UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
_____________________
Form 8-K
_____________________
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event Reported): November 1, 2019
BioCryst Pharmaceuticals, Inc.
(Exact Name of Registrant as Specified in Charter)
| Delaware | 000-23186 | 62-1413174 |
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (I.R.S. Employer Identification Number) |
| 4505 Emperor Blvd., Suite 200, Durham, North Carolina 27703 |
| (Address of Principal Executive Offices) (Zip Code) |
(919) 859-1302
(Registrant's telephone number, including area code)
(Former name or former address, if changed since last report)
| Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions: | ||
| [ ] | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | |
| [ ] | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | |
| [ ] | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | |
| [ ] | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) | |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
| Common Stock | BCRX | Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company [ ]
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. [ ]
Item 8.01. Other Events.
On November 1, 2019, BioCryst Pharmaceuticals, Inc. (the “Company”) announced that it has begun a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by the Company for the treatment of fibrodysplasia ossificans progressiva (FOP). The Phase 1 trial will evaluate single and multiple ascending doses of oral BCX9250 in healthy volunteers. The Company expects to report the results from the trial in the second half of 2020. On November 1, 2019, the Company issued a news release announcing the events described in this Item 8.01. A copy of the news release is filed as Exhibit 99.1 hereto and is incorporated herein by reference. Forward-Looking Statements This Current Report on Form 8-K 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 the Company’s actual results, performance or achievements to be materially different from any future results, performances 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: that developing BCX9250 may take longer or may be more expensive than planned; that ongoing and future preclinical and clinical development of BCX9250 may not advance as expected, enroll the required number of subjects or have positive results; that the FDA, EMA or other applicable regulatory agency may require additional studies beyond the studies planned, may not provide regulatory clearances, may impose a clinical hold or may withhold market approval with respect to BCX9250. Please refer to the documents the Company files periodically with the Securities and Exchange Commission, specifically the Company’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 the Company’s projections and forward-looking statements.
Item 9.01. Financial Statements and Exhibits.
(d) ExhibitsExhibit No. Description 99.1 Press release dated November 1, 2019 entitled “BioCryst Begins Phase 1 Trial with BCX9250, an Oral ALK-2 Inhibitor, for Treatment of Fibrodysplasia Ossificans Progressiva”
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| BioCryst Pharmaceuticals, Inc. | ||
| Date: November 1, 2019 | By: | /s/ Alane Barnes |
| Alane Barnes | ||
| Senior Vice President and Chief Legal Officer | ||
EXHIBIT 99.1
BioCryst Begins Phase 1 Trial With BCX9250, an Oral ALK-2 Inhibitor, for Treatment of Fibrodysplasia Ossificans Progressiva
—BCX9250 significantly suppressed heterotrophic ossification in preclinical studies—
—Urgent medical need with no approved treatments for FOP —
—Phase 1 data expected in 2H 2020—
RESEARCH TRIANGLE PARK, N.C., Nov. 01, 2019 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company has begun a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva (FOP).
The Phase 1 trial will evaluate single and multiple ascending doses of oral BCX9250 in healthy volunteers. The company expects to report the results from the trial in the second half of 2020.
“BCX9250 for FOP represents the third program for potent and selective oral compounds for rare diseases invented and developed by BioCryst to enter the clinic, joining our plasma kallikrein inhibitors for hereditary angioedema and Factor D inhibitor for complement-mediated diseases,” said Dr. William Sheridan, chief medical officer of BioCryst.
“FOP is a devastating disease with no approved therapies, and we look forward to seeing clinical data with BCX9250 from this initial study in healthy volunteers to inform how we proceed strategically with the program,” Sheridan added.
In preclinical studies, BCX9250 demonstrated potency for the target kinase, selectivity, safety and strong suppression of heterotopic ossification (HO) in animal models.
About Fibrodysplasia Ossificans Progressiva
Fibrodysplasia Ossificans Progressiva is a rare, severely disabling condition characterized by the irregular formation of bone outside the normal skeleton, also known as heterotopic ossification (HO). HO can occur in muscles, tendons and soft tissue. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities and premature mortality. There are currently no approved treatments for FOP.
About BCX9250
Discovered by BioCryst, BCX9250 is a novel, oral, inhibitor of the ALK-2 enzyme. The ALK-2 enzyme is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK-2 gene are seen in all cases of FOP. An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 kinase an ideal drug target for treatment of FOP. The goal of the ALK-2 inhibitor project at BioCryst is to discover and develop orally administered kinase inhibitor drug candidates that are able to slow or prevent the progressive formation of HO.
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. BioCryst has several ongoing development programs including BCX7353, an oral treatment for hereditary angioedema; BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases; galidesivir, a potential treatment for Marburg virus disease and Yellow Fever; and BCX9250, an oral ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. 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, performances 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: that developing BCX9250 may take longer or may be more expensive than planned; that ongoing and future preclinical and clinical development of BCX9250 may not advance as expected, enroll the required number of subjects or have positive results; that the FDA, EMA or other applicable regulatory agency may require additional studies beyond the studies planned, may not provide regulatory clearances, may impose a clinical hold or may withhold market approval with respect to BCX9250. 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
Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com