Ryan Livingston joined the MJH Life Sciences team in March of 2025, shortly after graduating from TCNJ with a bachelor’s degree in English. He enjoys creative writing, reading, and scuba diving. He can be reached at rlivingston@mjhlifesciences.com.

After a relatively quiet year, the last quarter of 2025 brought a landslide of major updates to the 

, such as mitapivat and narsoplimab, and the results of major trials like SUMMIT and VAYHIT3, the specialty has grown substantially during the last 3 months.

The editorial team at 

 has collected 9 of the most impactful hematology headlines from Q4 of 2025 – check them out below:


FDA Approves Mitapivat for Transfusion-Dependent and Non-Transfusion-Dependent Alpha- and Beta-Thalassemia


Mitapivat, a small-molecule oral PKR activator developed by Agios, received approval from the FDA on December 24, 2025, for the treatment of 

 in adults with transfusion-dependent or transfusion-independent alpha- or beta-thalassemia. The decision was based on positive results from the phase 3 ENERGIZE and ENERGIZE-T trials, during which more patients treated with mitapivat achieved a transfusion reduction response and had a hemoglobin response compared to those receiving placebo.

FDA Approves Narsoplimab (Yartemlea) As First TA-TMA Therapy


Narsoplimab-wuug was approved for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) on December 24, 2025, as announced by parent company Omeros Corporation. This decision makes narsoplimab the first and only approved lectin pathway inhibitor approved by the FDA. The decision was based on positive data from a single-arm, open-label study in adults with TA-TMA, which was supported by data from an expanded access program enrolling 221 adult and pediatric patients.

FDA Approves Fibrinogen, Human-chmt (Fesilty) for Congenital Fibrinogen Deficiency


Fibrinogen, human-chmt was approved on December 19, 2025, for the treatment of acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency (CFD), including hypo- or afibrinogenemia. The FDA’s decision comes in response to a phase I/III study, during which the only adverse events that occurred in >2% of patients receiving fibrinogen, human-chmt were pain in extremity, back pain, pyrexia, thrombosis, fibrin D dimer increase, headache, hypersensitivity reactions, and vomiting.


FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome


Etuvetidigene autotemcel became the first FDA-approved gene therapy for Wiskott-Aldrich syndrome on December 9, 2025. The drug uses the patient’s hematopoietic stem cells, which have been genetically modified to include functional copies of the WAS gene. These cells are then infused intravenously to restore blood cell production, as well as functional WAS protein expression in affected cells.


FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy


On December 8, 2025, the FDA approved omidubicel-onlv to treat severe aplastic anemia (SAA), establishing it as the first hematopoietic stem cell transplant therapy for this disease. Omidubicel-onlv involves donated cord blood stem cells chemically enhanced with nicotinamide and given to a patient to restore their blood and immune system. This circumvents issues such as delayed hematopoietic recovery and increased infections presented by umbilical cord blood cells, as well as providing additional graft options for patients with SAA.


BASIS: Marstacimab Confirms Efficacy and Safety for Hemophilia A and B


The BASIS trial was an open-label, 1-way crossover, multicenter phase 3 trial investigating marstacimab for a primary efficacy endpoint of annualized bleeding rates versus previous therapy during a prior phase 1b/2 study. Patients ultimately saw a mean bleeding rate reduction from 39.86 to 3.2, while those receiving routine prophylaxis saw a reduction from 7.9 to 5.09.


SUMMIT: Bezuclastinib Outperforms Placebo in Nonadvanced Systemic Mastocytosis


During the phase 2 SUMMIT trial, patients were randomly assigned in a 2:1 ratio to receive bezuclastinib or placebo, on top of supportive care medication. Patients assigned to bezuclastinib displayed substantially greater symptom improvement compared to placebo, with a reduction in serum tryptase of ≥50% achieved by 87.4% of bezuclastinib patients versus 0% on placebo. Parent company Cogent Biosciences also announced plans to present data from a 48-week follow-up period at an upcoming scientific meeting in the first quarter of 2026.


