BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported
financial results for the first quarter ended March 31, 2022, and
provided a corporate update.
“We are now over a year into the ORLADEYO launch
and are excited to see strong and continuing patient demand and
steady expansion in our prescriber base among both new and existing
prescribers. These trends continued in the first quarter of 2022
and reinforce our confidence that we will achieve no less than $250
million in net ORLADEYO revenue in 2022 and peak ORLADEYO sales of
$1 billion,” said Jon Stonehouse, president and chief executive
officer of BioCryst.
“We also have made substantial progress in our
investigation with BCX9930. Based on our initial findings, we
believe that both dose and dosing regimen could be contributing
factors to the safety signal we have observed. By the end of the
third quarter, we plan to discuss our proposed approach to resume
the REDEEM trials, under a revised dosing protocol, with
regulators,” Stonehouse added.
Program Updates and Key
Milestones
ORLADEYO®
(berotralstat): Oral, Once-daily Treatment for Prevention
of Hereditary Angioedema (HAE) Attacks
U.S. Launch
- ORLADEYO net revenue in the first
quarter of 2022 was $49.7 million.
- Approximately 50 percent of
patients currently on ORLADEYO have switched from another
prophylactic therapy, and half of those patients have come from
lanadelumab. These trends continued with new patients starting
ORLADEYO in Q1 2022.
- In the first quarter, new patient
prescriptions were evenly split between repeat prescribers and new
prescribers, with an approximately equal number of new
prescriptions coming from the top 500 HAE treaters and the broader
set of other HAE treaters.
- The company has completed its
latest quarterly survey of another 60 allergists who treat an
average of eight HAE patients. These physicians were already using
ORLADEYO on 13 percent of their patients and predicted growth to 23
percent over the next 12 months. These findings were in line with
previously reported market research from August 2021.
- Approximately 80 percent of HAE
patients in the U.S. are insured by payors and pharmacy benefit
managers that cover ORLADEYO. Despite prior authorization headwinds
early in 2022, approximately 80 percent of patients on ORLADEYO
were receiving paid product by the end of the first quarter, a
substantial increase from approximately 66 percent of patients in
the second half of 2021.
- Most patients are well-controlled
on ORLADEYO and remain on therapy. Once payor prior authorization
is complete, 78 percent of patients switching from lanadelumab and
73 percent of patients switching from subcutaneous C1 inhibitor
remain on ORLADEYO for at least six months. About 70 percent of all
patients who receive reimbursed product stay on ORLADEYO for at
least 12 months, compared to 60 percent of patients who remain on
long-term free product.
- The company expects steady
quarterly ORLADEYO net revenue growth throughout the remainder of
2022, with total 2022 ORLADEYO net revenue of no less than $250
million.
“We are very pleased that strong patient demand
continued to drive growth in the first quarter and overcome the
traditional Q1 reimbursement headwinds. With outstanding
reimbursement in place, patients continuing to enjoy an excellent
experience on ORLADEYO and the expansion of both our new and
existing prescriber bases, we expect steady growth throughout the
remainder of 2022 as we achieve no less than $250 million in
ORLADEYO net revenues for the year,” said Charlie Gayer, chief
commercial officer of BioCryst.
ORLADEYO: Global Updates
- ORLADEYO has been launched in
Denmark, France, Germany, Japan, Norway, Sweden, the United Arab
Emirates and the United Kingdom. The company expects launches in
additional countries throughout the year.
Complement Oral Factor D Inhibitor
Program – BCX9930
On April 8, 2022, BioCryst announced that the
company was voluntarily pausing enrollment in BCX9930 clinical
trials while it investigated observed elevations in serum
creatinine seen in some patients.
Patients in the REDEEM-1, REDEEM-2 and RENEW
clinical trials randomized to BCX9930 began those trials by
starting immediately at a dose of 500 mg twice-daily. Patients
receiving BCX9930 in the long-term extension trial started at lower
doses as part of the proof-of-concept dose escalation regimen and
were ultimately moved up to 500 mg twice-daily.
Preliminary evidence from the investigation
points to both the 500 mg twice-daily dosing level and the
immediate start of that dose, without a period at a lower dose
first, as plausible contributory factors for the observed increases
in serum creatinine.
Based on the initial results of the
investigation, and the safety and efficacy data observed in the
BCX9930 clinical program at 400 mg twice-daily, the company plans
to discuss with regulators whether clinical trials with amended
protocols could resume using stepped dosing to 400 mg twice-daily.
The company expects to have discussions with regulators by the end
of the third quarter.
