Bagsværd, Denmark, 13 May 2024 - Novo Nordisk today announced the headline
results from the FRONTIER 2 trial, a pivotal phase 3a, 26-week open-label,
randomised, controlled, multi-arm trial in 254 people. The trial investigated
the efficacy and safety of once-weekly and once-monthly subcutaneous Mim8 versus
no prophylaxis and versus prior coagulation factor prophylaxis treatment in
people aged 12 years or older with haemophilia A with or without inhibitors.
The trial achieved its co-primary endpoints by demonstrating a statistically
significant and superior reduction of treated bleeding episodes with both once-
weekly and once-monthly Mim8 versus no prophylaxis treatment and prior
coagulation factor prophylaxis treatment.
In people with no prior prophylaxis treatment, once-weekly and once-monthly Mim8
demonstrated superior reductions of 97% and 99% in treated bleeds, respectively,
compared to those who received no prophylaxis treatment. In addition, 86% of
people treated with once-weekly Mim8 and 95% of those treated with once-monthly
Mim8 experienced zero treated bleeds, compared to 0% of those treated with no
prophylaxis.
In the intra-patient analysis in people with prior coagulation factor
prophylaxis, once-weekly and once-monthly Mim8 demonstrated superior reductions
of 48% and 43% in treated bleeds, respectively, compared to prior coagulation
factor prophylaxis (during run-in period of 26-52 weeks prior to initiation of
Mim8 treatment). Additionally, 66% of people treated with once-weekly Mim8 and
65% of people treated with once-monthly Mim8 experienced zero treated bleeds.
In the trial, Mim8 appeared to have a safe and well-tolerated profile in line
with previous trials. No deaths or thromboembolic events were reported in the
trial.
"We are very pleased with the positive results from the FRONTIER 2 clinical
trial. These data demonstrate the ability of Mim8 to prevent bleeding episodes
effectively and safely in people with haemophilia A, regardless of their dosing
frequency," said Martin Holst Lange, executive vice president for Development at
Novo Nordisk. "Given the differing needs of people living with haemophilia A, a
convenient once-weekly or once-monthly dosing provides optionality and
flexibility for people living with haemophilia A with or without inhibitors."
Contingent on regulatory interactions, Novo Nordisk aims to submit Mim8 for the
first regulatory approval towards the end of 2024. Data from the phase 3
FRONTIER programme, including FRONTIER 2 will be disclosed at upcoming
congresses and in publications in 2024 and 2025.
About Haemophilia
Haemophilia is a rare inherited bleeding disorder that impairs the body's
ability to make blood clots, a process needed to stop bleeding. It is estimated
to affect approximately 1,125,000 people worldwide, and haemophilia A is
estimated to account for 80-85% of all haemophilia cases. Due to the nature of
haemophilia being a rare x-linked recessive disorder, it often presents
differently in males compared to females, with - 88% of people diagnosed with
haemophilia worldwide being male. There are different types of haemophilia,
which are characterised by the type of clotting factor protein that is defective
or missing. Haemophilia A is caused by a missing or defective clotting Factor
VIII (FVIII). Some people with haemophilia may also develop inhibitors, which
are an immune system response to the clotting factors in replacement therapy
that cause treatment to stop working. Currently, it is estimated that up to 30%
of people living with haemophilia A have inhibitors.
About Mim8
Mim8 is a next-generation FVIIIa mimetic bispecific antibody delivering
sustained haemostasis for once-weekly or once-monthly prophylaxis for people
living with haemophilia A, with and without inhibitors. Administered
subcutaneously, Mim8 bridges Factor IXa/X (FIXa/FX) together upon activation,
thereby replacing missing FVIII, which effectively restores the body's thrombin
generation capacity, helping blood to clot.
About the FRONTIER Programme
The FRONTIER clinical development programme investigates Mim8 as a preventative
treatment for people with haemophilia A, with or without inhibitors. The phase
3 programme includes:
FRONTIER 2 - a 52-week efficacy and safety phase 3 trial, with a 26-52-week run-
in period comparing once-weekly and once-monthly Mim8 versus no prophylaxis, and
versus prior coagulation factor prophylaxis treatment prior to enrolment in
people aged 12 years and over with haemophilia A, with or without inhibitors.
Following the completion of the 26-week main phase of the trial, a 26-week
extension phase is ongoing.
FRONTIER 3 - a 52-week safety and efficacy phase 3 trial in paediatric patients
with haemophilia A, with or without inhibitors (1-11 years). People will receive
once-weekly Mim8 during the first 26 weeks and may subsequently choose to
receive once-monthly Mim8.
FRONTIER 4 - an open-label extension following participation in the FRONTIER
phase 2 and phase 3 studies. The study allows the collection of long-term safety
data.
FRONTIER 5 - a 26-week phase 3 trial investigating pharmacokinetics,
pharmacodynamics and safety of switching from previous emizicumab to Mim8 in
adults and adolescents with haemophilia A, with or without inhibitors.
About Novo Nordisk
Novo Nordisk is a leading global healthcare company, founded in 1923 and
headquartered in Denmark. Our purpose is to drive change to defeat serious
chronic diseases, built upon our heritage in diabetes. We do so by pioneering
scientific breakthroughs, expanding access to our medicines, and working to
prevent and ultimately cure disease. Novo Nordisk employs about 66,000 people in
80 countries and markets its products in around 170 countries. Novo Nordisk's B
shares are listed on Nasdaq Copenhagen (Novo-B). Its ADRs are listed on the New
York Stock Exchange (NVO). For more information, visit novonordisk.com
(http://www.novonordisk.com), Facebook (http://www.facebook.com/novonordisk),
Instagram (https://www.instagram.com/novonordisk), X
(http://www.twitter.com/novonordisk), LinkedIn
(http://www.linkedin.com/company/novo-nordisk) and YouTube
(http://www.Youtube.com/novonordisk).
Contact for further information
Media:

 Ambre James-Brown                      Liz Skrbkova (US)
 +45 3079 9289                          +1 609 917 0632
 abmo@novonordisk.com                   lzsk@novonordisk.com
 (mailto:abmo@novonordisk.com)          (mailto:lzsk@novonordisk.com)

Investors:

 Daniel Muusmann Bohsen                 Jacob Martin Wiborg Rode
 +45 3075 2175                          +45 3075 5956
 dabo@novonordisk.com                   jrde@novonordisk.com
 (mailto:dabo@novonordisk.com)          (mailto:jrde@novonordisk.com)
 David Heiberg Landsted                 Mark Joseph Root (US)
 +45 3077 6915                          +1 848 213 3219
 dhel@novonordisk.com                   mjhr@novonordisk.com
 (mailto:dhel@novonordisk.com)          (mailto:mjhr@novonordisk.com)
 Sina Meyer                             Frederik Taylor Pitter
 +45 3079 6656                          +45 3075 8259
 azey@novonordisk.com                   fptr@novonordisk.com
 (mailto:azey@novonordisk.com)          (mailto:fptr@novonordisk.com)

Ida Melvold Gjøsund
+45 3077 5649
idmg@novonordisk.com
(mailto:idmg@novonordisk.com)
Company announcement No 41 / 2024
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