A new report highlights promising therapeutic developments for HIV, Parkinson’s disease, Crohn’s disease, alopaecia, multiple myeloma and breast cancer on the horizon, with personalised medicines strongly represented.
Analysts Clarivate have identified 15 late-stage experimental treatments that are each forecast to deliver annual sales of more than $1 billion within five years.
These promising advancements include a broad spectrum of therapeutic developments for rare diseases and tough-to-treat conditions, that have the potential to transform treatment paradigms and serve unmet patient needs.
- Bimekizumab (BIMZELX) developed by UCB. Bimekizumab is the first dual IL-17 A/F inhibitor to treat moderate to severe plaque psoriasis. Phase III trial results showed superior skin clearance outcomes than existing treatments. Its less-frequent dosing schedule and good safety profile will likely be attractive to clinicians and patients.
- Capivasertib (AZD5363) developed by AstraZeneca. For patients with breast cancer, Capivasertib is a novel, highly potent, selective ATP-competitive pan-AKT kinase inhibitor that exerts similar activity against the three isoforms AKT1, AKT2 and AKT3. Positive data have emerged from early-phase trials, with clinical benefit to patients irrespective of their PIK3CA/AKT1/PTEN mutational status, and several Phase III trials are now underway.
- Daprodustat (GSK1278863/Duvroq) developed by GSK plc. Daprodustat belongs to a novel class of oral treatments for chronic kidney disease (CKD)-related anemia and is a HIF-PHI developed to treat anemia associated with CKD, which has a high incidence rate and few effective, safe treatment options. Already available for CKD-related anaemia in Japan, its uptake has been impressive.
- Deucravacitinib (SOTYKTU/BMS-986165) developed by Bristol Myers Squibb. As a first-in-class oral, targeted agent that selectively inhibits tyrosine kinase 2 (TYK2), a Janus kinase (JAK) family member that mediates cytokine-driven immune and inflammatory signals, it has the potential to fill a gap in the treatment armamentarium for plaque psoriasis.
- Foscarbidopa/foslevodopa (ABBV-951) developed by AbbVie. Foscarbidopa/foslevodopa is a novel reformulation of the gold-standard Parkinson’s disease treatment (carbidopa/levodopa) delivered via a subcutaneous pump for the treatment of motor fluctuations in advanced Parkinson’s disease. In addition to serving a niche group of patients with high unmet need, it offers better efficacy than orally administered carbidopa-levodopa, dosing flexibility and a more convenient pump than existing and upcoming competitors.
- Lecanemab (BAN2401) developed by Eisai Co Ltd and Biogen Inc, and
- Donanemab (LY-3002813), developed by Eli Lilly and Company. Lecanemab and Donanemab are poised to help treat early-stage Alzheimer’s disease. Supported by landmark clinical data from a Phase III trial, next-in-class anti-Aβ monoclonal antibody (MAb) lecanemab appears poised for full US FDA approval and ex-US launches. Donanemab, and others in the class (e.g., Roche’s gantenerumab), may follow suit pending the results of ongoing trials.
- Lenacapavir (Sunlenca/GS-6207) developed by Gilead Sciences Inc. Approved in Europe and under evaluation by the US Food and Drug Administration (FDA), lenacapavir is the first-in-class, long-acting HIV-1 capsid inhibitor approved to treat multi-drug resistant (MDR) HIV in people who have been heavily treated, a patient population with unmet medical need. Also currently being investigated to treat HIV and for pre-exposure prophylaxis (PrEP), its infrequent dosing and self-administration will likely make it a favored choice in a population with treatment adherence challenges.
- Mirikizumab (LY-3074828) developed by Eli Lilly and Company. Mirikizumab, a monoclonal antibody targeting the p19 subunit of IL-23, will likely be first-in-class for ulcerative colitis and the third in the class approved for Crohn’s disease. Part of a set of emerging therapies with novel mechanisms of action, it will contribute to the growing market share held by these therapies.
- Pegcetacoplan (EMPAVELI/ASPAVELI/APL-2) developed by Apellis Pharmaceuticals Inc. Pegcetacoplan has launched already in the United States and Europe for Paroxysmal nocturnal hemoglobinuria (PNH), a rare hematological disease. As one of the few drugs to have completed Phase III trials for GA, pegcetacoplan is expected to be the first drug to launch for geographic atrophy (GA) or ‘dry late age-related macular degeneration (AMD)’, which has no approved pharmacotherapy.
- Ritlecitinib (PF-06651600) developed by Pfizer Inc. Ritlecitinib will likely benefit from its first-in-class status, rapid onset of action and expected label for both adults and adolescents, potentially providing an effective option to stimulate hair growth in a stigmatising disease – Alopaecia areata.
- Sparsentan developed by Travere Therapeutics Inc. Sparsentan is a first-in-class, orally active, single molecule that functions as a high-affinity, dual-acting antagonist of both endothelin type A (ETA) and angiotensin II subtype 1 (AT1) receptors, which are associated with progression of kidney disease. Its development for IgA nephropathy and focal segmental glomerulosclerosis (FSGS) promises to halt that progression for many patients and fills a gap in the treatment armamentarium.
- Teclistamab (TECVAYLI/JNJ-64007957) developed by Janssen Pharmaceutical Companies of Johnson & Johnson. After receiving conditional approval from the EC (European Commission), teclistamab is the first-in-class bispecific antibody targeted to B-cell maturation antigen (BCMA) to treat multiple myeloma. Ongoing Phase III trials are expected to provide confirmation of clinical benefit in teclistamab’s approved setting and lead to label expansions in other multiple myeloma patient populations, including in combination with other approved agents.
- Teplizumab (TZIELD/PRV-031) developed by Provention Bio Inc. Teplizumab is the first immunotherapy to launch for T1DM and is a landmark drug given its potential ability to preserve beta cell function and delay the need for insulin treatment in those with type 1 diabetes mellitus (T1DM).
- Valoctocogene roxaparvovec (ROCTAVIAN/BMN-270) developed by BioMarin Pharmaceutical Inc. Approved by the European Commission (EC) in August 2022, valoctocogene roxaparvovec is also poised to be the first gene therapy to launch in the United States for severe haemophilia A. Treatment benefit is expected to last for years, reduce the number of bleeding events, minimise the need for replacement factor VIII (FVIII) and negate the use of otherwise burdensome prophylaxis treatment.
The annual ‘Drugs to Watch’ report identifies drugs entering the market or launching key indications in 2023 which are predicted to achieve blockbuster status by 2027 or be clinical game changers for millions of patients worldwide.
Candidate drugs in Phase II or Phase III trials, at pre-registration or registration stage, or already launched early in 2022 were selected for analysis, including those pursuing new indications that could be particularly impactful; drugs launched prior to 2022 were excluded. Clarivate analysts evaluated each drug in its individual context, based on factors such as expected approval or launch dates, competitive landscape, regulatory status, trial results, market dynamics and other key factors, and added novel drugs that, while likely to fall short of blockbuster status, are poised to be therapeutic game-changers.