Ianalumab Improves Stable Response, Reduces Fatigue in ITP, With Hanny Al-Samkari, MD


Presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition by Hanny Al-Samkari, MD, the Peggy S. Blitz Endowed Chair in hematology and oncology at the Massachusetts General Hospital and associate professor of medicine at Harvard Medical School, the VAYHIT2 trial demonstrated ianalumab’s efficacy in prolonging time to treatment failure, improving stable response at 6 months, reducing fatigue, delaying need for subsequent therapy, and facilitating tapering off eltrombopag in patients with primary immune thrombocytopenia.


HOPE-B: 5-Year Etranacogene Dezaparvovec Maintains Durable Efficacy and Safety in Hemophilia B, With Steven Pipe, MD


The HOPE-B trial, presented at ASH 2025 by Steven Pipe, MD, a professor of pediatrics and pathology at the University of Michigan, showed that a single dose of etranacogene dezaparvovec maintains durable and endogenous factor IX Padua expression, producing sustained reductions in bleeding rates among patients with severe or moderately severe hemophilia B. During the trial, patients receiving etranacogene dezaparvovec exhibited a substantial reduction in bleed rates for all bleeds during the first 60 months post-therapy.

Articles

The last quarter of 2025 saw significant advancements in hematology, marked by key FDA approvals and pivotal trial results. Notable FDA approvals included mitapivat for thalassemia, narsoplimab for TA-TMA, and etuvetidigene autotemcel for Wiskott-Aldrich syndrome. Major trials like BASIS and SUMMIT demonstrated promising outcomes for hemophilia and systemic mastocytosis, respectively. The VAYHIT2 trial highlighted ianalumab's efficacy in immune thrombocytopenia, while the HOPE-B trial confirmed the long-term efficacy of etranacogene dezaparvovec in hemophilia B, underscoring substantial progress in hematology therapeutics.

The Q4 recap for cardiology spotlights major FDA decisions, key clinical trial updates, and more.

News

news page, resources on the topics of disease- and specialty-specific medical news and expert insight can be found. Content includes articles, interviews, videos, podcasts, and breaking news on health care research, treatment, and drug development.

Clinical

clinical page is a resource for content on every specialty, disease, and topic available on our medical news site.

Hematology

Anemia

FDA News

Q4 2025 Recap: Hematology News and Updates

Melinda Gooderham, MD, is a dermatologist and the medical director at the SKiN Centre for Dermatology.

An announcement was released on December 18 highlighting Takeda’s new positive 

 from two pivotal phase 3 studies on zasocitinib (TAK-279) in adults with moderate-to-severe 

Zasocitinib, a next-generation, highly selective oral tyrosine kinase 2 (TYK2) inhibitor, demonstrated superiority to placebo in the 2 studies’ co-primary endpoints: patients’ static Physician Global Assessment (sPGA) 0/1 and Psoriasis Area and Severity Index (PASI) 75 scores.

 spoke with Melinda Gooderham, MD, dermatologist and medical director of the SKiN Centre for Dermatology in Ontario, Canada.

New Topline Phase 3 Data on Zasocitinib in Psoriasis, with Melinda Gooderham, MD

“They're the 2 duplicate, sister studies looking at zasocitinib, which is in highly selective TYK-2 inhibitor, with both placebo and [they’re] active comparator-controlled,” Gooderham explained.

Gooderham described the Latitude studies as 2 randomized, multicenter, double-blind, placebo- and active comparator-controlled studies. Both were conducted in 21 countries and involved the recruitment of 693 and 1108 participants, respectively. The studies’ co-primary endpoints were determined to be the proportion of treated patients attaining sPGA 0/1 and PASI 75 response versus placebo at the 16-week mark.

Among the key secondary endpoints, Gooderham highlighted comparisons with placebo at Week 16 and apremilast at Weeks 16 and 24. Overall, she described the studies as having demonstrated the superiority of zasocitinib versus placebo for the co-primary endpoints. There had been a significantly greater number of PASI 75 responses observed as early as the 4-week mark and continuing to rise through to Week 24.

In Takeda’s announcement, all 44 ranked secondary endpoints were described as having been met, including sPGA 0, PASI 90, and PASI 100, against placebo and apremilast.

 The drug was generally well tolerated, and its safety findings were consistent with previous research. Gooderham noted the value of a once-daily oral agent delivering complete skin clearance for individuals living with psoriasis.

