- 77.6% of patients treated with palopegteriparatide in the PaTHway China Trial achieved the primary multi-component endpoint compared to 0.0% for placebo (p <0.0001)
- Palopegteriparatide was generally safe and well-tolerated, with no discontinuations related to study drug.
SHANGHAI, China I August 12, 2024 I VISEN Pharmaceuticals, an innovative biopharmaceutical company focused on endocrine diseases, today announced topline data from the 26-week randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway China Trial of Palopegteriparatide (TransCon PTH) in adults with chronic hypoparathyroidism. A statistically significantly higher proportion of patients treated with palopegteriparatide achieved the primary multi-component endpoint compared to placebo.
The primary multi-component endpoint of PaTHway China – defined as serum calcium levels in the normal range (8.3–10.6 mg/dL or 2.07-2.64 mmol/L) and independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium), with no increase in prescribed study drug within the four weeks prior to the Week 26 visit – was achieved by 77.6% of palopegteriparatide-treated patients (45 of 58), compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001). Results were consistent with those announced by Ascendis Pharma for its palopegteriparatide Phase 3 trial.
The clinical trial’s principal investigator, Professor XIA Weibo, Director of the Department of Endocrinology of Peking Union Medical College Hospital, said: “Hypoparathyroidism is the last endocrine deficiency state, where hormone replacement has not been established as the standard of care. The results of this trial suggest that parathyroid hormone (PTH) replacement therapy can effectively improve symptoms caused by blood calcium fluctuations, and facilitate attainment of normal serum calcium levels without concern for exacerbating hypercalciuria. In fact, treatment with TransCon PTH lowered urine calcium into the normal range. The dual effects of providing physiologic PTH while eliminating reliance on oral calcium supplements and active vitamin D for most treated patients may reduce the risks of long-term complications associated with conventional therapy, including soft-tissue calcifications and decreased kidney function. I believe the PTH replacement therapy could significantly improve the health and quality of life of patients living with hypoparathyroidism.”
Highlights of the Phase 3 PaTHway China Trial
In the trial, 81 patients with hypoparathyroidism were randomized 3:1 to palopegteriparatide or placebo. All patients were on conventional therapy at the start of the trial. Both treatment arms underwent titration of conventional therapy and study drug (palopegteriparatide or placebo) according to an algorithm guided by serum calcium and intended to facilitate independence from conventional therapy.
Primary Multi-component Endpoint:
- 77.6% of palopegteriparatide-treated patients (45 of 58) achieved the primary multi-component endpoint compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001).
Other Selected Data:
- Palopegteriparatide treatment resulted in statistically significant improvement in patient-reported disease-specific physical symptoms compared to placebo, as shown on Hypoparathyroidism Patient Experience Scales (HPES) Symptom-Physical domain scores (p-value = 0.022).
- At Week 26, 89.7% of palopegteriparatide-treated patients (52 of 58) were able to achieve independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium).
Safety Summary:
- In the trial, palopegteriparatide was generally safe and well tolerated, with no discontinuations related to study drug.
- No serious TEAEs related to study drug were reported in the palopegteriparatide arm or placebo group.
- Palopegteriparatide significantly decreased 24-hour urinary calcium compared with placebo group. While palopegteriparatide-treated patients decreased their mean 24-hour urine calcium from 313 mg/day at baseline to 165 mg/day (normal ≤250 mg/day) at Week 26, mean 24-hour urine for placebo-treated patients was 300 mg/day at baseline and 253 mg/day at Week 26.
Following an initial blinded 26-week study period of PaTHway China, 76 patients completing the blinded period opted to receive treatment with palopegteriparatide in the ongoing open-label extension portion of the study for up to three years.
“When PTH is missing, the body is not able to function normally, and patients may experience a range of severe and potentially life-threatening short-term symptoms and long-term complications.” Pony LU, CEO and Executive Director of VISEN Pharmaceuticals, said: “VISEN looks forward to bringing new treatment options for Chinese patients as quickly as possible to help them achieve normal quality of life. These positive results bring hope for a new potential treatment option for adult patient with hypoparathyroidism in China. We plan to file New Drug Application (NDA) to the NMPA as soon as all preparation work is completed.”
About Palopegteriparatide
Palopegteriparatide (TransCon PTH) is an investigational prodrug of PTH (1-34), administered once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day.
VISEN Pharmaceuticals has been granted exclusive rights to develop, manufacture, and commercialize palopegteriparatide (TransCon PTH) in Greater China by Ascendis Pharma. Palopegteriparatide has been granted marketing authorization as a PTH replacement therapy indicated for the treatment of adults with chronic HP by the European Commission and Medicines and Healthcare products Regulatory Agency. Its NDA for palopegteriparatide for adults with hypoparathyroidism is under review by the U.S. FDA.
About VISEN Pharmaceuticals
VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients’ need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. Our goal is to enable Chinese endocrine patients to benefit from the world’s most advanced and reliable treatment solutions earlier.
