New Spherix Global Insights research finds mitapivat (Agios) has surged to the top of hematologists’ most desired SCD pipeline therapies, as enthusiasm grows for both oral disease-modifying agents and potentially transformative gene therapies.

Exton, PA, Aug. 25, 2026 (GLOBE NEWSWIRE) -- New research from Spherix Global Insights reveals a shifting outlook for the future of sickle cell disease (SCD) treatment, as hematologists express growing enthusiasm for a more diverse therapeutic landscape that combines potentially transformative gene therapies with more accessible oral disease-modifying options. Findings from the recently published Market Dynamix™: Sickle Cell Disease (US) 2026 study, based on a July survey of 67 U.S. hematologists and qualitative interviews with actively treating physicians, show that interest in innovation is broadening across mechanisms and treatment modalities. While CRISPR-based approaches continue to generate considerable enthusiasm, pyruvate kinase (PK) activators have moved prominently into the spotlight as physicians look for therapies capable of addressing persistent disease burden while potentially reaching patients for whom gene therapy may not be appropriate, accessible, or preferred.

Mitapivat Leads Hematologists’ Most Desired Pipeline Therapy, Rising Threefold Year Over Year

Mitapivat, an oral PK activator from Agios currently approved for thalassemias and marketed as Aqvesme, emerged as the most desired pipeline agent in this year’s research, selected by 24% of hematologists compared with just 8% in 2025. BEAM-101, Beam Therapeutics’ investigational base-editing gene therapy, and Novo Nordisk’s oral PK activator etavopivat followed at 16% each, underscoring the breadth of physician interest spanning both potentially curative approaches and more scalable disease-modifying therapies.

The findings suggest that the future SCD treatment paradigm is unlikely to be defined by a single therapeutic approach. Rather, hematologists envision a market in which gene therapy, oral disease-modifying treatments, transfusion strategies, and existing therapies play complementary roles based on patient characteristics, disease severity, treatment goals, and ability to navigate complex treatment pathways.

Physicians who selected mitapivat as their most desired pipeline therapy frequently pointed to its efficacy, oral administration, and PK-activating mechanism of action. They also highlighted the potential advantages of simpler logistics and broader accessibility compared with more complex treatment approaches. At the same time, enthusiasm for BEAM-101 reflects continued interest in gene-editing technologies and the possibility of a durable treatment effect from a one-time intervention. “The SCD market is moving beyond a conversation centered on any single therapeutic modality,” said Sarah Hendry, Hematology Franchise Head at Spherix Global Insights. “Hematologists clearly remain excited about the transformative potential of gene therapy, but they are also telling us that practical, accessible disease-modifying options have tremendous value. The strong rise in interest in PK activators suggests physicians are increasingly envisioning a treatment landscape with multiple mechanisms and levels of treatment intensity that can be matched to the needs and circumstances of individual patients.”

Seven in Ten Hematologists Continue to Rate Unmet Need in SCD as High

That demand for additional options comes against a backdrop of substantial and persistent unmet need. Seven in ten hematologists surveyed rate unmet need in SCD as high, and physicians estimate that 51% of their SCD patients are not optimally managed with current treatment. Transfusions, pain crises, vaso-occlusive crises (VOCs) and end-organ damage remain common, while one in five SCD patients are estimated to rely exclusively on transfusion because they cannot take other prescription medication for their disease.

The gap between therapeutic innovation and current disease control helps explain why physicians appear receptive to multiple approaches rather than viewing emerging treatments as direct substitutes for one another. Even as physician experience with and interest in gene therapy increase, its use remains constrained by affordability, prior authorization, service coordination, treatment duration and facility capacity, potentially limiting the number of patients who can realistically pursue these options.

Oral PK Activators Gain Momentum Alongside Gene-Editing Innovation

Oral pipeline therapies may therefore play an important complementary role. Interest in etavopivat (Novo Nordisk) increased following positive Phase III HIBISCUS results, with 57% of surveyed hematologists reporting significant interest in prescribing the therapy, up from 42% in 2025. Forty-two percent now consider etavopivat a substantial advance over currently available SCD therapies, compared with 35% the prior year. Hematologists most often cite efficacy as its most appealing feature, while oral dosing and mechanism of action also contribute to interest.

Hematologists Look for a 46% Reduction in VOCs or Pain Crises from New SCD Therapies

Despite growing enthusiasm for the pipeline, hematologists continue to set a meaningful efficacy bar for new entrants. Hematologists indicate that a new SCD therapy would need to deliver, on average, approximately a 46% reduction in annual VOCs or pain crises and a 1.9 g/dL increase in hemoglobin to be considered successful. While these thresholds have moderated somewhat compared with 2025, they demonstrate that physicians continue to expect new entrants to deliver clinically meaningful improvements across multiple dimensions of disease.

Collectively, the findings point toward a new phase of SCD market development, one in which emerging treatment modalities may increasingly complement rather than directly compete with one another. Gene therapy continues to hold the promise of profound and potentially durable disease modification for appropriately selected patients, while emerging oral therapies may offer a pathway to extend meaningful treatment advances to patients for whom intensive gene therapy is not appropriate, accessible, or preferred.

As the pipeline advances, Spherix expects treatment decisions to become increasingly individualized, with efficacy, treatment burden, route of administration, access and patient preference all shaping how physicians position new therapies within an expanding SCD treatment arsenal.

About Market Dynamix ™

Market Dynamix™ is an independent, data-driven service focused on understanding the evolving dynamics of specialty markets poised for disruption. Leveraging quantitative and qualitative research, the service evaluates current treatment approaches, unmet needs, and likely impact of pipeline agents over a three-to-five-year horizon.

About Spherix Global Insights

Spherix Global Insights is a leading independent provider of market intelligence and advisory services for specialty pharmaceutical and biotech markets. By combining physician, patient, and payer perspectives into a single integrated view, Spherix helps brand teams, market access stakeholders, and investors understand how a specialty market actually behaves, not how any one stakeholder describes it. The firm operates eight dedicated therapeutic franchises – Central Nervous System, Dermatology, Gastroenterology, Hematology, Nephrology, Oncology, Ophthalmology, and Rheumatology – alongside a fully dedicated Market Access team. Each franchise is led by hyper-focused analysts and researchers who track these specialty areas full-time, drawing on independently curated communities of vetted practicing specialists, KOLs, patients recruited through treating physicians and advocacy partners, and medical and pharmacy directors at commercial and government payers. Spherix delivers quarterly trending, launch tracking, chart audits, KOL synthesis, patient experience research, and payer and market access intelligence that support confident, strategic decision-making across the pharma lifecycle. Trusted by 19 of the top 20 pharmaceutical and biotech organizations, Spherix has been a go-to resource for more than a decade for leading brands, market access stakeholders, industry media outlets, financial analysts, professional organizations, and patient advocacy groups seeking an unbiased, holistic view of specialty markets.

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CONTACT: Sarah Hendry, Hematology/Oncology Franchise Head
Spherix Global Insights
484-879-4284
sarah.hendry@spherixglobalinsights.com