“I think what's exciting is that we're seeing these levels of biologic efficacy,” Gooderham said. “In the past has been a bit of a trade-off with our oral therapies being a little less effective than the biologics.”

In Takeda’s release, the most commonly seen adverse events (AEs) through the 24-week mark were noted as upper respiratory tract infection, acne, and nasopharyngitis.

 No new safety signals were identified by the investigators.

As stated in a prior HCPLive release, these data are slated to be presented during upcoming medical congresses. Additionally, Takeda plans to submit a New Drug Application to the US Food and Drug Administration (FDA) along with other regulatory authorities in 2026.

The quotes contained in this summary were edited for the purposes of clarity.

Gooderham’s has reported nonfinancial support from Takeda in addition to personal fees from AbbVie, Amgen, Arcutis, Bristol Myers Squibb, Boehringer Ingelheim, Eli Lilly, Galderma, Incyte, Janssen, Leo Pharma, Meiji, Dermavant, Moonlake, Nektar, Nimbus, Novartis, Pfizer, Regeneron, Sanofi, Sun Pharma, Tarsus, Takeda, UCB, Union, Ventyx, and Apogee.

Takeda’s Zasocitinib Landmark Phase 3 Plaque Psoriasis Data Show Promise to Deliver Clear Skin in a Once-Daily Pill, Catalyzing a New Era of Treatment. Takeda. December 18, 2025. Accessed January 10, 2026. 

https://www.takeda.com/newsroom/newsreleases/2025/takeda-zasocitinib-phase-3-plaque-psoriasis-data-once-daily-pill/

Brooks A. Once-Daily Oral Zasocitinib Delivers Strong Phase 3 Efficacy in Plaque Psoriasis. HCPLive. December 18, 2025. Accessed January 10, 2026. 

https://www.hcplive.com/view/once-daily-oral-zasocitinib-delivers-strong-phase-3-efficacy-in-plaque-psoriasis

Videos

In her interview, Melinda Gooderham, MD, highlights the impact of recent topline findings on zasocitinib, a once-daily pill for adults with psoriasis.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

New Topline Phase 3 Data on Zasocitinib in Psoriasis, With Melinda Gooderham, MD

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

A phase 3 trial showed that subcutaneous anifrolumab (Saphnelo) demonstrated significant, clinically meaningful treatment benefits for 

“These meaningful results from the TULIP-SC trial provide confidence that the efficacy and DORIS-defined remission rates that we’ve seen with anifrolumab can be achieved in a new subcutaneous administration, allowing even more patients to benefit from this effective treatment,” said lead investigator Susan Manzi, MD, MPH, chair of the Allegheny Health Network (AHN) Medicine Institute and director of the Lupus Center of Excellence at the AHN Autoimmunity Institute, in a statement.

 “The results align with important changes in global lupus treatment recommendations, which now emphasize earlier intervention with biologics, driving remission and reduced use of oral corticosteroids as key treatment goals.”

Patients with SLE face an increased risk of early mortality, and approximately 50% develop irreversible organ damage within 5 years of diagnosis, largely driven by ongoing disease activity and chronic corticosteroid exposure. Reducing both disease activity and corticosteroid use lowers the risk of organ damage and other debilitating outcomes.

 The revised 2025 SLE recommendations identify achievement of DORIS remission as the primary treatment goal and strongly encourage corticosteroid discontinuation.

Anifrolumab, a first-in-class, fully human monoclonal antibody that binds to subunit 1 of the type 1 interferon receptor and blocks the activity of type I IFN, is available in the US as an IV infusion administered by healthcare professionals.

 Subcutaneous anifrolumab allows patients to self-administer the treatment outside of the clinic; this form was approved in the EU in December 2025.

In the multinational, phase 3, double-blind, placebo-controlled TULIP-SC trial, investigators assessed the efficacy and safety of subcutaneous anifrolumab in adults aged 18 – 70 years with moderate-to-severe SLE despite being on standard therapy, such as oral corticosteroids, antimalarial, or immunosuppressants. The primary endpoint, tested in the pre-planned interim analysis with 220 patients (109 on anifrolumab and 111 on placebo), was the treatment difference in the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) response at 52 weeks.

Participants were randomized 1:1 to receive 120 mg subcutaneous anifrolumab or placebo administered via a pre-filled, single-use syringe. A pre-specified interim analysis was performed once the first 220 participants reached Week 52. The study also included a 52-week open-label extension for participants who completed the initial 52-week treatment period.

