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): May 7, 2024
(Exact name of registrant as specified in its charter)
| (State or other jurisdiction of incorporation) | (Commission File Number) | (IRS Employer Identification Number) | ||||||||||||
(Address of principal executive offices, including Zip Code)
Registrant’s telephone number, including area code: (412 ) 586-5830
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 | KRYS | 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 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
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 7.01 Regulation FD Disclosure.
On May 7, 2024, Krystal Biotech, Inc. (the “Company”) presented a poster entitled “An HSV-1-Based Vector Platform for Localized Delivery to the Posterior of the Eye”, at the Association for Research in Vision and Ophthalmology (“ARVO”) 2024 Annual Meeting in Seattle, WA. A copy of the poster presented at the ARVO meeting is attached hereto as Exhibit 99.1 and is incorporated herein by reference. The poster is also available on the “Investors” section of the Company’s website at www.krystalbio.com.
The information in Item 7.01 of this Current Report on Form 8-K and in Exhibit 99.1 attached hereto shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section. The information in this Item 7.01 of this Current Report on Form 8-K and in Exhibit 99.1 attached hereto shall not be incorporated into any registration statement or other document filed with the Securities and Exchange Commission by the Company, whether made before or after the date hereof, regardless of any general incorporation language in such filing, except as shall be expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit No. | Description | |||||||
| 99.1 | ||||||||
| 104 | Cover Page Interactive Data file (embedded within the Inline XBRL document) | |||||||
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Date: May 7, 2024 | KRYSTAL BIOTECH, INC. | |||||||||||||||||||
| By: | /s/ Krish S. Krishnan | |||||||||||||||||||
| Name: | Krish S. Krishnan | |||||||||||||||||||
| Title: | Chairman and Chief Executive Officer | |||||||||||||||||||
An HSV-1-Based Vector Platform for Localized Delivery to the Posterior of the Eye Haley N. Cartwright, Ph.D.1; Jorge Guzman-Lepe, M.D.1; Trevor J. Parry, Ph.D.1; Suma M. Krishnan, M.S.1 1Krystal Biotech, Inc., Pittsburgh, Pennsylvania ARVO 2024 Annual Meeting PRESENTATION #3978 Purpose These studies were funded by Krystal Biotech, Inc. Krystal Biotech, Inc. would like to thank EyeCRO, Inc. for their contributions to the work presented here. All animal studies were performed in an AAALAC accredited facility, and protocols were IACUC approved prior to initiation. All authors are current employees of Krystal Biotech, Inc. 1Guide, NEJM 2022; 2Sabater, ARVO 2023; 3Vetencourt, NEJM 2024. Acknowledgements/Disclosures/ReferencesConclusions • Clinical use of a herpes simplex virus type 1 (HSV-1)-based gene therapy vector, beremagene geperpavec (B-VEC), has been successful in treating skin- and eye- related pathologies associated with dystrophic epidermolysis bullosa (DEB) 1-3. • The underlying platform technology is now being explored for its potential in treating additional genetic ocular disorders, necessitating determination of feasible routes for safe transgene delivery, particularly to the posterior of the eye. Figure 1. A single dose (1 µL) of an HSV-1-based fluorescent reporter vector was administered via subretinal, suprachoroidal, or intravitreal injection to mouse eyes. Eyes were collected after 24 hours for staining and qPCR. HSV-1-mCh = vector encoding mCherry fluorescent reporter gene. Created with BioRender.com. Methods: Routes of Administration to the Posterior of the Eye Ocular Injection Results in Minimal Inflammation in the Eye This HSV-1-based platform technology can transduce multiple clinically-relevant cell types in the eye, including both photoreceptors and RPECs in the retina, with little-to-no inflammation. These data support further development of this technology for ocular disorders, particularly inherited retinal diseases. Figure 4. Very few inflammatory cells were observed in the suprachoroidal and subretinal groups, while the intravitreal group showed mild inflammatory cell infiltration by hematoxylin and eosin (H&E) staining. The upper panels are imaged at 5x, and the lower panels are imaged at 10x. Vector Platform Successfully Transduces Both Photoreceptors and Retinal Pigment Epithelial Cells (RPECs) , as Visualized Through Co-IF 5x Figure 2. Suprachoroidal and subretinal injections resulted in disseminated mCherry expression across the retina, while intravitreal injection revealed mCherry signal in the cornea, iris, and ciliary body, as visualized through immunofluorescence (IF) staining. * = cornea Figure 3. Vector cell-type affinity in the retina after subretinal and suprachoroidal injection was assessed by co-localization of viral mCherry expression with rhodopsin (RHO; photoreceptor marker) or RPE65 (RPEC marker) fluorescent staining. DAPI was used to visualize nuclei. Suprachoroidal and Subretinal, but Not Intravitreal, Injections Result in mCherry Expression Across the Retina D AP I/m C he rr y Control 5x Intravitreal 5x Subretinal 5x Suprachoroidal 5x Control Intravitreal Subretinal Suprachoroidal 5x 5x 5x 5x 10x 10x 10x 10x Subretinal D AP I/m C he rr y R H O R PE 65 40x 40x 40x 40x 40x 40x Suprachoroidal D AP I/m C he rr y R H O R PE 65 40x 40x 40x 40x 40x 40x Ocular Injection Reveals Low Vector Dissemination into Circulation Figure 5. qPCR of murine plasma revealed low- detectable levels of vector genomes after subretinal injection; dissemination was at the limit of detection after intravitreal or suprachoroidal treatment. Vector genome copies in eye tissue after intravitreal injection in rodents (blue) is provided for comparison, demonstrating ~4-5 log-fold higher concentrations than those observed in the plasma. * * * *