recifercept (TA-46) / Pfizer - LARVOL DELTA

Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "The results contributed to the decision of terminating recifercept clinical development. This work delivers a framework that could eliminate the need for placebo in clinical trials yet provide sufficient evidence for evaluating the efficacy of an investigational drug."

Journal • P2 data • Genetic Disorders

SSR Genotyping and Marker-Trait Association with Yield Components in a Kazakh Germplasm Collection of Chickpea (Cicer arietinum L.). (PubMed, Biomolecules) - "In the studied chickpea accessions, unique alleles of the SSR loci TA14, TA46, TA76s, and TA142 were found that were not previously described by other authors...These traits showed maximal values in experiments with chickpea plants from autumn seed sowing. An analysis of the relationship between the SSR markers applied and morphological yield-related traits revealed several informative markers associated with important traits, such as plant height, height to first pod, number of branches, number of productive nodes, number of pods per plant, hundred seed weight, seed weight per plant, and seed yield."

Journal

A Study to Learn About Recifercept in Patients With Achondroplasia (clinicaltrials.gov) - P=N/A | N=250 | Completed | Sponsor: Pfizer | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=35 | Terminated | Sponsor: Pfizer | Active, not recruiting ➔ Terminated; The study was terminated due to lack of efficacy at any of the tested doses on 18th November 2022. The decision to terminate the study is not related to a safety concern.

Trial termination • Genetic Disorders

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia (clinicaltrials.gov) - P2 | N=60 | Terminated | Sponsor: Pfizer | Active, not recruiting ➔ Terminated; The study was terminated due to lack of efficacy at any of the tested doses on 18th November 2022. The decision to terminate the study is not related to a safety concern.

Trial termination • Genetic Disorders

Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Recifercept in Healthy Chinese Participants (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Pfizer | N=10 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal

"Indeed, $PFE called recifercept an FGFR3 decoy" (@JacobPlieth)

FGFR3

"$PFE recifercept FGFR specificity in vitro $BBIO $LLY $JNJ $INCY" (@JacobPlieth)

Preclinical • FGFR

"And let's not forget recifercept, also claimed to be specific for FGFR3. Project $PFE acquired through Theracon, took into ph2 for achondroplasia, then gave up in Jan 2023! $BBIO" (@JacobPlieth)

Genetic Disorders • FGFR3

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia (clinicaltrials.gov) - P2 | N=61 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2024 ➔ Mar 2023 | Trial primary completion date: Jan 2024 ➔ Jan 2023

Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Dec 2022 ➔ Mar 2023

Trial completion date • Genetic Disorders

A Study to Learn About Recifercept in Patients With Achondroplasia (clinicaltrials.gov) - P=N/A | N=250 | Active, not recruiting | Sponsor: Pfizer

New trial • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting | N=63 ➔ 30 | Trial completion date: Sep 2028 ➔ Dec 2022 | Trial primary completion date: Sep 2028 ➔ Dec 2022

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders

"También está bastante avanzado el Recifercept de Pfizer. Un FGFR3 soluble que compite con el FGFR3 mutado y parece funcionar bastante bien en modelos animales. Ya está en fase II." (@prokarionte)

FGFR3

Juvenile toxicity study of PF-07256472/recifercept, a recombinant human soluble fibroblast growth factor receptor 3, in 2-3-month-old cynomolgus monkeys. (PubMed, Birth Defects Res) - "No adverse effects were noted in this study, identifying the high dose as the no-observed-adverse-effect-level and supporting the use of recifercept in pediatric patients from birth. Considering that juvenile toxicity studies in nonhuman primates are not frequently conducted, and when they are conducted they typically utilize animals ≥9 months of age, this study demonstrates the feasibility of executing a juvenile toxicity study in very young monkeys prior to weaning."

Journal • Genetic Disorders • Pediatrics • FGFR3

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=63 | Recruiting | Sponsor: Pfizer | Trial completion date: Jun 2025 ➔ Sep 2028 | Trial primary completion date: Jun 2025 ➔ Sep 2028

Trial completion date • Trial primary completion date • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=63 | Recruiting | Sponsor: Pfizer | Trial completion date: Dec 2027 ➔ Jun 2025 | Trial primary completion date: Dec 2027 ➔ Jun 2025

Trial completion date • Trial primary completion date • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=63 | Recruiting | Sponsor: Pfizer | Trial completion date: Jan 2026 ➔ Dec 2027 | Trial primary completion date: Jan 2026 ➔ Dec 2027

Trial completion date • Trial primary completion date • Genetic Disorders

Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Recifercept in Healthy Chinese Participants (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Pfizer | Trial completion date: Jun 2022 ➔ Jun 2024 | Initiation date: Oct 2021 ➔ Oct 2023 | Trial primary completion date: Dec 2021 ➔ Dec 2023

Trial completion date • Trial initiation date • Trial primary completion date

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia (clinicaltrials.gov) - P2 | N=63 | Recruiting | Sponsor: Pfizer | Trial completion date: Apr 2023 ➔ Nov 2023 | Trial primary completion date: Apr 2023 ➔ Nov 2023

Trial completion date • Trial primary completion date • Genetic Disorders

Longitudinal Imaging of the Skull Base Synchondroses Demonstrate Prevention of a Premature Ossification After Recifercept Treatment in Mouse Model of Achondroplasia. (PubMed, JBMR Plus) - "© 2021 The Authors. JBMR Plus published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research"

Journal • Preclinical • Endocrine Disorders • Genetic Disorders • Immunology • FGFR3

Phase 2 study of long-term safety, tolerability, PK and efficacy of recifercept in achondroplasia Estudio en fase II de la seguridad, tolerabilidad, FC y eficacia a largo plazo de recifercept en la acondroplasia (clinicaltrialsregister.eu) - P2 | N=63 | Ongoing | Sponsor: Pfizer Inc.

New P2 trial • Genetic Disorders

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2 | N=63 | Recruiting | Sponsor: Pfizer | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders

Study To Evaluate The Pharmacokinetic, Safety And Tolerability Of Single Or Multiple Subcutaneous Doses of Recifercept (clinicaltrials.gov) - P1; N=24; Completed; Sponsor: Pfizer; Active, not recruiting ➔ Completed

Clinical • Trial completion

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (clinicaltrials.gov) - P2; N=63; Not yet recruiting; Sponsor: Pfizer

Clinical • New P2 trial • Genetic Disorders