An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *
Once-Weekly TransCon® CNP (Navepegritide) Receives Positive CHMP Opinion for the Treatment of Achondroplasia in Children
On October 16, Ascendis Pharma A/S announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for once-weekly TransCon CNP (navepegritide) in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed. The final European Commission (EC) decision on Ascendis Pharma’s Marketing Authorisation Application (MAA) is expected during Q4 2026.
“We are pleased to receive this positive opinion reflecting the unique profile of TransCon CNP,” said Jan Mikkelsen, president and chief executive officer at Ascendis Pharma. “With a highly favorable response from the achondroplasia community to its differentiated profile, TransCon CNP remains a foundational product in our growth disorder portfolio, positioned to support our Vision 2030 objectives.”
CHMP adopted its positive opinion following its review of the MAA for TransCon CNP, which included clinical data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.
TransCon CNP was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained.
If authorized by the EC, Ascendis expects its first EU launch of TransCon CNP under the brand name YUVIWEL® to be in Germany during Q1 2027.
About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns.
SAB BIO Receives FDA Fast Track Designation for SAB-142 in Stage 3 Type 1 Diabetes
On October 13, SAB Biotherapeutics, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAB-142, a potentially disease-modifying immunotherapy being developed for the treatment of individuals with new-onset Stage 3 type 1 diabetes.
SAB Biotherapeutics, Inc., is a clinical-stage biopharmaceutical company developing a fully human anti-thymocyte immunoglobulin (hATG) for type 1 diabetes and other autoimmune diseases.
Fast Track designation is intended to facilitate the development and expedite the review of therapies for serious conditions where there is an unmet medical need and to help bring important new treatments to patients earlier.
“Receiving FDA Fast Track designation is an important regulatory milestone for SAB-142 and reflects the agency’s determination that the program has the potential to address an unmet medical need for people diagnosed with type 1 diabetes,” said Samuel J. Reich, Chief Executive Officer of SAB BIO. “We look forward to continuing to work closely with the FDA as we advance the SAB-142 clinical program. This designation represents meaningful progress toward our goal of bringing SAB-142 to people living with type 1 diabetes as quickly as possible and transforming the meaning of a type 1 diabetes diagnosis.”
SAB-142 is currently being evaluated in the registrational Phase 2b SAFEGUARD study in people within 100 days of a Stage 3 type 1 diabetes diagnosis, with enrollment expected to be completed in Q4 2026 and topline data anticipated in 2H 2027. The program is further supported by the investigator-led Phase 3 PRISE-hATG study, which is designed to evaluate SAB-142 in people with Stage 3 type 1 diabetes from 100 days to two years following diagnosis.
Insulet’s Omnipod Now Available in the U.S.
On October 12, Insulet Corporation announced the U.S. full market release of Omnipod Discover™,a web-based data platform designed for Omnipod 5 users, their caregivers, and healthcare providers (HCPs).
Omnipod Discover turns critical diabetes data into clear visualizations and personalized insights, giving people with diabetes and their HCPs a shared understanding of therapy patterns to make more informed diabetes management decisions.
“The future of diabetes care is not just about generating more data, it’s about making that data meaningful,” said Trang Ly, MBBS, FRACP, PhD, senior vice president and chief medical officer at Insulet. “Omnipod Discover simplifies data review for Omnipod 5 users and their healthcare providers so they spend less time gathering information and more time collaborating on diabetes management.”
With Omnipod Discover, Omnipod 5 users get simple, personalized glucose and insulin delivery reports sent directly to their smartphone weekly, so they know where to focus their diabetes management efforts. Omnipod Discover also helps HCPs make confident clinical recommendations from a concise report by overlaying a licensed Ambulatory Glucose Profile (AGP) with key adjustable pump settings and bolus events.
Omnipod Discover has already been adopted in the United States by more than 15,000 Omnipod 5 users and over 1,800 healthcare professionals as part of Insulet’s limited market release program in the U. S. It is also available to users and HCPs in Spain, Australia, Saudi Arabia, Qatar, Kuwait, UAE, and Israel.
“In today’s AID (automated insulin delivery) era, clinicians need actionable insights, not more data. Omnipod Discover brings key diabetes metrics and insights together in a streamlined, easy-to-use workflow that supports clinical decision-making and patient conversations,” said Martin Sarkar, DO, ECNU, endocrinologist at EndoCARE Diabetes & Wellness Center in Frederick, Md. “It also gives people with diabetes greater visibility into their therapy, helping foster engagement and trust. Omnipod Discover is an important step toward a more connected diabetes ecosystem that supports both providers and patients.”
