BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
new long-term efficacy and safety data from the APeX-2 and APeX-S
clinical trials evaluating oral, once-daily ORLADEYO®
(berotralstat) for the prophylactic treatment of hereditary
angioedema (HAE) showing sustained reductions in attack rates and
improvement in quality of life (QoL) among patients living with
HAE, and improved management of symptoms after switching to
ORLADEYO from an injectable long-term prophylactic treatment (LTP).
The data are being presented in Flash Talks at
the European Academy of Allergy and Clinical Immunology (EAACI)
Hybrid Congress 2022, which is being held live in Prague, Czech
Republic, and virtually, from July 1-3, 2022.
“From each additional analysis of our long-term
data from APeX-2 and APeX-S, we continue to see more examples of
the positive long-term impact our oral, once-daily prophylactic
therapy is having on patients with HAE. Since first reporting our
96-week data at last year’s EAACI congress, we have seen increased
evidence of sustained improvement in quality of life, reductions in
attack rates and decreased need for use of injectable on-demand
therapy. These data, coupled with the real-world evidence and
feedback we are gathering from patients and physicians, confirm our
confidence in ORLADEYO as a meaningful treatment option for
patients with HAE,” said Dr. William Sheridan, chief medical
officer of BioCryst.
“The analysis of the attack-free status of
patients with HAE who switched from injectable LTP therapy to
ORLADEYO in APeX-S builds on previous data that have shown
consistently low attack rates in ORLADEYO patients. These findings
suggest there are excellent reasons for patients to speak with
their physicians to determine if switching to an oral prophylactic
therapy could be an option to help them reduce their burden of
disease while maintaining strong efficacy. Additionally, the
analysis from APeX-2 of long-term efficacy and quality of life data
following initiation of the 150 mg dose demonstrates ORLADEYO is
having a positive impact on patients as they focus on their daily
activities,” said Teresa Caballero, M.D., Ph.D., Allergy
Department, Hospital Universitario La Paz, Madrid, Spain.
BioCryst EAACI 2022 Presentation Highlights
APeX-S was an open-label, international study
that evaluated safety and tolerability of ORLADEYO 110 mg and 150
mg in HAE patients. In APeX-S, the switch from LTP (lanadelumab) to
ORLADEYO was generally well-tolerated among patients, with no
patients in this analysis discontinuing ORLADEYO due to an adverse
event.
- Attack-free Status in
Patients who Switched from Subcutaneous Lanadelumab to Oral
Berotralstat (Poster #311); FT 3 – Flash Talks on
hereditary angioedema; Friday, July 1, 2022; 11:15am-12:45pm CET;
Room D7
- This analysis assessed the
attack-free status in all patients who switched from lanadelumab to
ORLADEYO 150 mg at U.S. sites (n=21).
- Consistently low attack rates were
observed in these patients, with median attack rates following the
switch to ORLADEYO monotherapy of 0.0 throughout 12 months of
treatment with ORLADEYO. The mean (SEM) monthly attack rate after
Month 1 was 0.1 (0.08), which was sustained through Month 6 (0.5
[0.24]) and Month 12 (0.2 [0.15]).
- Patients remained attack free an
average of 98 percent of days during treatment with ORLADEYO, with
a mean (SEM) of 144 (23.7) days and a maximum duration of 411 days
between attacks.
- These data demonstrate that
ORLADEYO is effective at maintaining good control of HAE symptoms
in patients who switch from subcutaneous LTP treatments such as
lanadelumab.
APeX-2 included 121 HAE patients who were
randomized 1:1:1 to ORLADEYO 110 mg or 150 mg, or placebo, once
daily for 24 weeks (part 1 of the study). At Week 24, patients on
ORLADEYO continued on the same dose and placebo patients were
re-randomized to ORLADEYO 110 mg or 150 mg for another 24 weeks
(part 2 of the study). At Week 48 and thereafter, all patients
continued on ORLADEYO 150 mg (open-label phase). In APeX-2,
ORLADEYO was safe and generally well tolerated, with no
drug-related serious adverse events reported.