The primary endpoint was met, with a difference of 59.4% for anifrolumab vs 43.9% for placebo (BICLA response difference, 15.5%; 95% confidence interval [CI], 2.3 – 28.6; 

The full analysis assessed secondary endpoints across 367 patients, with 184 on anifrolumab and 183 on placebo. More patients treated with anifrolumab vs placebo obtained a BICLA response while maintaining low or reduced oral glucocorticoid doses (≤ 7.5 mg per day) through week 52 (56.2% vs 34.0%; difference, 22.3%; 95% CI, 12.3 – 32.2; 

 <.0001). Patients on anifrolumab also had a reduced time to first sustained BICLA response (hazard ratio [HR], 2.2; 95% CI, 1.5 – 3.2; 

At Week 52, remission by DORIS criteria and attainment of a Low Lupus Disease Activity State were both significantly greater with anifrolumab than with placebo, with treatment differences of 14.2% (95% CI, 5.6 – 22.8; 

 =.0012) and 14.1% (95% CI, 4.6 – 23.6; 

=.0038), respectively. Among patients on anifrolumab, 29% achieved DORIS remission and 40.1% attained low-disease activity.

The anifrolumab arm had a slightly greater frequency of serious adverse events than placebo (11.9% vs 10.4); safety findings were consistent with the known clinical profile of intravenous anifrolumab. Herpes zoster occurred in 3.8% on anifrolumab vs 1.1% on placebo.

“These results reinforce Saphnelo’s unique approach of targeting the type 1 interferon receptor to reduce disease activity, with the added convenience of subcutaneous self-administration,” said Sharon Barrr, executive vice president at BioPharmaceuticals R&D, AstraZeneca, in a statement. “The TULIP-SC findings build on the compelling body of evidence for Saphnelo, which has helped patients achieve remission and significantly reduce reliance on oral corticosteroids – further reinforcing our ambition to transform lupus care.”

Manzi S, Bruce IN, Morand EF, et al. Efficacy and Safety of Subcutaneous Anifrolumab in Systemic Lupus Erythematosus: the Randomized, Phase 3, TULIP-SC Study. 

Saphnelo self-administration TULIP-SC trial demonstrates statistically significant and clinically meaningful reduction in systemic lupus erythematosus disease activity. AstraZeneca. Published on January 6, 2026. 

https://www.astrazeneca.com/media-centre/press-releases/2026/saphnelo-self-administration-tulip-sc-trial-demonstrates-statistically-significant-clinically-meaningful-reduction-sle-disease-activity.html

Sammaritano LR, Askanase A, Bermas BL, et al. 2025 American College of Rheumatology (ACR) Guideline for the Treatment of Systemic Lupus Erythematosus. 

A phase 3 trial demonstrated that subcutaneous anifrolumab significantly benefits systemic lupus erythematosus (SLE) patients when added to standard therapy. The TULIP-SC trial showed improved remission rates and reduced reliance on oral corticosteroids, aligning with updated global lupus treatment guidelines. Anifrolumab, a monoclonal antibody targeting the type 1 interferon receptor, allows for self-administration, enhancing patient convenience. The trial's primary endpoint was met, with significant differences in BICLA response and DORIS remission rates. Safety findings were consistent with known profiles, with slightly higher serious adverse events in the anifrolumab group.

Subcutaneous anifrolumab for SLE met its primary BICLA endpoint at 52 weeks and significantly increased DORIS-defined remission and low disease activity.

Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Lupus

Phase 3 Trial Supports Subcutaneous Anifrolumab in Systemic Lupus Erythematosus

Welcome to The HCPFive, your go-to roundup for the latest healthcare news and breakthroughs, curated specifically for busy healthcare professionals.

Each week, we highlight 5 key developments or headlines from healthcare that you need to know—whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include the US Food and Drug Administration (FDA) approvals of caplacizumab-yhdp (Cablivi) for pediatric patients ≥ 12 years of age with acquired thrombotic thrombocytopenic purpura (aTTP) and Fibrinogen (Human) Lyophilized Powder for Reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency, Priority Review designations for a BLA atacicept for the treatment of adults with IgA nephropathy (IgAN) and an sBLA for teplizumab-mzwv to expand the age indication from ≥8 years to ≥1 year, aiming to delay the onset of stage 3 type 1 diabetes, and Breakthrough Therapy Designation for pemvidutide for the treatment of metabolic dysfunction-associated steatotic liver disease (MASH).