In June, Insulet presented Real-World Glycemic Outcomes using the Omnipod Discover platform at the American Diabetes Association’s Scientific Sessions in New Orleans, La. Use of Omnipod Discover was associated with improvements in time in range (TIR), with 32% of users seeing a clinically meaningful increase in TIR of 3% or more. These findings support the vital role that user engagement with Omnipod Discover can play in diabetes self-management.
The platform is now available to all Omnipod 5 users nationwide, with healthcare professionals able to enroll through onboarding and training provided by their dedicated Omnipod sales representative.
VeraMorph Completes Positive Pre-IND Meeting on Oral Fulvestrant Product, Receives NIH Grant
Veramorph LLC (VeraMorph) is a specialty pharmaceutical company utilizing its “degradable gel” oral drug delivery platform to develop a pipeline of clinically differentiated orally reformulated drug products. The lead product is the first effective oral formulation of fulvestrant, which is the potential best in class endocrine therapy for estrogen receptor positive (ER+) metastatic breast cancer (mBC) patients without ESR1 gene mutations.
The potential clinical benefit of orally administered fulvestrant is to prolong survival and improve compliance, which is typically correlated with better outcomes, and quality of life for ER+ mBC patients. VeraMorph’s degradable gel technology will enable this outcome by maximizing the drug’s oral bioavailability, which has already demonstrated 20 times higher maximum plasma concentrations in preclinical studies relative to the current injectable product without generating severe adverse events. The initial beneficiaries of oral fulvestrant’s improved exposure are the roughly 70% of ER+ mBC patients that retain a wild-type ESR1 gene. Several oral alternatives to fulvestrant have been approved for patients with ESR1 mutations after progression on a prior endocrine therapy (i.e., in the second line setting). In contrast, fulvestrant efficacy is independent of ESR1 mutation status. With this context, an oral fulvestrant product could capture a substantial portion of the estimated $3B to $6B oral selective estrogen receptor degrader (SERD) therapy market.
VeraMorph plans to develop a dosage strength of oral fulvestrant that achieves an equivalent area under the curve (AUC) over 28 days relative to the approved monthly injectable product. The proposed clinical strategy comprises an initial dose range finding study, a PK bridging study, and an additional safety study given the new route of administration and use of a novel excipient. During a pre-IND meeting, the FDA conveyed openness to the proposed clinical strategy that entails generating clinical data from healthy volunteers. Based on this feedback, VeraMorph expects to submit an NDA in 2029.
Using funds from an SBIR Phase I grant sponsored by the NIH, VeraMorph recently replicated results indicating non-inferior efficacy of oral fulvestrant relative to the approved injectable product in orthotopic and metastatic models of ER+ mBC. Based on these positive results, VeraMorph has received a $2.1M NIH SBIR Phase II grant in order to complete remaining IND-enabling studies on oral fulvestrant as well as PDX studies to generate additional preclinical proof of concept data. These studies will enable VeraMorph to bring oral fulvestrant into the clinic in 2027.
VeraMorph is actively raising a Seed round to finance the upcoming dose ranging study on oral fulvestrant, including the scale-up of production processes for the degradable gel technology and corresponding oral solid dosages to produce clinical trial material. These funds will enable validation of the safety and effectiveness of VeraMorph’s platform technology and the oral fulvestrant product in humans. All of these efforts are aligned with VeraMorph’s mission, which is to improve and prolong the lives of patients across all indications with cost-effective therapies by enabling the full potential of existing therapies.
Rezolute Announces Completion of Enrollment in Phase 3 upLIFT Study of Ersodetug in Tumor Hyperinsulinism
On October 14, Rezolute, Inc., a late-stage ultra-rare disease company focused on treating refractory hypoglycemia caused by any form of hyperinsulinism (HI), announced that the open-label Phase 3 study of ersodetug in tumor HI (upLIFT) is now fully enrolled and topline data are expected mid-December 2026. The company plans to continue its expanded access program for purposes of providing ersodetug on a compassionate use basis to individuals living with serious and refractory hypoglycemia due to tumor HI.
The upLIFT study is a single-arm, open-label, registrational study in 16 participants with insulinoma or non-islet cell tumors who have uncontrolled hypoglycemia caused by tumor HI. The study’s primary endpoint is the percent of participants who require intravenous glucose for refractory hypoglycemia that achieve at least a 50 percent reduction from baseline in glucose infusion rate (GIR) within the 8-week pivotal treatment phase. For statistical significance, the lower bound of the 95% confidence interval (CI) for the percent responder rate must be ≥30%, equating to a response rate of at least 56% (95% CI of 30-80%) for 16 participants.
Earlier this year, the company announced that seven of the eight participants enrolled in the first half of the study met the responder criterion for the study’s primary endpoint. Furthermore, each of these participants also achieved complete discontinuation of intravenous glucose with the administration of ersodetug. Ersodetug has been well-tolerated in the study thus far, with no drug-related safety findings reported. The eighth participant in the first half of the study elected to pursue hospice care at home shortly after enrollment, where they died one week later due to cancer progression. The Company considers this participant as a non-responder, for purposes of the primary endpoint.