- Improvement in Quality of
Life and Hereditary Angioedema (HAE) Attack Rates Observed in
Patients Treated with Long-term Berotralstat in the APeX-2
Study; Poster #312; FT 3 – Flash Talks on
hereditary angioedema; Friday, July 1, 2022; 11:15am-12:45pm CET;
Room D7
- This analysis stratified patients
who remained on study through at least Week 96 into three groups
according to their duration of treatment with ORLADEYO 150 mg:
patients who received ORLADEYO 150 mg for 96 weeks (n=21), patients
who received ORLADEYO 110 mg or placebo for part 1, ORLADEYO 110 mg
for part 2 and 150 mg for part 3 (n=37), and patients who received
placebo for part 1 and ORLADEYO 150 mg for parts 2 and 3 (n=12).
Quality of life (QoL) was assessed using the Angioedema Quality of
Life Questionnaire (AE-QoL), a validated tool to measure QoL
impairment in patients with recurrent angioedema. The minimal
clinically important difference (MCID) was defined as a change of
six points in total score.
- A sustained reduction in HAE attack
rates was observed across all three groups, as patients treated
with ORLADEYO 150 mg had median attack rates of 0.0 in 21 of 24
months of treatment following initiation of the 150 mg dose.
Patients also reported an overall improvement in their QoL, with
the largest improvement observed in the functioning domain. For
total AE-QoL score, a mean improvement (SEM) of 16.4 (2.79) points
from baseline to Week 96 was observed, suggesting ORLADEYO had a
positive impact on day-to-day activities.
- These data further demonstrate that
ORLADEYO is generally well tolerated and an effective prophylactic
therapy that reduces attack rates and improves QoL in patients with
HAE.
- 96 Weeks of Treatment with
Berotralstat Consistently Decreases the Use of Injectable On-Demand
Medication to Treat Hereditary Angioedema (HAE) Attacks: Analysis
from APeX-2; Poster #313; FT 24 – Flash Talks on
urticaria and angioedema II; Sunday, July 3, 2022; 5:15-6:45pm CET;
Room D6
- This analysis assessed injectable
on-demand medication use in patients who were originally randomized
to ORLADEYO 150 mg and completed 96 weeks of therapy (n=21).
- Overall, 96 weeks of treatment with
ORLADEYO 150 mg resulted in 2.4 fewer doses per month of injectable
on-demand therapy at Week 96 as compared to baseline. The median
number of attacks per month requiring treatment with injectable
on-demand medication was 0.0 attacks at all post-baseline
timepoints throughout the 96 weeks of treatment, and a similar
trend was observed with mean attack rates.
- These data suggest that use of
ORLADEYO leads to an increase in needle-free days for patients with
HAE due to the reduced need for use of injectable on-demand
medication.
ORLADEYO was safe and well tolerated in clinical
trials. The most frequently reported adverse reactions in patients
receiving ORLADEYO compared with placebo were back pain and
gastrointestinal reactions. The gastrointestinal reactions
generally occurred early after initiation of treatment with
ORLADEYO, became less frequent with time and typically
self-resolved.
About
ORLADEYO® (berotralstat)ORLADEYO® (berotralstat)
is the first and only oral therapy designed specifically to prevent
attacks of hereditary angioedema (HAE) in adult and pediatric
patients 12 years and older. One capsule of ORLADEYO per day works
to prevent HAE attacks by decreasing the activity of plasma
kallikrein.
U.S. Indication and Important Safety
Information
INDICATIONORLADEYO® (berotralstat) is a
plasma kallikrein inhibitor indicated for prophylaxis to prevent
attacks of hereditary angioedema (HAE) in adults and pediatric
patients 12 years and older.
Limitations of useThe safety
and effectiveness of ORLADEYO for the treatment of acute HAE
attacks have not been established. ORLADEYO should not be used for
the treatment of acute HAE attacks. Additional doses or dosages of
ORLADEYO higher than 150 mg once daily are not recommended due to
the potential for QT prolongation.
IMPORTANT SAFETY INFORMATIONAn increase in QT
prolongation was observed at dosages higher than the recommended
150 mg once-daily dosage and was concentration dependent.
The most common adverse reactions (≥10% and
higher than placebo) in patients receiving ORLADEYO were abdominal
pain, vomiting, diarrhea, back pain, and gastroesophageal reflux
disease.