With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here’s your quick dive into the top stories for the week of January 4, 2025—let’s jump in!

FDA Approves Caplacizumab-yhdp (Cablivi) as First Pediatric aTTP Therapy

On January 5, 2026, the FDA approved Sanofi’s caplacizumab-yhdp (Cablivi) for injection to treat pediatric patients ≥ 12 years of age with aTTP in combination with plasma exchange and immunosuppressive therapy. The decision builds upon caplacizumab’s initial 2019 approval for adults with aTTP and was supported by efficacy data from a retrospective chart review study of 30 patients 2-18 years of age showing 80% of patients achieved clinical remission. Of note, the approval made caplacizumab the first treatment for pediatric aTTP.

Pemvidutide Gets Breakthrough Therapy Designation for MASH

On January 5, 2026, the FDA granted Breakthrough Therapy Designation (BTD) to Altimmune’s pemvidutide for the treatment of MASH based on 24-week data from the phase 2b IMPACT trial demonstrating statistically significant MASH resolution without worsening of fibrosis and early and substantial improvements in liver fat and non-invasive tests (NITs) of fibrosis and hepatic inflammation.

Altimmune has aligned with the FDA on the parameters for a planned registrational phase 3 trial of pemvidutide for MASH patients with moderate to advanced liver fibrosis evaluating multiple pemvidutide doses over a 52-week treatment period and incorporating biopsy-based endpoints to support a potential accelerated approval and the use of AIM-MASH AI Assist, the first AI pathology tool qualified by the FDA for use in MASH clinical trials.

FDA Grants Priority Review Of Biologics License Application For Atacicept In IgA Nephropathy

On January 7, 2026, the FDA granted priority review to Vera Therapeutics’ Biologics License Application (BLA) for atacicept for the treatment of adults with IgAN. The BLA submission was supported by data from the phase 3 ORIGIN 3 trial’s prespecified interim analysis showing the trial met its primary endpoint for reduction in proteinuria at week 36, with a 46% reduction compared to baseline and a 42% reduction compared to placebo (

FDA Approves 2-Gram Presentation of Fibryga for Acquired Fibrinogen Deficiency

On January 7, 2026, the FDA approved a 2-gram presentation of Octapharma’s Fibrinogen (Human) Lyophilized Powder for Reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency. Initially approved as a 1-gram single-dose bottle in 2024, Fibryga is the first and only virus-inactivated, human plasma-derived fibrinogen concentrate approved for AFD in the US. The updated kit also contains 100 mL of Water for Infusion and continues to feature the nextaro reconstitution device.

FDA Accepts for Priority Review sBLA for Teplizumab for Children With Stage 2 T1D

On January 5, 2026, the FDA accepted for priority review the supplemental biologics license application (sBLA) for Sanofi’s teplizumab-mzwv to expand the age indication from ≥8 years to ≥1 year, aiming to delay the onset of stage 3 type 1 diabetes. The sBLA submission was based on positive interim one-year data from the ongoing PETITE-T1D phase 4 study, a 2 year single-arm, open-label, multicenter study of children <8 years with stage 2 T1D.

The FDA has approved caplacizumab-yhdp (Cablivi) for pediatric patients with aTTP, marking the first treatment for this age group. Pemvidutide received Breakthrough Therapy Designation for MASH, with promising phase 2b trial results. Atacicept's BLA for IgA nephropathy was granted priority review, supported by significant proteinuria reduction data. Fibryga's 2-gram presentation was approved for acquired fibrinogen deficiency, enhancing treatment options. Teplizumab's sBLA was accepted for priority review to expand its use in younger children with stage 2 type 1 diabetes, based on positive interim study data.

Stay updated with the latest healthcare breakthroughs, including FDA approvals and other regulatory updates, in this week’s essential news roundup.

Hepatology

Nephrology

The HCPLive nephrology page is a resource for medical news and expert insights on kidney disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments in nephrology, and more.

