Will pending clinical readouts make it across the line?

Several key data readouts slated for the first quarter remain unannounced, from Roche, Lilly, Axsome and others, but the Covid-19 pandemic puts many at risk of delay.

With just under two weeks left of the first quarter several major data readouts slated for this period are still outstanding. The coronavirus pandemic means that delays are now inevitable, of course.

Provention Bio and Iveric Bio will surely not be the only companies having to pause clinical programmes this week. Still, there is a glimmer of hope that those that managed to get their studies across the finish line before global lockdowns commenced could still report topline results.

Vantage has compiled a list of these likely candidates, though they all will be considering what sort of market reaction they might expect. Those with positive data could be considering delaying for fear of a muted response from investors; on the flip side, some could take the opportunity to bury bad results.

Clinical results due from the big cap arena includes Roche’s pivotal Mirros study, which is likely to show idasanutlin plus chemo beating cytarabine alone in terms of overall survival in AML. The Swiss company is hoping to replicate the success it has had with Venclexta in this cancer type with this follow-on project, an antagonist of MDM2.

Elsewhere, the first phase III test of Lilly's mirikizumab is due to read out. The company has invested a lot of money in this anti-IL-23 MAb, with seven pivotal studies ongoing; the Oasis-2 study tests mirikizumab versus Novartis’s Cosentyx in psoriasis, and the Lilly project needs to shine. 

Looking to the smaller players, the phase II Path Forward study of Ascendis’s hypoparathyroidism treatment Transcon PTH was due to report late last year. Despite the recent market rout Ascendis still sports a $4.7bn market cap, largely based on hopes for its lead project in growth hormone deficiency, and a second success would add another string to its bow.

Biohaven is targeting migraine prevention with its latest phase II/III study of oral rimegepant, and hopes to position the drug as a more convenient alternative to approved injectable CGRP inhibitors. Meanwhile, Axsome’s AXS-05 is in the Stride-1 trial in treatment-resistant depression. At the end of last year, the Gemini study hit in major depressive disorder, causing Axsome’s shares to rocket 70%.

Looking at even smaller companies, Akero is taking on Nash with its fibroblast growth factor AKR-001. Non-invasive MRI results are expected first, with biopsy data due in the second quarter. And Proqr’s exon-skipping oligonucleotide QR-421a should report early data in Usher syndrome, a rare genetic disorder.

Despite the late stage of these studies, imminent reporting of results cannot be relied on. And, for those with clinical work at more critical stages, the prospect of sticking to timelines looks even bleaker. 

Selected Q1 data readouts
Project Company booking most of sales 2024e sales ($m) NPV ($m) NPV as % of market cap Event (Vantage story link)
Idasanutlin Roche 245 +1,287 +1% Pivotal Mirros data in r/r AML patients
Mirikizumab Lilly 848 +4,547 +3% Phase III Oasis-2 in psoriasis
TransCon PTH Ascendis Pharma 214 +1,487 +28% Phase II Path Forward in hypoparathyroidism 
AXS-05 Axsome Therapeutics 753 +623 +29% Phase III Stride-1 in treatment-resistant depression
Rimegepant Biohaven 868 +1,335 +64% Phase II/III in migraine prevention
Relugolix Myovant Sciences 655 +1,551 +243% Phase III in endometriosis, Spirit 2 due Q1, Spirit 1 due Q2
AKR-001 Akero Therapeutics - - - Phase II Balanced study, non-invasive MRI results due Q1, biopsy due Q2
Etripamil Milestone Pharmaceuticals 92 +428 +111% Pivotal Node-301 in paroxysmal supraventricular tachycardia
QR-421a Proqr Therapeutics 56 +200 +74% Phase I/II Stellar in Usher syndrome type 2
AB-729 Arbutus Biopharma - - - Phase Ia/II single ascending dose in hepatitis B
Sulopenem franchise (oral & IV) Iterum Therapeutics 202 +329 n/m Phase III Sure 1 and Sure 2 in urinary tract infections
Ranked on market cap. n/m=not meaningful. Source: analyst notes, NPV as at March 16 via EvaluatePharma.

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