JPM18 Day 2: Roche on gene therapy, Aimmune nears key readout and outpatient CAR-T
Investors came out in droves Monday night to attend the parties in San Francisco hosted by nearly every law firm, investment house and pharma company. Evercore ISI's Burgers and Beers featured black suited men spilling into the bedding section of Macy's, while the famed Tweetup saw communications folks waiting in the rain with the venue at capacity.
Despite the festivities, day two at the J.P. Morgan Healthcare Conference and the Biotech Showcase kicked off early with attendants crowding into panels eager to chat about the trends of the moment.
J.P. Morgan's little brother Biotech Showcase is boasting 3,000 attendees this year from more than 1,900 different companies.
Heard ‘round the halls
Panelists at the Endpoints breakfast expressed concern the immuno-oncology space has become a little too crowded. Aiman Shalabi of the Cancer Research Institute noted already 26 immuno-oncology agents are approved globally and that PD-1/L1s are in combination trials with more than 100 different modalities.
And they weren't the only ones asking how many PD-1/L1s the industry really needs. One business development exec told BioPharma Dive that immuno-oncology deals aren't getting the same buzz as last year or the year before. Now, investors are waiting to see how earlier deals pan out. Organizations like MassBIO, meanwhile, are pushing investors to finance other spaces that haven't won the same sort of financial love.
Roche not impressed by gene therapy
Roche CEO Severin Schwan isn’t worried about the new wave of gene therapies in the clinic replacing his new blockbuster hemophilia medicine Hemlibra (emicizumab-kxwh).
When asked at the conference if gene therapies are a threat, Schwan pointed to the strong data the drug has shown in the clinic and the ongoing clinical trials sponsored by Roche. The HAVEN4 study is testing Hemlibra as an injection given every four weeks and Schwan thinks this will satisfy patient needs.
"The data is really stunning," he noted.
"I think gene therapy is an option, but it’s early days. The other thing that I think is important to mention here is that Hemlibra delivers a significant reduction in bleeding events and bleeding rates and you know these patients are treated already. I think Hemlibra brings patients an additional benefit of already reduced bleeding rates and competing against that is challenging. I’m not saying it’s not possible, but it’s quite challenging to do that," the exec added.
"It’s a little bit further out and we’ll see how that works out. It might be a better option in the future, but I think we’ve raised the bar."
Driving toward a treatment for peanut allergy
Aimmune Therapeutics Inc. is weeks away from reading out results from a Phase 3 study of its lead treatment, an oral drug called AR101 that's designed to protect people with peanut allergies from severe allergic reactions.
The biotech, which saw its stock rise by 85% in 2017, hopes to file for approval by the end of the year if all goes well.
Looking back at the company's growth over the past four years, Aimmune CEO Stephen Dilly said investor questions at the J.P. Morgan meeting have moved from ‘Who the hell are you?' to ‘Tell me about your commercial plans.'
With AR101, Aimmune is betting it can desensitize allergic individuals to peanut protein by administering successively higher doses of the drug over time.
“The term we use as sort of a holy grail is 'bite-proof protection,'” Dilly explained, referring to reaching a level of protection where an allergic child could take a bite of a peanut-butter sandwich, spit it out and be fine.
Success in PALISADE would help support a potential Biologics License Application and keep the treatment moving toward market. A setback, however, could cause Aimmune to suffer a similar fate as its rival DBV Technologies, which saw its shares fall 40% on a Phase 3 miss for its own peanut allergy product.
Bayer leaps at innovation
Known as the maker of aspirin and for that little acquisition of Monsanto, Bayer wants to get the word out that it is also an innovator in spaces like oncology and cardiovascular disease.
Participants strolling San Francisco's Union Square this week may see one of the many ads Bayer has plastered across ped-cabs and other public places.
Launched last year, the big pharma created an investment arm, dubbed Leaps, which is pouring money into early biotechs and joint ventures across a variety of disciplines.
Bayer's Head of U.S. Pharma Carsten Brunn told BioPharma Dive the program hopes to provide cures for problems related to health and the food supply caused by an aging and growing population. In the pharma space, Bayer will focus on six disease areas including blood disorders, blindness, and heart disease.
Leaps will invest $575 million over five years and provide companies with strategic direction. Bayer will ultimately get first right of refusal to license any products that result from the collaborations.
Brunn explained that the program is called Leaps because the investments are expected to take five to ten years to mature — a long time horizon than is typical of venture investing — and are meant to be great leaps in science, not just incremental advancements.
Cell therapy is a complex and potentially risky treatment, requiring cautious monitoring after infusion of souped-up T cells into a patient. Even so, improving product safety profiles, as well as growing physician expertise, have raised the possibility of using CAR-T in an outpatient setting
It's a possibility that Juno Therapeutics believes it can make a reality with its experimental candidate for lymphoma, lisocabtagene maraleucel. The biotech had previously discussed the prospect of outpatient CAR-T alongside updated clinical data presented the American Society of Hematology in December.
At the J.P. Morgan meeting, outpatient treatment with liso-cel was a talking point for company CEO Hans Bishop's presentation to investors and analysts.
"With a safety database from this study of now approaching one hundred patients, you can see a tolerability profile that we think will allow this drug to be used in the outpatient setting," Bishop said.
In particular, the biotech chief noted that approximately 80% of lymphoma patients targeted by liso-cel are treated outside of major academic transplant centers.
"That is why we find our tolerability profile so encouraging, that it will allow us to move outside of those transplant centers and take JCAR017 or liso-cel to the site of care for many of these patients."
Doing so will require caution, as Bishop took care to note. But it could reduce CAR-T's burden on hospitals and help manage associated treatment costs.
Not all CAR-T companies share Juno's eagerness to test the waters with outpatient treatment, though.
"This is not something where we are going to rush to that," said bluebird bio CEO Nick Leschly, commenting in a session Tuesday on questions about outpatient treatment for his company and Celgene Corp.'s multiple myeloma candidate bb2121.