REG - GSK PLC - FDA New Drug Application File Acceptance for MMB

GSKAnnouncement

17/08/2022 07:00

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RNS Number : 2395W  GSK PLC  17 August 2022

Issued: 17 August 2022, London UK

 

US FDA accepts New Drug Application for GSK's momelotinib for the treatment of
myelofibrosis

 

·   Regulatory submission included data from the pivotal MOMENTUM phase III
clinical trial that met all primary and key secondary efficacy endpoints

 

 

GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug
Administration (FDA) accepted the New Drug Application (NDA) for momelotinib,
a potential new medicine with a proposed differentiated mechanism of action
that may address the significant unmet medical needs of myelofibrosis patients
with anaemia. The US FDA has assigned a Prescription Drug User Fee Act action
date of 16 June 2023.

 

The NDA is based on the results from key phase III trials, including the
pivotal MOMENTUM trial, which met all primary and key secondary endpoints,
including Total Symptom Score (TSS), Transfusion Independence (TI) rate and
Splenic Response Rate (SRR). The primary analysis data from the MOMENTUM trial
were recently presented at the 2022 American Society of Clinical Oncology
Annual Meeting and the European Hematology Association 2022 Hybrid Congress.

 

Momelotinib is not currently approved in any market.

 

About the pivotal MOMENTUM phase III clinical trial

MOMENTUM is a global, randomised, double-blind phase III clinical trial of
momelotinib versus danazol in patients with myelofibrosis who were symptomatic
and anaemic and had been previously treated with an FDA-approved JAK
inhibitor. The trial was designed to evaluate the safety and efficacy of
momelotinib for treating and reducing key hallmarks of the disease: symptoms,
blood transfusions (due to anaemia) and splenomegaly (enlarged spleen).

 

The trial's primary efficacy endpoint was TSS reduction of ≥50% over the 28
days immediately before the end of Week 24 compared to baseline TSS, using the
Myelofibrosis Symptom Assessment Form. Key secondary endpoints included TI
rate for ≥12 weeks immediately before the end of Week 24 with haemoglobin
levels ≥ 8 g/dL and SRR based on splenic volume reduction of ≥35% at Week
24 from baseline.

 

Patients were randomised at 2:1 to receive either momelotinib or danazol
(n=130 and n=65, respectively). After 24 weeks of treatment, patients on
danazol were allowed to crossover to receive momelotinib. Early crossover to
momelotinib was available for confirmed splenic progression. The trial
enrolled 195 patients across 21 countries.

 

About momelotinib

Momelotinib is a potential new medicine with a differentiated mechanism of
action, with inhibitory ability along three key signalling pathways: Janus
kinase (JAK) 1, and JAK2 and activin A receptor, type I
(ACVR1). i (, ii , iii , iv ) Inhibition of JAK1 and JAK2 may improve
constitutional symptoms and splenomegaly.(i)(,)(ii)(,)(iv) Additionally,
direct inhibition of ACVR1 leads to a decrease in circulating hepcidin, which
is elevated in myelofibrosis and contributes to
anaemia.(i)(,)(ii)(,)(iii)(,)(iv)

 

Momelotinib was most recently developed by Sierra Oncology, Inc., which GSK
acquired in July 2022, building on GSK's expertise in haematology and
portfolio of specialty medicines and vaccines.

 

About myelofibrosis

Myelofibrosis is a rare blood cancer that results from dysregulated JAK-signal
transducer and activator of transcription protein signalling and is
characterised by constitutional symptoms, splenomegaly, and progressive
anaemia. Myelofibrosis affects approximately 20,000 patients in the US, with
about 40% of patients already anaemic at the time of diagnosis and nearly all
patients estimated to develop anaemia eventually.(i)(, v ) Patients will often
require transfusions, and more than 30% will discontinue treatment due to
anaemia. vi  Anaemia and transfusion dependence strongly correlate with poor
prognosis and shortened survival. vii 

 

GSK in oncology

GSK is focused on maximising patient survival through transformational
medicines. GSK's pipeline is focused on immuno-oncology, cell therapy, tumour
cell targeting therapies and synthetic lethality. Our goal is to achieve a
sustainable flow of new treatments based on a diversified portfolio of
investigational medicines utilising modalities such as small molecules,
antibodies, antibody-drug conjugates, and cell therapy, either alone or in
combination.

 

About GSK

GSK is a global biopharma company with a purpose to unite science, technology,
and talent to get ahead of disease together. Find out more at gsk.com/company
(https://www.gsk.com/en-gb/about-us/)

 

 

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 Investor Relations:  Nick Stone         +44 (0) 7717 618834   (London)
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Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made
by GSK, including those made in this announcement, are subject to risks and
uncertainties that may cause actual results to differ materially from those
projected. Such factors include, but are not limited to, those described in
the Company's Annual Report on Form 20-F for 2021, GSK's Q2 Results for 2022
and any impacts of the COVID-19 pandemic.

 

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 i  Chifotides, H.T., Bose, P. & Verstovsek, S. Momelotinib: an emerging
treatment for myelofibrosis patients with anemia. J Hematol Oncol 15, 7
(2022). https://doi.org/10.1186/s13045-021-01157-4

 ii  Verstovsek S, et al. MOMENTUM: momelotinib vs danazol in patients with
myelofibrosis previously treated with JAKi who are symptomatic and anemic.
Future Oncol. 2021;17(12):1449-1458. https://doi.org/10.2217/fon-2020-1048

 iii  Asshoff M, et al. Momelotinib inhibits ACVR1/ALK2, decreases hepcidin
production, and ameliorates anemia of chronic disease in rodents. Blood.
2017;129(13):1823-1830.

 iv  Oh S, et al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion
dependency and suppresses hepcidin in myelofibrosis phase 2 trial. Blood Adv.
2020;4(18):4282-4291.

 v  Naymagon, L., & Mascarenhas, J. (2017). Myelofibrosis-Related Anemia:
Current and Emerging Therapeutic Strategies. HemaSphere, 1(1), e1.
https://doi.org/10.1097/HS9.0000000000000001

 vi  Palandri, F., Palumbo, G.A., Elli, E.M. et al. Ruxolitinib
discontinuation syndrome: incidence, risk factors, and management in 251
patients with myelofibrosis. Blood Cancer J. 11, 4 (2021).
https://doi.org/10.1038/s41408-020-00392-1

 vii  Pardanani, A., & Tefferi, A. (2011). Prognostic relevance of anemia
and transfusion dependency in myelodysplastic syndromes and primary
myelofibrosis. Haematologica, 96(1), 8-10.
https://doi.org/10.3324/haematol.2010.035519

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