KLKB1

Full EAMS designation would allow treatment of patients with sebetralstat prior to receiving a Marketing Authorization from UK CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced the UK Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Promising Innovative Medicine (PIM) designation for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE). The PIM is the first step in the Early Access to Medicines Scheme (EAMS) which would allow KalVista to treat patients with sebetralstat prior to receiving a Marketing Authorisation. “We are proud to have sebetralstat designated as a Promising Innovative Medicine by the MHRA, which is similar to Expanded Access in the US,” said Ben Palleiko, Chief Executive Officer of KalVista. “Receiving the PIM designation shows that the MHRA believes that we have a promising candidate for the EAMS to treat people living with HAE.” The PIM designation also gives companies the opportunity to have early in-depth discussions with both the National Health Service (NHS) and the UK’s Health Technology Agencies. KalVista recently provided phase 3 data for sebetralstat at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, which displayed clinically and statistically significant results across all endpoints, and an excellent safety and tolerability profile. About the Promising Innovative Medicine Designation A Promising Innovative Medicine Designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS), intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition with the potential to address an unmet medical need. The designation is issued after an MHRA scientific designation meeting on the basis of non-clinical and clinical data available on the product, in a defined disease area. Following designation, the applicant is expected to complete a clinical development program within a reasonable time period, in order to continue with an application under the EAMS (step II). A designation is a prerequisite to enter the EAMS scientific opinion assessment step. About Sebetralstat Discovered by KalVista, sebetralstat is an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE). Sebetralstat has received Fast Track and Orphan Drug designations from the U.S. FDA, as well as Orphan Drug Designation and an approved Pediatric Investigational Plan from the European Medicines Agency (EMA). About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc. is a pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors for diseases with significant unmet need. KalVista disclosed positive phase 3 data for the KONFIDENT trial for its oral, on-demand therapy sebetralstat in February 2024. The Company anticipates submitting a new drug application to the U.S. FDA for sebetralstat in the first half of 2024 and expects to approval in Europe and Japan later in 2024. In addition, KalVista’s oral Factor XIIa inhibitor program represents a new generation of therapies that may further improve the treatment for people living with HAE and other diseases. For more information about KalVista, please visit . Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and their results, our ability to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2023, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. View source version on businesswire.com: Contacts KalVista Pharmaceuticals, Inc. Jarrod Aldom Vice President, Corporate Communications (201) 705-0254 jarrod.aldom@kalvista.com Ryan Baker Head, Investor Relations (617) 771-5001 ryan.baker@kalvista.com Source: KalVista Pharmaceuticals, Inc. View this news release online at:

- Phase 2b ODYSSEY Wet AMD Trial Remains on Track with Topline Data Expected in Q3 2024 - - Partner Programs Continue to Report Positive Clinical Data Utilizing SCS Microinjector® - - Strengthened Capital Position from Registered Direct Equity Offering and Recent SCS Microinjector License Agreement - - Management to Host Webcast and Conference Call Today at 4:30 P.M. ET - ALPHARETTA, Ga., March 12, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the fourth quarter and year ended December 31, 2023, and provided a corporate update. “This is an exciting time for Clearside with substantial progress in our tyrosine kinase inhibitor (TKI) program and expanded use of our injection device technology for drug delivery into the suprachoroidal space,” said George Lasezkay, Pharm.D., J.D., Clearside’s President and Chief Executive Officer. “We are laser focused on completing our ODYSSEY Phase 2b clinical trial of CLS-AX (axitinib injectable suspension) in wet AMD and expect to report topline data in the third quarter of this year.” Dr. Lasezkay continued, “Our proprietary SCS Microinjector® enables reliable, simple, in-office, non-surgical drug delivery into the suprachoroidal space targeted directly to the