From neuroscience and endocrinology to oncology, rare disease and chronic blood disorders, a number of new drugs are set to hit the market in 2024.
We’ve zeroed in on five launches, all involving late-stage or recently approved treatments, with the most disruptive potential. Selections are based on an outlook recently issued by investment bank J.P. Morgan and other analyst commentary.
In no particular order, then, here are a handful of key launches to keep close tabs on in the months ahead. Note: Food and Drug Administration approval times are subject to change and are approximate, as are revenue forecasts.
1. Bristol Myers Squibb’s/Karuna Therapeutics’ KarXT (schizophrenia)
A new drug application for KarXT is under review, with a FDA decision likely by the end of November. However, the muscarinic agent has been in the spotlight of late as the centerpiece of Bristol Myers Squibb’s $14 billion takeout of Karuna Therapeutics last week. (Days later, BMS scooped up cancer drug developer RayzeBio for about $4.1 billion.)
Invented at PureTech Health and developed by its founded entity Karuna, KarXT has emerged as an efficacious alternative to standard of care in schizophrenia with an improved safety profile. It’s also in development for adjunctive schizophrenia and Alzheimer’s disease psychosis.
Expected to debut in late 2024, the drug is expected to have a solid launch. “As a reminder, our physician feedback on this asset has been overwhelmingly positive with data supporting a differentiated safety and efficacy profile,” J.P. Morgan analysts wrote in a 2024 investor outlook.
Indeed, real-world adherence of atypical antipsychotics is 50% to 60%, Leerink Partners’ Marc Goodman pointed out in a November note. “Management expects that persistency would be longer with fewer missed days for its asset,” he wrote.
A recent study showed KarXT was not associated with clinically meaningful increases in blood pressure in adults with schizophrenia, eliminating an overhang. Wall Street forecasts 2025 revenue of $211 million.
BMS may not have the muscarinic antipsychotic space all to itself, though. AbbVie’s recent $8.7 billion acquisition of Cerevel Therapeutics, including Cerevel’s late-stage asset emraclidine, sets the stage for a faceoff between the two organizations.
2. Ascendis Pharma’s TransCon PTH (hypoparathyroidism)
Ascendis Pharma’s NDA for TransCon Parathyroid Hormone (PTH) bounced back from a May complete response letter to score an FDA target action date, set for 12 months later.
The agency cited concerns around manufacturing control strategy, not the drug’s clinical data. Having worked to resolve those issues, Ascendis should have no problem getting the drug to pass regulatory muster this time around, especially considering its clearly differentiated efficacy and safety profile in adult hypoparathyroidism.
Leerink Partners’ Joseph Schwartz recently raised his approval odds for the drug to 85%, up from 65%. U.S. market entry is expected in the back half of the year. J.P. Morgan analysts “see the potential for a robust launch given strong data shown to date and unmet need in the space,” they wrote.
That stems, in part, from PTH’s power to facilitate patients’ transition away from supplements and to normalize their urinary calcium.
As such, the JPM team forecast a bullish 2025 revenue estimate of $305 million, versus the street’s consensus of $164 million. Leerink’s Schwartz projects the drug could rack up peak U.S./EU sales of about €1.1 billion by 2030.
The agent is built on Ascendis’ proprietary TransCon platform, a pro-drug technology for creating longer-acting versions of protein, peptide and small-molecule therapeutics. Also in development are TransCon C-Type Natriuretic Peptide (CNP) in achondroplasia, TransCon TLR7/8 agonist for solid tumors and TransCon hGH in adult growth hormone deficiency.
“Net/net, we see multiple products in the Ascendia portfolio with potential to drive value and see an attractive entry point assuming TransCon PTH comes to market in a reasonable time frame,” the JPM analysts commented.
3. Janssen’s/Legend Biotech’s Carvykti (2L+ multiple myeloma)
Janssen’s Carvykti (cilta-cel) immunotherapy is already approved for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy. By this spring, the FDA is set to decide whether to greenlight the CAR-T drug for use as early as the second-line setting. And that could unlock further growth — a lot more.