A reduced dosage of 110 mg taken orally once
daily with food is recommended in patients with moderate or severe
hepatic impairment (Child-Pugh B or C) and in patients taking
chronically administered P-glycoprotein (P-gp) or breast cancer
resistance protein (BCRP) inhibitors (eg, cyclosporine).
Berotralstat is a substrate of P-gp and BCRP.
P-gp inducers (eg, rifampin, St. John’s wort) may decrease
berotralstat plasma concentration, leading to reduced efficacy of
ORLADEYO. The use of P-gp inducers is not recommended with
ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate
inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a
narrow therapeutic index that are predominantly metabolized by
CYP2D6 or CYP3A4, appropriate monitoring and dose titration is
recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor.
Appropriate monitoring and dose titration is recommended for P-gp
substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in
pediatric patients <12 years of age have not been
established.
There are insufficient data available to inform
drug-related risks with ORLADEYO use in pregnancy. There are no
data on the presence of berotralstat in human milk, its effects on
the breastfed infant, or its effects on milk production.
To report SUSPECTED ADVERSE REACTIONS,
contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at
1-800-FDA-1088
or www.fda.gov/medwatch.
Please see
full Prescribing
Information.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule
medicines that treat rare diseases in which significant unmet
medical needs exist and an enzyme plays a key role in the
biological pathway of the disease. Oral, once-daily ORLADEYO®
(berotralstat) is approved in the United States and multiple global
markets. BioCryst has several ongoing development programs
including BCX9930, an oral Factor D inhibitor for the treatment of
complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the
treatment of fibrodysplasia ossificans progressiva, and
galidesivir, a potential treatment for Marburg virus disease and
yellow fever. RAPIVAB® (peramivir injection) is approved in the
U.S. and multiple global markets, with post-marketing commitments
ongoing. For more information, please visit the company’s website
at www.biocryst.com.
Forward-Looking StatementsThis press release
contains forward-looking statements, including statements regarding
future results, performance or achievements. These statements
involve known and unknown risks, uncertainties and other factors
which may cause actual results, performance or achievements to be
materially different from any future results, performance or
achievements expressed or implied by the forward-looking
statements. These statements reflect our current views with respect
to future events and are based on assumptions and are subject to
risks and uncertainties. Given these uncertainties, you should not
place undue reliance on these forward-looking statements. Some of
the factors that could affect the forward-looking statements
contained herein include: the ongoing COVID-19 pandemic, which
could create challenges in all aspects of BioCryst’s business,
including without limitation delays, stoppages, difficulties and
increased expenses with respect to BioCryst’s and its partners’
development, regulatory processes and supply chains, negatively
impact BioCryst’s ability to access the capital or credit markets
to finance its operations, or have the effect of heightening many
of the risks described below or in the documents BioCryst
periodically files with the Securities and Exchange Commission;
BioCryst’s ability to successfully implement its commercialization
plans for, and to commercialize, ORLADEYO, which could take longer
or be more expensive than planned; the commercial viability of
ORLADEYO, including its ability to achieve market acceptance; the
FDA or other applicable regulatory agency may require additional
studies beyond the studies planned for products and product
candidates, may not provide regulatory clearances which may result
in delay of planned clinical trials, may impose certain
restrictions, warnings, or other requirements on products and
product candidates, may impose a clinical hold with respect to
product candidates, or may withhold, delay, or withdraw market
approval for products and product candidates; BioCryst’s ability to
successfully manage its growth and compete effectively; risks
related to the international expansion of BioCryst’s business; and
actual financial results may not be consistent with expectations,
including that revenue, operating expenses and cash usage may not
be within management's expected ranges. Please refer to the
documents BioCryst files periodically with the Securities and
Exchange Commission, specifically BioCryst’s most recent Annual
Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current
Reports on Form 8-K, which identify important factors that could
cause the actual results to differ materially from those contained
in BioCryst’s forward-looking statements.
BCRXW
Investor Contact:John Bluth+1
919 859 7910jbluth@biocryst.com
Media Contact:Catherine
Collier Kyroulis+1 917 886 5586ckyroulis@biocryst.com
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