Endocrinology

The HCPLive endocrinology page is a comprehensive resource for clinical news and insights on endocrine system conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for diabetes, hormonal disease, and more.

The HCPFive: Top News for Healthcare Providers from the Week of 01/04

Robert S. Rosenson, MD is a professor of cardiology at the Icahn School of Medicine at Mount Sinai.

Evinacumab May Lower Triglycerides in Severe Hypertriglyceridemia, With Robert Rosenson, MD

Evinacumab may be capable of lowering triglyceride concentrations in patients with 

 and a history of sHTG-associated acute pancreatitis (AP), according to a recent phase 2b study.

Evinacumab is a recombinant human monoclonal antibody which binds to and inhibits angiopoietin-like protein 3 (ANGPTL3), which leads to a reduction in LDL-C, HDL-C, and triglycerides. It reduces LDL-C irrespective of the presence of LDL receptors by promoting very low-density lipoprotein processing and clearance upstream of LDL formation.

“We know that individuals who have had episodes of pancreatitis are highly susceptible to having a recurrent hospitalization for pancreatitis in the next year. For these patients, the incident rate increases 24-fold,” Robert Rosenson, MD, professor of medicine and director of cardiometabolic disorders at the Icahn School of Medicine at Mount Sinai, said in an interview with 

. “These patients require our prompt attention with therapies that effectively lower triglycerides.”

Rosenson and colleagues conducted a phase 2b, randomized, double-blind, placebo-controlled study consisting of 3 periods – a screening period lasting ≥4 weeks, a double-blind treatment period of 52 weeks, and an off-drug safety follow-up of 20 weeks. Patients were eligible for inclusion if they had ≥1 of the following within 24 months prior to screening:

Fasting serum triglycerides >880 mg/dL with a history of 1 episode of HTG-associated AP

Fasting serum triglycerides >500 mg/dL with a history of ≥2 episodes of HTG-associated AP

Fasting serum triglycerides >500 mg/dL with a documented fasting triglyceride level of >1000 mg/dL and a history of ≥1 episode of HTG-associated AP

All patients were also required to be taking stable doses of lipid-lowering therapy for ≥8 weeks prior to the screening visit. Patients were excluded if they had FCS due to biallelic loss-of-function variants in LPL, if they had been hospitalized for AP within 4 weeks of screening, or if they had an AP episode within 12 weeks of screening.

Eligible patients were randomly assigned in a 1:1 ratio to intravenous evinacumab 20 mg/kg or matching placebo every 4 weeks for the duration of the 52-week treatment period. The study’s primary efficacy endpoint was the proportion of patients with ≥1 positively adjudicated episode of AP during this period. Key secondary endpoints included the percentage change from baseline to week 52 in fasting triglyceride and ApoC3 levels, as well as percentage change from baseline to week 52 in other lipid parameters like total cholesterol and non-HDL-C.

A total of 21 patients were enrolled and randomized to evinacumab (n = 11) or placebo (n = 10). However, the trial was terminated early by the sponsor. 20 patients did not complete the treatment period due to early termination (n = 16), adverse events (n = 2), patient decision (n = 1), and being lost to follow-up (n = 1). The 16 patients who discontinued due to the end of the trial terminated their treatments between days 1 and 169. Ultimately, 57.1% (n = 12) of patients completed the end-of-study visit.

The proportion of patients with ≥1 positively adjudicated AP episode during the treatment period was 27.3% (n = 3) with evinacumab versus 10% (n = 1) with placebo. All positively adjudicated AP episodes took place ≥58 days after the final dose, at which point evinacumab concentrations were near or below the limit of quantitation. Rosenson and colleagues also saw a median percentage change in triglycerides of -55.3% with evinacumab versus +1.5% with placebo. By week 16, evinacumab had reduced triglycerides by 95.7%, an effect which was maintained in 1 patient who received treatment through week 52.

Ultimately, although the sample size was too small to adequately determine whether it could prevent AP, investigators determined that the data gathered before the trial’s termination indicated evinacumab’s potential efficacy in lowering triglyceride concentrations in patients with sHTG and a history of sHTG-associated AP.