Expectations have been quite high for the drug moving into earlier lines of therapy in multiple myeloma (MM), an area in which treatment demand far outstrips supply. “We expect Carvykti capacity to increase (though still partly constrained) in 2024 and expect fast uptake in the 2L+ setting, particularly in high-risk patients, if approved by its April 5 PDUFA date,” J.P. Morgan analysts wrote.
The drug is expected to achieve blockbuster status in 2024. If the supplemental BLA is approved based on results of the CARTITUDE-4 trial, the street will look for Carvykti to double revenues (to $2.4 billion) by the following year.
Meanwhile, an FDA investigation into malignancies related to these drugs implicated all six approved CAR-T therapies. But it’s expected to have minimal impact on the category, analysts noted.
The JPM team characterized Carvykti as the “best-in-class” CAR-T cell product in MM with “unprecedented efficacy and practice-changing potential.” It added that sales could eclipse $7 billion by 2035.
4. AstraZeneca’s/Ionis Pharma’s Wainua (hATTR-PN)
The FDA greenlit Wainua (eplontersen) in hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN) last week. AstraZeneca and Ionis Pharma said they will begin commercializing the drug in the U.S. starting in January.
Wainua is the only approved medicine that can be self-administered monthly via auto-injector to combat ATTR-PN, a rare, debilitating disease which leads to peripheral nerve damage with motor disability within five years of diagnosis. Without treatment, it is generally fatal.
Wall Street is expecting about $44 million in 2024 sales, rising to $151 million in 2025.
Beyond its launch, attention for the asset will shift to a forthcoming topline readout — expected in early 2025 — involving Phase 3 data from the CARDIO-TTRansform study of Wainua in the larger ATTR cardiomyopathy (ATTR-CM) market. An exploratory analysis had hinted at cardiac benefit in patients with a related condition.
All of this brings us to one of the sector’s most high-profile catalysts of 2024, Alnylam’s pivotal HELIOS-B readout for Amvuttra (vutrisiran) in ATTR-CM. Slated for the first half of the year, the readout is seen as “key to unlocking a multibillion-dollar opportunity in ATTR cardiomyopathy,” the J.P. Morgan team observed. It could have lateral impact on Ionis, AstraZeneca and other competitors.
5. CRISPR Therapeutics’/Vertex’s Casgevy and Bluebird Bio’s Lyfgenia (sickle cell disease)
The FDA approval of these two gene-based therapies for sickle-cell disease (SCD), which came on the same day in December, provides a rare opportunity to witness a head-to-head launch.
Casgevy (exa-cel), which is the first approved drug utilizing CRISPR/Cas9 technology, enjoys a strong clinical profile in both SCD and transfusion-dependent beta thalassemia (TDT), another genetic blood disorder. Regulators are set to decide on the drug’s TDT application on March 30.
Lyfgenia (lovo-cel), Bluebird Bio’s cell-based gene therapy, uses a lentiviral vector for genetic modification. The label includes a black box warning for hematologic malignancy, with lifelong monitoring recommended.
Both offer the potential of a one-time, transformative therapy for eligible patients. There’s little differentiation on efficacy or safety between the two.
On the other hand, their wholesale acquisition costs diverge considerably: $2.2 million for Casgevy and $3.1 million for Lyfgenia, although Bluebird’s outcomes-based contracts may trim its net price.
The prevailing forecasts confer an edge to Casgevy, at least going by the street’s consensus of $173 million in 2024 and $585 million in 2025. (J.P. Morgan analysts project much lower Casgevy revenues — 2024 and 2025 sales of just $11 million and $44 million, respectively — due to their belief that “improved conditioning regimens” are needed to fully unlock its value.) For Lyfgenia, street consensus is $50 million for the coming year and $163 million the next.
What may be underappreciated, however, is Bluebird’s prior experience in the gene therapy space. The firm nabbed a greenlight for TDT treatment Zynteglo in 2022, followed by Skysona in CALD. That means the company “just” needs to append Lyfgenia to its existing contracts with qualified treatment centers.
If that strategy enables Bluebird to benefit from first-mover advantage, it could prove consequential as the two SCD therapies’ near-concurrent launches play out.
