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Genentech's HEMLIBRA (emicizumab-kxwh) Continued to Substantially Reduce Bleeds in People with Hemophilia A with Inhibitors

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– Newly approved HEMLIBRA demonstrated superior efficacy compared to
prior treatment with bypassing agents as prophylaxis or on-demand –

– Nearly 95 percent of children who received HEMLIBRA experienced
zero treated bleeds –

Genentech, a member of the Roche Group ((SIX: RO, ROG, OTCQX:RHHBY),
announced today that new data from the ongoing HEMLIBRA®
(emicizumab-kxwh) clinical development program were presented at the
59th American Society of Hematology (ASH) Annual Meeting. These data
include longer-term results from the pivotal HAVEN 1 and HAVEN 2 studies
in people with hemophilia A with inhibitors to factor VIII, showing
once-weekly subcutaneous HEMLIBRA prophylaxis demonstrated superior
efficacy compared to prior treatment with bypassing agents (BPAs) as
prophylaxis or on-demand. These new data from the largest pivotal
studies in people with hemophilia A with inhibitors further support
HEMLIBRA as an important new treatment option for these adults,
adolescents and children.

In updated results from the HAVEN 2 study with six additional months of
data and 40 more children (younger than 12 years of age), 94.7 percent
(95 percent CI: 85.4; 98.9) of children with hemophilia A with
inhibitors who received HEMLIBRA prophylaxis had zero treated bleeds
(n=57). The intra-patient analysis comparing the effects of different
therapies in the same child (n=13) showed a 99 percent reduction in
treated bleeds with HEMLIBRA prophylaxis compared to prior treatment
with a BPA, either as prophylaxis (n=12) or on-demand (n=1). Substantial
improvements in health-related quality of life and aspects of caregiver
burden, measured by the haemophilia-specific quality of life short form
(Haemo-QoL-SF) and adapted health-related quality of life in hemophilia
patients with inhibitors (Inhib-QoL) questionnaires, were also observed
with HEMLIBRA prophylaxis compared to prior BPA prophylaxis. These data
were featured today in the official press program of the ASH Annual
Meeting.

With nearly 10 additional months of follow-up, updated results from the
HAVEN 1 intra-patient analysis of adults and adolescents showed an 88
percent (risk rate [RR]=0.12, 95 percent CI: 0.05; 0.28) reduction in
treated bleeds with HEMLIBRA prophylaxis compared to prior BPA
prophylaxis (n=24). The results also showed a 95 percent (RR=0.05, 95
percent CI: 0.02; 0.12) reduction in treated bleeds in patients who
received HEMLIBRA prophylaxis compared to prior on-demand BPA treatment
(n=24). After more than one year, substantially more patients continued
to experience zero bleeds with HEMLIBRA prophylaxis compared to their
prior prophylaxis or on-demand BPA treatment across bleed endpoints,
including treated bleeds and all bleeds. The previously reported
improvement in health status after 24 weeks, measured by the
haemophilia-specific quality of life (Haem-A-QoL) and EuroQol
5-Dimensions 5-level (EQ-5D-5L) questionnaires, was also maintained with
longer follow-up.

"These data demonstrate the continued reduction in bleeds over time with
HEMLIBRA prophylaxis and reinforce the potential of this medicine,
recently approved by the FDA for hemophilia A with inhibitors, to
redefine the standard of care," said Sandra Horning, M.D., chief medical
officer and head of Global Product Development. "We are continuing to
study HEMLIBRA in a robust clinical development program to help advance
care for all people with hemophilia A, regardless of age or inhibitor
status, and provide even less frequent dosing options."

Data from the run-in cohort of the ongoing Phase III HAVEN 4 study
showed that HEMLIBRA prophylaxis dosed once every four weeks in people
12 years of age or older with hemophilia A, with or without inhibitors,
resulted in levels of HEMLIBRA in the blood (pharmacokinetics) that were
consistent with predictions. These data supported opening the expansion
cohort of the study to further evaluate this dosing regimen. After a
median observation time of eight weeks, 85.7 percent of patients (six
out of seven) had zero bleeds while receiving HEMLIBRA prophylaxis once
every four weeks. These data follow the recent announcement that an
interim analysis of the Phase III HAVEN 4 study showed a clinically
meaningful control of bleeding in people 12 years of age or older with
hemophilia A who received HEMLIBRA prophylaxis once every four weeks.

The most common adverse events (AEs) in the HAVEN 1 and HAVEN 2 studies
at the time of these follow-up data were consistent with those observed
previously in the studies. No unexpected safety findings were observed
in the run-in cohort of the HAVEN 4 study. No new cases of thrombotic
microangiopathy (TMA) or thrombotic events were observed in HAVEN 1, and
no cases occurred in HAVEN 2 or HAVEN 4.

Based on earlier results from the HAVEN 1 and HAVEN 2 studies, HEMLIBRA
was approved by the U.S. Food and Drug Administration (FDA) for routine
prophylaxis to prevent or reduce the frequency of bleeding episodes in
adults and children with hemophilia A with inhibitors. Data from HAVEN 1
and HAVEN 2 are also being reviewed under accelerated assessment by the
European Medicines Agency (EMA) and submissions to health authorities
around the world are ongoing. The clinical development program also
includes the ongoing Phase III HAVEN 4 study and the Phase III HAVEN 3
study, which showed a statistically significant and clinically
meaningful reduction in the number of treated bleeds over time in people
aged 12 years or older with hemophilia A without inhibitors who received
HEMLIBRA prophylaxis every week or every other week, compared to those
receiving no prophylaxis.