“I think this trial should encourage other pharmaceutical companies that have angiopoietin-like 3 inhibitors to explore this hypothesis with their inhibitors,” Rosenson said. “I’m not sure if this is going to be done with evinacumab again, but there are other RNA inhibitors of angiopoietin-like 3 that are in development by other companies, and this suggests that those inhibitors will have a profound effect on triglycerides and, hopefully, on the risk of acute pancreatitis.”

Rosenson RS, George RT, Sanchez RJ, et al. Efficacy of evinacumab in patients with severe hypertriglyceridemia and a history of severe hypertriglyceridemia-related acute pancreatitis: A phase 2B trial. Journal of Clinical Lipidology. Published online July 2025. 

Dingman R, Bihorel S, Gusarova V, Mendell J, Pordy R. Evinacumab: Mechanism of action, clinical, and translational science. 

Rosenson explains the data gained from a terminated phase 2b trial, highlighting evinacumab’s potential as a triglyceride-lowering therapy.

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Dyslipidemia

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

In this analysis, the 12-month use of a home-use helmet-type LLLT device was evaluated among individuals with androgenetic alopecia (AGA).

Alopecia

Dermatology

The HCPLive dermatology page is a resource for medical news and expert insights on skin disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for dermatologic conditions, and more.

12 Months of Low-Level Laser Therapy at Home Improves Androgenetic Alopecia

This segment looks ahead to remaining challenges in FCS diagnosis and access, and to the broader implications of APOC3 inhibition for hypertriglyceridemia management.

Video Series

Perspectives on Ongoing Unmet Need and the Future of FCS Care

This segment emphasizes the clinical and quality‑of‑life value of achieving deep triglyceride reductions, particularly below 500 mg/dL, in FCS.

Importance of Reducing Triglycerides in FCS Care

This segment addresses practical considerations in choosing between newly approved APOC3 inhibitors for patients with FCS. 

Embracing Newly FDA Approved Therapies for FCS Management

This segment explores the mechanisms of APOC3‑targeting agents and compares the triglyceride‑lowering profiles of olezarsen and plozasiran in FCS.

Assessing Therapeutic Options in FCS Management

This segment frames the longstanding unmet clinical need in FCS and the limitations of traditional management strategies.

Contextualizing Historic Unmet Need in FCS in a Contemporary Landscape

An 18-month study of children with up to 5 nut allergies found that a 30-mg per-nut maintenance dose significantly improved tolerated doses without serious adverse events.

Allergy

Allergy condition center page is a comprehensive resource for clinical news and insights on allergic and inflammatory diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for food and seasonal allergies, as well as allergic asthma and related conditions.

Anaphylaxis

The HCPLive anaphylaxis page is a comprehensive resource for clinical news and insights on acute allergic reactions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for preventing anaphylaxis, and more.

Very Low-Dose OIT Safely Increases Multi-Nut Tolerance in Young Children

Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

A prospective study reports an association between diabetes mellitus and anuria with high post-void residual post-transplantation. 


Chronic Kidney Disease

Diabetes, Anuria May Predict High Postvoid Residual After Kidney Transplantation

Laura Wingate is the Chief Education, Support & Advocacy Officer at the Crohn's & Colitis Foundation.

Wingate reviews new Crohn’s and Colitis Foundation data on insurance barriers faced by young adults with IBD and offers practical insights for overcoming these challenges.

Gastroenterology

Gastroenterology condition center page is a comprehensive resource for clinical news and insights on digestive and GI conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for 

, IBS and IBD, Crohn's disease, and more.

Inflammatory Bowel Disease

On the HCPLive inflammatory bowel disease page, resources on the topics of medical news and expert insight into IBD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on IBD and more.

Strategic Alliance Partnership

Bridging the Insurance Gap in Transition-Age IBD Care, With Laura Wingate

Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

Topline 52 week results show durable growth without compromising safety or tolerability, as well as benefits beyond linear growth with combination treatment.

Latest News

Q4 2025 Recap: Hematology News and Updates

New Topline Phase 3 Data on Zasocitinib in Psoriasis, With Melinda Gooderham, MD

Phase 3 Trial Supports Subcutaneous Anifrolumab in Systemic Lupus Erythematosus

The HCPFive: Top News for Healthcare Providers from the Week of 01/04

Evinacumab May Lower Triglycerides in Severe Hypertriglyceridemia, With Robert Rosenson, MD