During the ongoing investigation the company has
observed the following:
- Three patients with PNH receiving
BCX9930 in the REDEEM trials had early onset, and moderate or
severe, elevations in their serum creatinine (2-4 xULN) after
several weeks of dosing with 500 mg twice-daily. Two of these
patients have been discontinued from therapy and one patient (who
had the smallest increase in serum creatinine) continues on BCX9930
at this time.
- The company estimates that
one-third of subjects randomized to BCX9930 in the REDEEM studies
have had early increases in serum creatinine.
- The company also found a different
pattern of slowly evolving, late onset, mild to moderate increases
in serum creatinine in approximately 40 percent of patients in the
long-term extension of the proof-of-concept trial, after those
patients switched to the 500 mg twice-daily dose. This pattern was
not observed during treatment with doses lower than 500 mg.
Subsequent to BioCryst voluntarily pausing trial
enrollments, the U.S. Food and Drug Administration (FDA) informed
the company that it has placed the clinical program for BCX9930 on
a partial clinical hold. Consistent with BioCryst’s voluntary
action, the company may not enroll new patients in its BCX9930
clinical trials, however patients already enrolled who are
receiving clinical benefit from BCX9930 treatment, and have no
other available treatment options, can continue to be dosed and
remain in the trials.
“As we complete our investigation, we will
continue to be comprehensive and deliberate, with a primary focus
on patient safety. After consultation with regulators, we will
determine the next step for the BCX9930 program,” said Dr. William
Sheridan, Chief Medical Officer of BioCryst.
The company does not plan to provide additional
updates on the BCX9930 program until it completes additional
regulatory discussions and has more clarity on the next steps for
the program.
Additional Updates
- On February 8, 2022, the company
announced the appointment of Machelle Sanders to its board of
directors.
First Quarter 2022 Financial
Results
For the three months ended March 31, 2022, total
revenues were $49.9 million, compared to $19.1 million in the first
quarter of 2021 (+161.3 percent year-over-year (y-o-y)). The
increase was primarily due to $49.7 million in ORLADEYO net revenue
in the first quarter of 2022, compared to $10.9 million in ORLADEYO
net revenue in the first quarter of 2021 (+356.0 percent
y-o-y).
Research and development (R&D) expenses for
the first quarter of 2022 increased to $65.4 million from
$42.4 million in the first quarter of 2021 (+54.2 percent y-o-y),
primarily due to increased investment in the development of our
Factor D program, including BCX9930, as well as other research,
preclinical and development costs.
Selling, general and administrative (SG&A)
expenses for the first quarter of 2022 increased to $34.3 million,
compared to $22.1 million in the first quarter of 2021 (+55.2
percent y-o-y). The increase was primarily due to increased
investment to support the commercial launch of ORLADEYO and
expanded international operations.
Interest expense was $23.8 million in the first
quarter of 2022, compared to $12.9 million in the first
quarter of 2021 (+84.5 percent y-o-y). The increase was due to
service on the royalty financings, which were completed in November
2021.
Net loss for the first quarter of 2022 was $74.2
million, or $0.40 per share, compared to a net loss of $64.3
million, or $0.36 per share, for the first quarter of 2021.
Cash, cash equivalents, restricted cash and
investments totaled $446.8 million at March 31, 2022, compared to
$244.4 million at March 31, 2021. Operating cash use for the first
quarter of 2022 was $71.0 million.
Financial Outlook for
2022
Based on the strength of the ORLADEYO launch,
and continued steady growth from new patient demand anticipated
throughout the year, the company expects full year 2022 net
ORLADEYO revenue to be no less than $250 million.
The company had previously expected operating
expenses for full year 2022, not including non-cash stock
compensation, to be between $440 million to $480 million. Once the
company completes its investigation into BCX9930 and has clarity on
the next step for the program it expects to provide an updated
outlook on full year 2022 operating expenses. If BCX9930 program
enrollment resumes, then operating expenses are likely to be at the
lower end of the previously provided range. If we discontinue the
BCX9930 program, then operating expenses for the year would be
lower than that.
Conference Call and Webcast
BioCryst management will host a conference call
and webcast at 8:30 a.m. ET today to discuss the financial results
and provide a corporate update. The live call may be accessed by
dialing 877-303-8027 for domestic callers and 760-536-5165 for
international callers and using conference ID # 9498023. A live
webcast of the call and any slides will be available online at the
investors section of the company website at www.biocryst.com. A
telephone replay of the call will be available by dialing
855-859-2056 for domestic callers or 404-537-3406 for international
callers and entering the conference ID # 9498023.
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, the
European Union, Japan, the United Kingdom and the United Arab
Emirates. 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) has received
regulatory approval in the U.S., Canada, Australia, Japan, Taiwan
and Korea. Post-marketing commitments for RAPIVAB are ongoing. For
more information, please visit the company’s website
at www.biocryst.com.
