The hum in San Francisco dimmed noticeably on day three of the JP Morgan healthcare conference, with the last full day of presentations effectively closing out a distinctly downbeat affair. Perhaps expectations were too high for deal-making given the lull in M&A in 2017, though few corporate updates made waves this year either.
Standing out on Wednesday was Novavax, whose management put a rocket under the company’s share price with some very bold predictions for an important data readout later this year. Global Blood Therapeutics similarly managed to add to share price gains with a confident performance. And Editas mounted a defence of its gene editing technology in the wake of recently surfacing doubts. EP Vantage rounds up these and other updates from the meeting.
Novavax is in the midst of running a huge 8,000-plus patient trial of its respiratory syncytial virus candidate, called Prepare, examining whether vaccinating mothers can prevent infants from contracting the illness. An interim read out is due mid-year, ahead of a primary endpoint analysis early in 2019, and at the conference yesterday the company presented details of an “informational analysis”. This was conducted by a DSMB statistician who received unblinded data from the ongoing study.
Novavax said this analysis targeted an efficacy threshold against the primary endpoint at day 90 of > 40%, and was conducted to reassure management that further investment was justified. The answer it received was yes, with the analysis meeting the criteria, indicating that the vaccine is efficacious, the company claimed.
Of course extrapolating from this that the trial will succeed is still a risky bet. Particularly when remembering that a previous phase III trial failed – the 12,000 elderly patient Resolve study – and that earlier data have been far from entirely convincing (The puzzling excuse for Novavax's pivotal failure, 16 September 2016). But this was enough for some investors, who added 56% to the company’s market value, taking it to $685m. RSV is a space that has never failed to disappoint, and Novavax will remain a controversial story until the final data are in.
Global Blood Therapeutics was also the big share price winner of day three, which EP Vantage has covered in more depth today (JP Morgan – Vox pops Global Blood stock ahead of actual data, January 11, 2018).
Along with rival Crispr players Intellia and Crispr Therapeuticas, Editas shares slumped Monday as investors digested the implications of a scientific paper that suggested patients could harbour innate immunity to the Cas9 protein. This is vital to much of the Crispr gene-editing work currently on-going and with the technology still largely unknown, investors were rattled.
The question was raised at Editas’s breakout session but was largely brushed off by executives, who stated that their data suggest that the magnitude of the problem is not as reported in the paper. “This is obviously something we’ve been focusing on for some time,” said Vic Myer, chief technology officer. “We have a very deep scientific package to ask this question. We haven’t been sharing it broadly with scientific community, but will do so.”
Recovery in Crispr stock prices this week suggests that investors are not too perturbed, but these companies will remain closely watched this year. Progress has been very slow – for investors at least – but at least Editas confirmed that the IND for its first candidate, against a form of inherited blindness, remains on track to be filed mid-2018. The company said it aims to have three projects in early stage trials by 2022.
Sign us, please
Esperion left investors with the very clear message that the management team is currently working to one goal – finding a partner or buyer. With several late-stage studies of its cholesterol lowering project bempedoic acid due to read out over 2018, the burning question is whether partners need to see pivotal data before signing on the dotted line.
What they do not need to see is outcome data, chief exec Tim Mayleben said, boldly describing the data as not very important commercially, and more about “checking boxes and confirming what everybody knows”.
Those data are due in around 2022, by when a partner will hopefully be in place. Mr Mayleben even refused to speculate on what it might cost Esperion to set up a commercial infrastructure, saying “if I answered that, it would imply we’re even considering launching ourselves, and I want to stay away from that.”
Around 1,000 reps would be needed to target primary care physicians, he estimated, and there are between five and 10 companies globally that fit the bill as potential partners. “We know who they are, they know who we are,” he said.
Accentuate the positive
German mRNA player Curevac attempted to demonstrate its progress over the last year, after having to announce the failure of its lead candidate at JP Morgan last year. Chief executive Ingmar Hoerr admitted that they realised things had to change when prostate cancer vaccine CV9104 failed in phase II, but insisted that the bad results had turned into something positive.
He boasted about the oncology collaboration with Lilly, which he claimed as the biggest mRNA deal last year, and a partnership with Crispr Technologies. And pledged to have six phase I trials running by the end of 2018 – two vaccines and four oncology projects. mRNA will change paradigms, Mr Hoerr stated, predicting that the technology will eventually replace recombinant proteins.
Curevac, which has been around for considerably longer than Moderna, seems to have similarly bold ambitions. Both have achieved “unicorn” status, with their private valuations exceeding $1bn, and the pressure for all of these players to prove themselves in the clinic will only build with such audacious pronunciations.
Mountain to climb
Historically, Denali Therapeutics has been notably reluctant to reveal much of what it is working on, though in the wake of December’s IPO it seems that information might begin flowing more readily. A thorough look at the neurodegeneration specialist’s pipeline at JP Morgan revealed some interesting titbits, with chief exec Ryan Watts detailing six programmes – saying a further five are being kept under wraps.
Much of the focus was on LRRK2 inhibition in Parkinson’s disease; the company has two assets in phase I in healthy volunteers, which should progress into phase Ib and Parkinson’s patients by the end of the year. And management made no secret of their desire to do more deals, having only last week finalised a huge collaboration with Takeda, which included $150m of up-front payments.
Mr Watts said they were continuing to look at potential partnerships with bigger companies, although admitted that the bar was now higher after the Takeda deal. And with a cash pile of $600m, presumably the urgency to sign up new partners has also waned.
Not playing for peanuts
Peanut allergy player Aimmune tightened the timelines for data from its pivotal study, Palisade. A top-line readout will occur in February, with a full presentation planned for the AAAAI annual meeting in March.
Executives presented data at JP Morgan showing how patients become increasingly desensitised over time, which they claimed gives them confidence about the phase III trial. In Palisade the primary endpoint, the proportion of subjects ages 4 to 17 years who tolerate at least 600 mg of peanut protein with no more than mild symptoms, is measured over 12 months.
Hopes have risen for the project in the wake of disappointing results for rival DBV Technologies; shares in Aimmune closed at close to a record high Wednesday, valuing the company at $2bn.
An EP Vantage staff report, with reporting by Madeleine Armstrong in San Francisco. For live updates from the JP Morgan healthcare conference in San Francisco on January 9-12 follow @ByMadeleineA on Twitter. To contact the writers of this story email firstname.lastname@example.org.
See our full coverage from the JP Morgan conference here.