About HAVEN 1 (NCT02622321)

HAVEN 1 is a randomized, multicenter, open-label, Phase III study
evaluating the efficacy, safety and pharmacokinetics of once-weekly
subcutaneous administration of HEMLIBRA prophylaxis compared to no
prophylaxis in adults and adolescents with hemophilia A with inhibitors
to factor VIII. The study included 113 patients (12 years of age and
older) with hemophilia A with inhibitors to factor VIII, who were
previously treated with BPAs on-demand or as prophylaxis. Patients
previously treated with on-demand BPAs were randomized in a 2:1 ratio to
receive HEMLIBRA prophylaxis (Arm A) or no prophylaxis (Arm B). Patients
previously treated with BPAs as prophylaxis received HEMLIBRA
prophylaxis (Arm C). Additional patients previously treated with
on-demand BPAs were also enrolled in a separate arm (Arm D). On-demand
treatment of breakthrough bleeds with BPAs was allowed per protocol in
all arms.

The updated HAVEN 1 intra-patient analysis data presented at ASH
comparing treatment with HEMLIBRA prophylaxis to prior BPAs as
prophylaxis or on-demand showed:

 
HAVEN 1 (NCT02622321)
Phase III randomized, multicenter, open-label study evaluating the
efficacy, safety and pharmacokinetics of HEMLIBRA prophylaxis versus
no prophylaxis in patients with hemophilia A with inhibitors to
factor VIII
Patients with hemophilia A with inhibitors aged ≥12 years on
bypassing agent(s) (BPAs) on-demand (episodic) or as prophylaxis
Endpoint   Arm A (n=24)   Arm C (n=24)
   

HEMLIBRA
prophylaxis

 

Prior episodic
BPAs

 

HEMLIBRA
prophylaxis

 

Prior prophylactic
BPAs

Median (range) duration of efficacy period (weeks)   76.3

(0.1-94.3)

  21.1

(10.6-33.9)

  75.6

(24.1-90.7)

  32.1

(8.1-49.3)

Treated bleeds
ABR

(95% CI)

  1.0

(0.4; 2.4)

  21.4

(15.2; 30.1)

  1.8

(0.7; 4.6)

  15.7

(11.2; 22.0)

% reduction

(RR [95% CI]), p-value

  95%

(0.05 [0.02; 0.12]), p<0.0001

  88%

(0.12 [0.05; 0.28]), p<0.0001

% patients with 0 bleeds

(95% CI)

  62.5

(40.6; 81.2)

  8.3

(1.0; 27.0)

  58.3

(36.6; 77.9)

  12.5

(2.7; 32.4)

% patients with 1-3 bleeds (95% CI)   33.3

(15.6; 55.3)

  25.0

(9.8; 46.7)

  25.0

(9.8; 46.7)

  16.7

(4.7; 37.4)

% of patients with ≥4 bleeds (95% CI)   4.2

(0.1; 21.1)

  66.7

(44.7; 84.4)

  16.7

(4.7; 37.4)

  70.8

(48.9; 87.4)

All bleeds
ABR

(95% CI)

  3.1

(1.6; 6.2)

  37.8

(28.6; 50.1)

  3.6

(1.9; 6.7)

  24.6

(18.4; 32.9)

% reduction

(RR [95% CI]), p-value

  92%

(0.08 [0.04; 0.17]), p<0.0001

  85%

(0.15 [0.08; 0.28]), p<0.0001

% patients with 0 bleeds

(95% CI)

  45.8

(25.6; 67.2)

  0

(0.0; 14.2)

  33.3

(15.6; 55.3)

  0

(0.0; 14.2)

% patients with 1-3 bleeds (95% CI)   25.0

(9.8; 46.7)

  4.2

(0.1; 21.1)

  29.2

(12.6; 51.1)

  16.7

(4.7; 37.4)

% of patients with ≥4 bleeds (95% CI)   29.2

(12.6; 51.1)

  95.8

(78.9; 99.9)

  37.5

(18.8; 59.4)

  83.3

(62.6; 95.3)

ABR, annualized bleeding rate; BPA, bypassing agent; NIS,
non-interventional study; RR, risk ratio

 

No new AEs resulted in treatment discontinuation. No new cases of TMA or
thrombotic events were observed. As previously reported, three people
experienced TMA events and two people experienced serious thrombotic
events in the HAVEN 1 study when on average a cumulative amount of >100
U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was
administered for 24 hours or more to patients receiving HEMLIBRA
prophylaxis.

About HAVEN 2 (NCT02795767)

HAVEN 2 is a single-arm, multicenter, open-label, clinical study in
children younger than 12 years of age with hemophilia A with inhibitors
to factor VIII. The study is evaluating the efficacy, safety and
pharmacokinetics of once-weekly subcutaneous administration of HEMLIBRA
prophylaxis.

The updated HAVEN 2 analysis after a median of nine weeks of treatment
(range 1.6-41.6 weeks) included 60 children with hemophilia A with
inhibitors to factor VIII. The updated data presented at ASH showed:

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