For more information, please visit our website:
http://www.visenpharma.com/
Contact us:
info@visenpharma.com
SOURCE: VISEN Pharmaceuticals
Post Views: 5,637
- 77.6% of patients treated with palopegteriparatide in the PaTHway China Trial achieved the primary multi-component endpoint compared to 0.0% for placebo (p <0.0001)
- Palopegteriparatide was generally safe and well-tolerated, with no discontinuations related to study drug.
SHANGHAI, China I August 12, 2024 I VISEN Pharmaceuticals, an innovative biopharmaceutical company focused on endocrine diseases, today announced topline data from the 26-week randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway China Trial of Palopegteriparatide (TransCon PTH) in adults with chronic hypoparathyroidism. A statistically significantly higher proportion of patients treated with palopegteriparatide achieved the primary multi-component endpoint compared to placebo.
The primary multi-component endpoint of PaTHway China – defined as serum calcium levels in the normal range (8.3–10.6 mg/dL or 2.07-2.64 mmol/L) and independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium), with no increase in prescribed study drug within the four weeks prior to the Week 26 visit – was achieved by 77.6% of palopegteriparatide-treated patients (45 of 58), compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001). Results were consistent with those announced by Ascendis Pharma for its palopegteriparatide Phase 3 trial.
The clinical trial’s principal investigator, Professor XIA Weibo, Director of the Department of Endocrinology of Peking Union Medical College Hospital, said: “Hypoparathyroidism is the last endocrine deficiency state, where hormone replacement has not been established as the standard of care. The results of this trial suggest that parathyroid hormone (PTH) replacement therapy can effectively improve symptoms caused by blood calcium fluctuations, and facilitate attainment of normal serum calcium levels without concern for exacerbating hypercalciuria. In fact, treatment with TransCon PTH lowered urine calcium into the normal range. The dual effects of providing physiologic PTH while eliminating reliance on oral calcium supplements and active vitamin D for most treated patients may reduce the risks of long-term complications associated with conventional therapy, including soft-tissue calcifications and decreased kidney function. I believe the PTH replacement therapy could significantly improve the health and quality of life of patients living with hypoparathyroidism.”
Highlights of the Phase 3 PaTHway China Trial
In the trial, 81 patients with hypoparathyroidism were randomized 3:1 to palopegteriparatide or placebo. All patients were on conventional therapy at the start of the trial. Both treatment arms underwent titration of conventional therapy and study drug (palopegteriparatide or placebo) according to an algorithm guided by serum calcium and intended to facilitate independence from conventional therapy.
Primary Multi-component Endpoint:
- 77.6% of palopegteriparatide-treated patients (45 of 58) achieved the primary multi-component endpoint compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001).
Other Selected Data:
- Palopegteriparatide treatment resulted in statistically significant improvement in patient-reported disease-specific physical symptoms compared to placebo, as shown on Hypoparathyroidism Patient Experience Scales (HPES) Symptom-Physical domain scores (p-value = 0.022).
- At Week 26, 89.7% of palopegteriparatide-treated patients (52 of 58) were able to achieve independence from conventional therapy (requiring no active vitamin D and ≤600 mg/day of oral calcium).
Safety Summary:
- In the trial, palopegteriparatide was generally safe and well tolerated, with no discontinuations related to study drug.
- No serious TEAEs related to study drug were reported in the palopegteriparatide arm or placebo group.
- Palopegteriparatide significantly decreased 24-hour urinary calcium compared with placebo group. While palopegteriparatide-treated patients decreased their mean 24-hour urine calcium from 313 mg/day at baseline to 165 mg/day (normal ≤250 mg/day) at Week 26, mean 24-hour urine for placebo-treated patients was 300 mg/day at baseline and 253 mg/day at Week 26.
Following an initial blinded 26-week study period of PaTHway China, 76 patients completing the blinded period opted to receive treatment with palopegteriparatide in the ongoing open-label extension portion of the study for up to three years.
“When PTH is missing, the body is not able to function normally, and patients may experience a range of severe and potentially life-threatening short-term symptoms and long-term complications.” Pony LU, CEO and Executive Director of VISEN Pharmaceuticals, said: “VISEN looks forward to bringing new treatment options for Chinese patients as quickly as possible to help them achieve normal quality of life. These positive results bring hope for a new potential treatment option for adult patient with hypoparathyroidism in China. We plan to file New Drug Application (NDA) to the NMPA as soon as all preparation work is completed.”
About Palopegteriparatide
Palopegteriparatide (TransCon PTH) is an investigational prodrug of PTH (1-34), administered once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day.
VISEN Pharmaceuticals has been granted exclusive rights to develop, manufacture, and commercialize palopegteriparatide (TransCon PTH) in Greater China by Ascendis Pharma. Palopegteriparatide has been granted marketing authorization as a PTH replacement therapy indicated for the treatment of adults with chronic HP by the European Commission and Medicines and Healthcare products Regulatory Agency. Its NDA for palopegteriparatide for adults with hypoparathyroidism is under review by the U.S. FDA.
About VISEN Pharmaceuticals
VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients’ need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. Our goal is to enable Chinese endocrine patients to benefit from the world’s most advanced and reliable treatment solutions earlier.
For more information, please visit our website:
http://www.visenpharma.com/
Contact us:
info@visenpharma.com
SOURCE: VISEN Pharmaceuticals
Post Views: 5,637
