 2013).
Daiichi Sankyo will also be at the conference, presenting data on Lixiana (edoxaban) from a trial called Hokusai in venous thromboembolism. Data in the much bigger indication of atrial fibrillation are expected in autumn.
Meanwhile Amgen will report full results from a phase IIb heart failure study testing an intravenous form of omecamtiv mecarbil, which it licensed from Cytokinetics. Results should help determine whether the agent is destined for phase III development (Amgen’s global ambitions for omecamtiv spark renewed interest in Cytokinetics, June 24, 2013)
Isis will be unveiling phase II results from a pipeline project that is attracting increasing amounts of attention: ISIS-APOCIIIRx, in patients with severely high triglycerides. Positive data from a small phase II trial earlier this year raised hopes for the project, which works by knocking out the gene coding for ApoC-III, a glycoprotein that inhibits lipase and thus prevents clearance of triglycerides from the blood (ADA – Knockdown result boosts Isis and antisense alike, June 25, 2013).
|Phase III in US and Canada||NCT01542034
|Completed Phase III in Europe||NCT01294644
Please note that EP Vantage will be closed August 26 for the UK bank holiday.
All information is sourced to analyst notes and the EvaluatePharma Calendar of Events tool.