On December 14, 2021 Ascendis Pharma A/S (Nasdaq: ASND) reported to provide an update on two of its investigational endocrinology rare disease development programs, TransCon PTH for hypoparathyroidism, and TransCon CNP for achondroplasia, and on one of its investigational oncology product candidates, TransCon TLR7/8 Agonist at the Company’s virtual R&D Program Update (Press release, Ascendis Pharma, DEC 14, 2021, View Source [SID1234597057]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Today marks an important milestone for Vision 3×3, our strategic roadmap for achieving sustainable growth and building a leading global biopharma company. In addition to providing updates on our Endocrinology Rare Disease pipeline, we are also presenting the first early-stage clinical data from our second therapeutic area, Oncology," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "I am very encouraged that TransCon TLR7/8 Agonist is so far performing as designed, demonstrating early signs of anti-tumor activity with a well-tolerated safety profile as monotherapy or in combination with pembrolizumab."

Ascendis’ management outlined continued clinical advancement on each of the three rare disease development programs. Highlights include:

Update from ongoing TransCon PTH development program:
Reviewed clinical data from ongoing Phase 2 PaTH Forward Trial
Topline results from the Phase 3 PaTHway Trial expected in Q1 2022
Planned NDA submission in Q3 2022 for the treatment of adults with hypoparathyroidism
Topline results from the PaTHway Japan Trial expected in Q3 2022
Planned MAA submission in Q4 2022 for the treatment of adults with hypoparathyroidism
Initiation of the pediatric hypoparathyroidism program planned for Q4 2022

Update from ongoing TransCon CNP development program:

Interim Phase 2 ACcomplisH Trial Update
ACcomplisH enrollment complete (N=57)
Preliminary PK data demonstrated that TransCon CNP provided continuous and dose-dependent exposure with a half-life of ~110 hours mirroring Phase 1 data
Well tolerated in children with achondroplasia with up to 65 weeks follow-up, with mean orthostatic vital signs unchanged
Interim blinded data from the ACcomplisH Trial informed dose selection of 50 and 100 µg/kg/week for the ACcomplisH China Trial
ACcomplisH Infants Trial (0-2 years of age) IND or equivalent submission planned for Q2 2022
ACcomplisH Trial topline unblinded data anticipated in Q4 2022
Interim update from ongoing TransCon TLR7/8 Agonist first-in-human trial (transcendIT-101):
Early signs of clinical activity in three out of three efficacy-evaluable cancer patients treated with TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab
PK data indicate sustained release of active drug with a half-life of ~7 days and low systemic exposure providing a wide safety margin
Consistent and robust immune activation in tumor tissue was observed for at least 7 days post dose
In the safety-evaluable population observed to date (n=8), TransCon TLR7/8 Agonist was well-tolerated with no dose-limiting toxicities or drug-related systemic side effects; the only related adverse events reported were transient, mild injection site-related reactions (Grade 1/2)
transcendIT-101 dose expansion expected to start enrollment in Q2 2022
Virtual R&D Program Update Conference Call & Webcast information

Date Tuesday, December 14, 2021
Time 9:00 a.m. to 11:30 a.m. Eastern Time
Dial In (U.S.) 877-870-9135
Dial In (International) 646-741-3167
Access Code 2169055
A live webcast of the event will be available on the Investors & News section of the Ascendis Pharma website at View Source A webcast replay will be available on the site shortly after conclusion of the event and will stay available for 30 days.