Welcome to your weekly digest of approaching regulatory and clinical readouts. Investors betting on Allergy Therapeutics’ most important near-term catalyst, readout of a phase III study of Birch MATA MPL in birch pollen allergy, have had an agonising few months: readout of the data, aiming to secure the vaccine’s full approval in Germany, has now been delayed twice.
Results were initially expected at the end of last summer, but in September Allergy said they would not come until the end of 2018, claiming that independent statistical analysis was taking longer than expected (After 11 long years Allergy sees a glimmer of hope, May 21, 2018).
This seemed to point the finger at the contract research organisation, but in November Allergy delayed the readout again, to the current quarter, this time without giving a reason.
The data are important because the German TAV (therapy allergens ordinance) requires products currently sold on a named-patient basis, as several of Allergy’s vaccines are, to be fully authorised with the backing of clinical trials. Named-patient sales bring Allergy some $90m a year, and are profitable.
The study, PQBirch301, completed enrolment in January 2018, randomising over 560 subjects and seeking to beat placebo on efficacy. The efficacy measure is the total combined score of the rhinoconjunctivitis daily symptom and rhinoconjunctivitis daily medication scores, averaged over the peak 2018 birch pollen season, which is understood to have been severe.
Allergy this week insisted that it remained blinded to the data. This should be reassuring unless you suspect that it gleaned something from blended, blinded scores that made it fear that Birch MATA MPL would fail.
As long as the group can blame third-party incompetence there might be no reason to panic, though the markets are unlikely to take kindly to a third delay.
Tregs for type 1 diabetes
In the next few weeks Caladrius Biosciences should get a definitive answer on whether there is merit in its approach to treating some type 1 diabetics.
The group is due to report phase II results from the T-rex study of CLBS03, its T regulatory cell (Treg) candidate. CLBS03 is designed to expand and enhance patients’ Tregs, which damp down the immune system, the idea being to stop the destruction of beta cells in the pancreas that leads to the disease.
However, Caladrius is not offering a cure. The goal of CLBS03 is to reduce or stop disease progression and lessen the need for insulin treatment by preserving patients’ remaining beta cells. As such, one of the big issues for this approach is patient identification: candidates have to be identified early enough in their disease to still have functioning beta cells.
To this end, T-rex has enrolled 110 newly diagnosed patients aged eight to 17. Subjects have been randomised to placebo in the form of insulin, or 2.5 million Tregs or 20 million Tregs per kilo of body weight. The primary endpoints are preservation of C-peptide levels, an accepted measure for pancreatic beta cell function, insulin use, severe hypoglycaemic episodes and glucose and haemoglobin A1c levels.
The project passed an important test when an interim analysis gave T-rex the go-ahead to continue to completion. CLBS03 also has US fast-track and orphan drug designation because of the paucity of treatments for type 1 diabetes, which is addressed by life-long insulin use.
Despite the limited options for patients, analysts are not assigning much value to Caladrius’s approach. Sellside forecasts from EvaluatePharma see revenues of $21m in 2024.
The low expectations might also be down to the limited uptake that might be expected if CLBS03 is priced in line with other cells therapies, especially given the availability of cheap insulin. But reimbursement worries are a long way down the road, and for now Caladrius has to get through phase II trials.
|Birch MATA MPL||PQBirch301||Phase III to back full German approval||2016-002781-31|