More from Stat today on JPM from AF-
On the cusp of the ‘JPM,’ here’s a cheat sheet for what to watch in biotech in 2019
By ADAM FEUERSTEIN @adamfeuerstein JANUARY 2, 2019
It’s that time of year again.
I’m speaking, of course, of the J.P. Morgan Healthcare Conference. Starting Monday, management teams from hundreds of biotech and pharma companies will file into the Westin St. Francis Hotel on the western side of San Francisco’s Union Square. Over four days, these executives will stride up to podiums in conference rooms large and small to pitch their company’s investment story to crowds of Wall Street investors sitting in uncomfortably close quarters.
This is the 37th edition of what is known colloquially as “JPM.” It’s the oldest, largest, and most consequential investor conference on the biopharma industry’s calendar. With the new year still fresh, JPM is the uniquely scheduled confab where biotech and pharma executives offer their first forecasts, milestone timelines, and financial guidance for the 12 months ahead.
Investors take notes, ask questions, listen for answers (truthful or otherwise), and start figuring out which stocks they want to buy, sell, or avoid.
And journalists like myself are on hand to listen and report on what’s being said in those presentations — or more importantly, in the hallways during breaks.
To set the table for JPM19, here are what I see as some of the burning questions that will make or break the most closely followed biotech companies in the coming year.
Amgen: The commercial launch of its migraine drug Aimovig exceeded expectations in 2018, but will that trend continue this year as more competitors hit the market? What happens to Neulasta sales now that a biosimilar has been launched? How will the Enbrel patent litigation case be decided? Will Amgen continue to use its cash on share buybacks and dividends, or engage in a blockbuster M&A deal? Is a mega-merger between Amgen and a Big Pharma company in the cards for 2019?
Gilead Sciences: Welcome, new CEO Daniel O’Day. What is your business and strategic plan for Gilead? Is this the last year of declining earnings and revenue before the company starts growing again? Will two key late-stage clinical trial readouts — selonsertib in NASH and filgotinib in rheumatoid arthritis — succeed or fail? Can Biktarvy keep Gilead’s dominant position in HIV intact? Can cancer cell therapy — Yescarta, in particular — grow into a commercially meaningful business?
Biogen: Will there be an interim analysis of the aducanumab Phase 3 Alzheimer’s study? How will Spinraza continue to perform if (when) Novartis’ gene therapy for spinal muscular atrophy is approved toward the middle of the year? Will Biogen acquire an innovative biotech to diversify away from its reliance on an aging multiple sclerosis franchise and an ultra-risky Alzheimer’s drug candidate?
Alexion Pharma: Ultomiris is approved, so how quickly can the company convert patients from Soliris? Can Alexion fend off competitors seeking to develop their own drugs to treat complement disorders?
Celgene: Is a CEO change necessary to turn around rock-bottom investor negativity and the plunging stock price? Will a settlement with Dr. Reddy’s over generic Revlimid finally happen — and on favorable terms? Can the company execute on regulatory filings and late-stage clinical trial readout for its “Big 5” pipeline products: fedratinib, ozanimod, luspatercept, JCAR017, and bb2121? Even if all goes well with the Big 5, will investors start buying the stock or does Celgene still need to do more?
Vertex Pharma: How fast can the triple combination regimens in cystic fibrosis be filed, approved, and launched? Is there any remaining threat from cystic fibrosis competitors? How will Vertex manage patient access and reimbursement, particularly in Europe where regulators have balked at high prices? With profits from cystic fibrosis treatments accelerating, how does Vertex choose to use its cash?
Bluebird Bio: The first approval for Lentiglobin in beta-thalassemia is expected this year in Europe, so how will the gene therapy be priced and are enough patients willing to undergo the procedure? The multiple myeloma CAR-T partnership with Celgene is complicated by Celgene’s ownership of potentially competing therapies from Juno, so how is the company planning for potential conflicts? How fast can the “accelerated” Lentiglobin development plan in sickle cell disease deliver regulatory filings and approvals?
Sage Therapeutics: Will SAGE-217 succeed in the Phase 3 study in postpartum depression? If the study reads out negative, what changes, if any, will be made to the SAGE-217 development plan or regulatory filing timeline? Can the company deliver a strong commercial launch of Zulresso (brexanolone) in postpartum depression once the drug is approved in March?
Regeneron Pharma: Will the Dupixent commercial launch in asthma duplicate the drug’s strong showing in atopic dermatitis? Can Regeneron maintain growth in its core Eylea franchise? Will the recent approval of Libtayo and the advancement of its pipeline products help the company become a significant player in immuno-oncology?
Biomarin: Will the efficacy of the hemophilia A gene therapy valrox remain durable when three-year data are disclosed in the middle of the year? Can Biomarin file valrox for accelerated approval in 2019? Will the Phase 3 study of vosoritide in achondroplasia (the most common form of dwarfism) yield positive results at year end? Revenue is growing but when will the company stop losing money and finally deliver sustained profitability?
Sarepta Therapeutics: Will the next clinical trial updates from gene therapy programs targeting Duchenne muscular dystrophy and Limb-Girdle muscular dystrophy impress as much as last year’s initial data readout? Is this the year that Sarepta is finally acquired?
Agios: New CEO Jackie Fouse starts in February. Will she make major changes to the company that differ from founder and first CEO David Schenkein?
Intercept Pharma: Will the first-half 2019 interim analysis of the obeticholic acid (OCA) Phase 3 study in NASH read out positive with no significant safety issues? And if the OCA data in NASH look clean, how quickly can an accelerated approval filing be submitted? What impact will competing clinical trials of NASH drugs have on investor outlook for OCA?
Alnylam Pharma: Will the report of fourth-quarter 2018 sales for Onpattro plus management discussion of first quarter performance be strong enough to overcome doubts investors have about this orphan drug launch? Will Alnylam’s next RNAi drugs givosiran and lumasiran succeed in their own respective Phase 3 trials?
Related: Massachusetts is trying to make the Berkshires a biotech hub. Can it succeed where so many other cities have failed?
Acorda Therapeutics: Inbrija was approved early, but can the Parkinson’s drug meet or beat sales expectations in its first year on the market — enough to make up for falling Ampyra sales?
Alkermes: How big is the commercial market opportunity for ALKS3831 in schizophrenia? Assuming the FDA rejects ALKS5461 in depression, will the company shake up its R&D strategy and pipeline?
Amarin: What evidence can management provide — higher sales, a boost in prescriptions — to show that Vascepa is leveraging the REDUCE-IT study results to blockbuster status?
Solid Biosciences: Can the first look at data generated by its gene therapy for Duchenne muscular dystrophy match or surpass what’s been shown by Sarepta Therapeutics?
Sangamo Therapeutics: Will updated data from MPS II patients treated with its zinc finger genome editing therapy erase doubts raised last year by the first, confounding study results?
Myovant Sciences: Will readouts from a trio of Phase 3 clinical trial programs for lead drug Relugolix vindicate Vivek Ramaswamy and his Roivant business model? Or, will his hard luck continue?
GW Pharma: How much will the growing awareness of (and enthusiasm for) cannabinoid-based drugs help Epidiolex perform commercially in its first year on the market?
AI