Forward-Looking Statements
This 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
BioCryst’s 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 files
periodically 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 results of BioCryst’s
partnerships with third parties may not meet BioCryst’s current
expectations; risks related to government actions, including that
decisions and other actions, including as they relate to pricing,
may not be taken when expected or at all, or that the outcomes of
such decisions and other actions may not be in line with BioCryst’s
current expectations; the commercial viability of ORLADEYO,
including its ability to achieve market acceptance; the timing and
results of the ongoing investigation described in this press
release regarding BCX9930 and of any related discussions with
regulators; ongoing and future preclinical and clinical development
of BioCryst’s Factor D program, BCX9250 and galidesivir may not
have positive results; BioCryst may not be able to enroll the
required number of subjects in planned clinical trials of product
candidates; BioCryst may not advance human clinical trials with
product candidates as expected; 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; product candidates, if approved, may not achieve market
acceptance; BioCryst’s ability to successfully commercialize its
products and product candidates, 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, all of which
identify important factors that could cause the actual results to
differ materially from those contained in BioCryst’s projections
and forward-looking statements.
BCRXW
Investors:John Bluth+1 919 859
7910jbluth@biocryst.com
Media:Catherine Collier
Kyroulis+1 917 886 5586ckyroulis@biocryst.com
BIOCRYST PHARMACEUTICALS, INC. |
CONSOLIDATED FINANCIAL SUMMARY |
(in thousands, except per share) |
|
|
|
|
|
|
Statements of
Operations (Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
March 31, |
|
|
|
2022 |
|
|
|
2021 |
|
Revenues: |
|
|
|
|
|
Product sales |
|
$ |
49,546 |
|
|
$ |
17,871 |
|
Royalty revenue |
|
|
347 |
|
|
|
(897 |
) |
Collaborative and other research and development |
|
|
30 |
|
|
|
2,085 |
|
Total revenues |
|
|
49,923 |
|
|
|
19,059 |
|
|
|
|
|
|
|
Expenses: |
|
|
|
|
Cost of product sales |
|
|
236 |
|
|
|
5,923 |
|
Research and development |
|
|
65,360 |
|
|
|
42,435 |
|
Selling, general and administrative |
|
|
34,282 |
|
|
|
22,114 |
|
Royalty |
|
|
2 |
|
|
|
(36 |
) |
Total operating expenses |
|
|
99,880 |
|
|
|
70,436 |
|
|
|
|
|
|
|
Loss from operations |
|
|
(49,957 |
) |
|
|
(51,377 |
) |
|
|
|
|
|
|
Interest and other income |
|
|
54 |
|
|
|
26 |
|
Interest expense |
|
|
(23,837 |
) |
|
|
(12,904 |
) |
Foreign currency (losses)
gains, net |
|
|
(177 |
) |
|
|
(29 |
) |
Loss before income taxes |
|
|
(73,917 |
) |
|
|
(64,284 |
) |
Income tax expense |
|
|
279 |
|
|
|
- |
|
Net loss |
|
$ |
(74,196 |
) |
|
$ |
(64,284 |
) |
|
|
|
|
|
|
Basic and diluted net loss per
common share |
|
$ |
(0.40 |
) |
|
$ |
(0.36 |
) |
|
|
|
|
|
|
Weighted average shares
outstanding |
|
|
184,898 |
|
|
|
177,343 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheet
Data (in thousands) |
|
|
|
|
|
|
|
|
March 31, 2022 |
|
|
|
December 31, 2021 |
|
|
|
|
(Unaudited) |
|
|
|
(Note 1) |
|
Cash, cash equivalents and
investments |
|
$ |
443,468 |
|
|
$ |
514,430 |
|
Restricted cash |
|
|
3,354 |
|
|
|
3,345 |
|
Receivables |
|
|
36,415 |
|
|
|
29,413 |
|
Total assets |
|
|
527,720 |
|
|
|
588,151 |
|
Secured term loan |
|
|
140,236 |
|
|
|
136,082 |
|
Royalty financing
obligation |
|
|
464,225 |
|
|
|
449,375 |
|
Accumulated deficit |
|
|
(1,281,700 |
) |
|
|
(1,207,504 |
) |
Stockholders’ deficit |
|
|
(164,215 |
) |
|
|
(106,986 |
) |
Shares of common stock
outstanding |
|
|
185,572 |
|
|
|
184,350 |
|
|
|
|
|
|
|
Note 1: Derived from audited
financial statements. |
|
|
|
|
|
|
|
|
|
|
|
BioCryst Pharmaceuticals (NASDAQ:BCRX)
Historical Stock Chart
From Mar 2024 to Apr 2024
BioCryst Pharmaceuticals (NASDAQ:BCRX)
Historical Stock Chart
From Apr 2023 to Apr 2024