“Completing enrollment in the upLIFT study marks an important milestone as we advance a potentially transformative treatment option for patients living with tumor hyperinsulinism,” said Dr. Brian Roberts, chief medical officer at Rezolute. “Now that we have a path forward for congenital hyperinsulinism, we look forward to announcing topline data from upLIFT and to the extent the study outcomes are favorable, the potential to advance both indications in 2027. We are deeply grateful to the patients, families, investigators and advocacy groups whose dedication and partnership have enabled us to reach this point.”
Efzimfotase Alfa Showed Numerical Improvement in HICKORY Phase III Trial
On October 11, Astra Zeneca announced the results from the HICKORY Phase III trial that showed that efzimfotase alfa (ALXN1850), an investigational enzyme replacement therapy, did not achieve statistical significance but demonstrated a numerical improvement in the primary endpoint of Six-Minute Walk Test (6MWT) in adolescents and adults (≥12 years of age) with hypophosphatasia (HPP) who had not been previously treated with STRENSIQ®(asfotase alfa) at week 25, compared to placebo.
In the overall population, at week 25, efzimfotase alfa achieved a least squares (LS) mean difference of 17.05 meters (p=0.1675) change from baseline in the primary endpoint of 6MWT, compared to placebo. The lack of statistical significance was largely due to better-than-expected results observed in the adult-onset HPP placebo group.
These results from the HICKORY Phase III trial signal benefits across multiple clinically relevant endpoints and represent an important step toward potentially bringing this advanced enzyme replacement therapy to a broad range of people who may benefit.
Kathryn Dahir, MD, director of the Program for Metabolic Bone Disorders at Vanderbilt Health and associate director for Clinical Research Translation, professor in the Department of Internal Medicine, Division of Endocrinology, Diabetes and Metabolism and lead investigator in the HICKORY trial
In a post hoc analysis, at week 25, 45.2% of patients treated with efzimfotase alfa achieved the minimum clinically important difference (MCID) from baseline in 6MWT (defined as 43 meters in adolescents and 31.1 meters in adults), compared to 30.0% in the placebo group (odds ratio = 2.50 [95% CI: 1.06, 5.91; nominal p=0.0363]), indicating a 2.5-fold higher odds of achieving a clinically meaningful 6MWT response.
In the overall population, at week 25, efzimfotase alfa demonstrated nominally significant improvement in the key secondary endpoint of FACIT-Fatigue, achieving an LS mean difference of 5.74 (nominal p=0.0016) vs placebo. Other key secondary endpoints numerically favored efzimfotase alfa, including 30-second Sit-to-Stand (30s-STS), Lower Extremity Functional Scale (LEFS) and BPI-SF Pain Severity.
In the open-label extension, at week 49, patients treated with efzimfotase alfa showed continued improvement across the primary and key secondary endpoints, including 6MWT, 30s-STS, LEFS, BPI-SF Pain Severity and FACIT-Fatigue, and patients switching from placebo to efzimfotase alfa following the randomized evaluation period at week 25 demonstrated similar improvement in these key measures of mobility, physical function, pain and fatigue.
In a combination of prespecified subgroups of adolescents and adults with pediatric-onset HPP, efzimfotase alfa demonstrated nominally significant and clinically meaningful benefits in the primary endpoint of 6MWT, achieving an LS mean difference of 35.6 meters (nominal p=0.0386) vs placebo at week 25, as well as the key secondary endpoints of 30s-STS (LS mean difference vs placebo: 1.3; nominal p=0.0384) and BPI-SF Pain Severity (LS mean difference vs placebo: -1.0; nominal p=0.0260). In a prespecified subgroup of adults with adult-onset HPP, efzimfotase alfa demonstrated clinically meaningful improvement in the key secondary endpoint of LEFS (LS mean difference vs placebo: 3.5; nominal p=0.2376) and nominally significant and clinically meaningful improvement in FACIT-Fatigue (LS mean difference vs placebo: 7.41; nominal p=0.0021).
Endocrine Society member Kathryn Dahir, MD, director of the Program for Metabolic Bone Disorders at Vanderbilt Health and associate director for Clinical Research Translation, professor in the Department of Internal Medicine, Division of Endocrinology, Diabetes and Metabolism and lead investigator in the HICKORY trial, said: “For adolescents and adults, the chronic and progressive disease burden of HPP can have far-reaching impact on physical function and emotional wellbeing, potentially impairing people’s ability to live independently, form connections and sustain employment. These results from the HICKORY Phase III trial signal benefits across multiple clinically relevant endpoints and represent an important step toward potentially bringing this advanced enzyme replacement therapy to a broad range of people who may benefit.”
Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, AstraZeneca Rare Disease, said: “The HICKORY Phase III trial builds on the positive results from the MULBERRY and CHESTNUT trials and demonstrates the potential of efzimfotase alfa to address the diverse and complex clinical manifestations of HPP across a broad population of patients. With the recently granted Priority Review in the US, we look forward to bringing this potential new therapy with favorable tolerability and dosing convenience to the HPP community, including those with limited treatment options.”
Pasatru™ (garetosmab-grts) Demonstrates Zero New Heterotopic Ossification (HO) Lesions in Adult Fibrodysplasia Ossificans Progressiva (FOP) Patients During OPTIMA Trial Extension
On October 12, Regeneron Pharmaceuticals, Inc., announced positive 84-week results from the Phase 3 OPTIMA trial evaluating Pasatru™ (garetosmab-grts) in adults with fibrodysplasia ossificans progressiva (FOP). These longer-term findings for Pasatru demonstrated sustained efficacy in halting new, rogue bone growth and in significantly reducing disease flare-ups, and were presented during a late-breaking oral session at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting.
Pasatru was recently approved by the U.S. Food and Drug Administration (FDA) based on a 56-week, placebo-controlled trial to reduce the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with FOP. Pasatru is a VelocImmune®-derived, fully human monoclonal antibody that binds to and inhibits signaling by the Activin A protein, which Regeneron scientists discovered to be a critical protein involved in the development of HO lesions in people with FOP.
“FOP is a devastating, relentless condition in which every flare-up can bring anxiety around potential new, abnormal bone growth and resulting loss of mobility,” said Endocrine Society member Professor Richard Keen, Director of the Metabolic Bone Disease Centre, Royal National Orthopaedic Hospital, London, and global primary investigator of the OPTIMA trial. “What patients urgently need is long-term control over new HO lesions and flare-ups to help bring peace of mind. The 84-week data from the Phase 3 OPTIMA trial demonstrate sustained disease-modifying control alongside a generally well-tolerated safety profile, which is important for the adult FOP community.”
OPTIMA, a global, multi-center, randomized, double-blind, placebo-controlled trial, enrolled 63 people with FOP aged 18 years and older. Trial participants were randomized to receive intravenously administered Pasatru 10 mg/kg, Pasatru 3 mg/kg, or placebo once every four weeks for 56 weeks. Following this, 58 participants completed the double-blind extension phase to at least 84 weeks.
“What patients urgently need is long-term control over new HO lesions and flare-ups to help bring peace of mind. The 84-week data from the Phase 3 OPTIMA trial demonstrate sustained disease-modifying control alongside a generally well-tolerated safety profile, which is important for the adult FOP community.”
Professor Richard Keen, Director of the Metabolic Bone Disease Centre, Royal National Orthopaedic Hospital, London, and global primary investigator of the OPTIMA trial
At 56 weeks, OPTIMA met its primary endpoint, demonstrating a 90% or greater reduction in new HO lesions, with a dramatic reduction in clinician-assessed flare-ups in adults with FOP. Detailed results on these 56-week data were recently published in The Lancet. The endpoints were then re-assessed at 84 weeks. From week 56, there were no new HO lesions observed at week 84 for Pasatru compared to 9 new lesions for placebo as assessed by computed tomography (CT) scan. Additionally, one new HO lesion previously detected at week 56 for Pasatru 3 mg/kg was no longer detectable at week 84. The number of new clinician-assessed flare-ups was also reduced compared to placebo, notably with 1 observed for Pasatru 10 mg/kg, 14 for Pasatru 3 mg/kg, and 17 for placebo.
Through week 84, both doses of Pasatru had a generally well-tolerated safety profile, consistent with that observed at 56 weeks. At 56 weeks, the most common adverse reactions occurring in ≥10% of adults with FOP treated with Pasatru were abscess, acne, increased hair growth, madarosis (hair loss of eyebrows or lashes), oral ulcers, epistaxis (nosebleeds), folliculitis, paronychia (nail infection), and rash. At 84 weeks, the most common treatment-emergent adverse events were acne in participants treated with Pasatru 10 mg/kg and epistaxis (all non-severe) in participants treated with Pasatru 3 mg/kg. Treatment-emergent bleeding events were lower in participants treated with Pasatru 10 mg/kg than those treated with Pasatru 3 mg/kg or placebo. There was a dose-dependent increase in skin and soft tissue infections (33% on placebo, 53% on Pasatru 3 mg/kg, and 78% on Pasatru 10 mg/kg) that were all resolved with further management, with no patients discontinuing treatment as a result of infection. No deaths were reported in the trial.
For more information, visit the Regeneron clinical trials website, contact [email protected].
