Spotlight On: The 2021 JP Morgan Healthcare conference – five key takeaways

A smattering of deals and other headline plays

Sanofi and Novartis both used the first day of the conference as a backdrop to announce notable deals. 

Sanofi is to acquire the UK biotech Kymab for up to $1.5 billion to strengthen its longer-term aspirations in the atopic dermatitis and broader immunology markets.

Novartis agreed to in-license the PD-1 inhibitor tislelizumab in major markets outside of China at an upfront cost of $650 million. The Swiss company has so far (and somewhat surprisingly) struggled to break into the immuno-oncology market.

Analysis - ViewPoints: Sanofi looks beyond Dupixent in atopic dermatitis and ViewPoints: Novartis rejigs its PD-1 game with some help from BeiGene

Elsewhere, Eli Lilly confirmed positive data from a Phase II study evaluating its investigational Alzheimer's disease treatment donanemab.

Though significant questions remain, these data were cheered by Biogen, which claims positive read across to its more advanced aducanumab programme (see ViewPoints: Eli Lilly’s early donanemab win elicits cheers from across the aisle).

Speaking at the conference on Monday, senior management at Biogen appeared bullish towards the FDA's pending approval decision for aducanumab and expressed their surprise that a panel of experts convened by the agency late last year had unanimously voted to recommend against marketing authorisation.

Bluebird bio also announced this week that it plans to split into two separate companies focused on rare diseases and oncology. The company has struggled over the past year to retain prior momentum, but investors appear reticent to buy into the value-unlocking thesis of a split.

Analysis – ViewPoints: More bluebirds to love same questions to answer

More M&A to come

There has been considerable speculation since the turn of the year that industry deal-making will increase in 2021, following disruption last year caused by the COVID-19 pandemic. Comments made by senior management at multiple large-cap companies presenting at JP Morgan this week would attest to this.

Perhaps most striking was confirmation from Bristol Myers Squibb CEO Giovanni Caforio, who oversaw the $74-billion acquisition of Celgene in 2019 and then signed off a $13-billion deal to buy MyoKardia in October, that another deal similarly sized to the latter is well within the company's reach this year due to its "significant financial flexibility."

He described business development as a top priority in the year ahead. Whilst expensive, the acquisition of Celgene not only delivered Bristol Myers Squibb a large cash generator in Revlimid, but cost savings have exceeded expectations.  

Elsewhere at JP Morgan, Vertex Pharmaceuticals CEO Reshma Kewalramani provided a bullish outlook on the M&A front: "We have $6.2 billion in cash and no debt. We continue to believe the greatest value we can generate is from investing in innovation – both internal and external. On business development, our strategy is to identify assets that complement our pipeline."

See ViewPoints: Pandemic to help shape deal-making in 2021 and beyond says new report, as JP Morgan kicks off

Can gene therapy recover?

The burgeoning field of gene therapy has entered 2021 under a cloud of uncertainty, shaped by a number of regulatory and clinical setbacks, the most significant of which is arguably the FDA's decision last year not to approve BioMarin's investigational haemophilia A gene therapy Valrox, but instead ask the company to gather more data.

On the eve of this year's JP Morgan Healthcare Conference, Sarepta Therapeutics added fuel to the fire by reporting mixed top-line Phase II results for its closely watched Duchene muscular dystrophy (DMD) gene therapy SRP-9001, prompting its share price to fall as much as 54%.

The conference gave both companies an opportunity to provide updates. In BioMarin's case management was armed with updated one-year Phase III data for Valrox, which was released on Monday. This makes European approval look more likely, but does not appear sufficient to meet the demands of the FDA, which wants two-year data before making a decision. BioMarin will continue its discussions with the US regulator, regardless, with the suggestion being that management is hopeful a compromise can be met. At the very least it still expects two-year data to be compelling enough to secure (albeit delayed) approval.

For Sarept,a things may be more challenging as new data raise doubts as to whether SRP-9001 actually works at all (see ViewPoints: Lost lead in DMD gene therapy race may be least of Sarepta’s worries). At JP Morgan management reiterated that study design, specifically unlucky randomisation of patients between the treatment and placebo arms of the trial, were to blame for the results unveiled last week. Fresh data, showing encouraging levels of microdystrophin levels in patients who were initially treated with placebo and subsequently received SRP-9001, were put forward by the company, though the jury remains out on whether this particular gene therapy has a future.

There was retained momentum last year for gene therapy approaches using gene editing, most notably for the partnership between CRISPR Therapeutics and Vertex, with data presented at ASH in December showing early promise in sickle cell disease and beta thalassaemia. At JP Morgan, the emerging company Verve Therapeutics presented intriguing preclinical data from animal models for a one-time gene therapy designed to put the brake on PCSK9 and lower LDL cholesterol. The company touted the data as showing comparable levels of LDL cholesterol reduction to that seen with monoclonal antibody treatment, six months after administration of the gene therapy. The plan is to dose initial patients next year.

mRNA / COVID-19 in the spotlight

Twelve months is a long time in biopharma and 2020 is a testament to that. At last year's JP Morgan Healthcare conference, which occurred against the backdrop of early-reports of a potential new coronavirus in China, BioNTech and Moderna were still thought to be years away from bringing novel products to the market.

We all know what happened next and both companies were given prominent presenting slots during the first day of this year's conference. Moderna in particular now looks poised to use its success in bringing a highly effective COVID-19 vaccine to market within 10 months as a slingshot to accelerate a broad development of mRNA vaccines and therapeutics, with a particular focus on the infectious disease market.

See ViewPoints: COVID’s first responders blueprint their longer-term growth strategies at JPM

How established players in the vaccine market such as Sanofi and GlaxoSmithKline respond to the arrival of mRNA will be an important trend to watch over the next 12 to 18 months. Both companies have invested in this approach, but it remains to be seen if they can keep pace with upstarts such as Moderna. The market for influenza vaccines, which BioNTech and Pfizer describe as primed for disruption with mRNA technology, could be a key battleground.

BMS firms up its post-Celgene growth outlook

Typically (when not in a virtual format) as much a networking event as anything else, the JP Morgan conference also acts to set the scene for the year ahead. On this front then, various Big Pharma companies plotted out a roadmap of a key products to watch over the next 12 months and beyond.

Most notable, perhaps, was Bristol Myers Squibb's disclosure of revenue targets for a number of emerging growth driver products through to 2029 for the first time. Management projects sales of at least $4 billion by 2029 for its Tyk-2 inhibitor deucravacitinib (in psoriasis and other indications), Reblozyl (for anaemia in patients with beta thalassaemia) and mavacamten (for symptomatic obstructive hypertrophic cardiomyopathy), the latter integrated into Bristol Myers Squibb's pipeline through last year’s acquisition of MyoKardia. There is hope also that the cell therapy franchise comprising liso-cel (lymphomas) and ide-cel (multiple myeloma) will generate sales of at least $4 billion by the end of the decade. Zeposia – for multiple sclerosis and ulcerative colitis – is expected to generate at least $3 billion in sales by 2029.

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