site of disease in the back of the eye. This trusted delivery method, combined with axitinib’s differentiated mechanism of action and high potency, offers the potential for CLS-AX to be a best-in-class product for long-term maintenance therapy for wet AMD patients. In ODYSSEY, we are looking to replicate the excellent safety profile, stable vision, and reduced frequency of injections over 6 months that we observed in our OASIS Phase 1/2a and Extension Study.” “In addition, our development and commercialization partners continue to report positive safety and efficacy results utilizing our SCS Microinjector. We are very excited to begin working closely with our newest licensing partner, BioCryst Pharmaceuticals, on their program targeting diabetic macular edema (DME) exclusively using suprachoroidal delivery of their proprietary plasma kallikrein inhibitor, avoralstat. With the upfront licensing fee from BioCryst combined with our recently completed equity financing, we expect our existing cash and cash equivalents will enable us to fund our operating expenses into the third quarter of 2025,” concluded Dr. Lasezkay. Key Highlights Completion of a registered direct offering in February 2024, which generated $15.0 million in gross proceeds to Clearside. On January 1, 2024, a new permanent Category 1 Current Procedural Terminology (CPT) code for XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use became available for physician use. Completion of participant randomization in December 2023 in ODYSSEY, Clearside’s Phase 2b clinical trial of CLS-AX using suprachoroidal delivery in neovascular age-related macular degeneration (wet AMD), with topline data expected in the third quarter of 2024. Entered into an exclusive, worldwide license with BioCryst Pharmaceuticals in November 2023 to use Clearside’s SCS Microinjector for the delivery of BioCryst’s proprietary plasma kallikrein inhibitor, avoralstat, for the treatment of DME. The terms of the agreement include an upfront license fee from BioCryst of $5.0 million and an additional $77.5 million in potential clinical, regulatory and post-approval sales-based milestone payments plus tiered mid-single digit royalties on annual global net product sales. Multiple data presentations on the use of Clearside’s suprachoroidal delivery platform were featured at prominent medical meetings, including American Academy of Ophthalmology (AAO), American Society of Retina Specialists, The Retina Society, Macula Society and Hawaiian Eye and Retina. Presentations of data on CLS-AX in wet AMD highlighted the excellent safety profile, stable vision and reduced frequency of injections observed for up to 6-months in the OASIS® Phase 1/2a clinical trial and Extension Study. Positive data was presented on the extended treatment duration of XIPERE utilizing suprachoroidal delivery. Real-world data showed excellent durability in which more than 75% of eyes did not require retreatment for 6 months after a single dose of XIPERE, supporting Clearside’s approach to extended drug release and reduced treatment burden for patients by delivering drug directly to the back of the eye via suprachoroidal administration with the SCS Microinjector. REGENXBIO’s ABBV-RGX-314 gene therapy using suprachoroidal delivery continues to be well tolerated in the Phase 2 ALTITUDE® trial for treatment of diabetic retinopathy and the Phase 2 AAVIATE® trial for treatment of wet AMD. Aura Biosciences presented positive clinical safety and efficacy updates of bel-sar for early-stage choroidal melanoma from its ongoing Phase 2 clinical trial with suprachoroidal administration. Additionally, in November 2023, Aura received FDA Agreement under a Special Protocol Assessment (SPA) for its CoMpass Phase 3 clinical trial of belzupacap sarotalocan (bel-sar) in early-stage choroidal melanoma. Aura dosed the first patient in the CoMpass trial in December 2023. Fourth Quarter 2023 Financial Results License Revenue: License and other revenue for the fourth quarter of 2023 was $6.3 million, compared to $0.3 million for the fourth quarter of 2022. The $6.0 million increase was primarily attributable to the receipt of $5.0 million in an upfront license fee from BioCryst and $1.0 million in a milestone payment from Aura Biosciences. Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2023 were $6.3 million, compared to $5.0 million for the fourth quarter of 2022. The increase was primarily due to ODYSSEY clinical trial costs. General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2023 were $2.9 million, compared to $3.2 million for the fourth quarter of 2022. The decrease was primarily due to lower insurance premiums and timing of patent-related costs. Other Expense: Non-cash interest expense for the fourth quarter of 2023 was $2.3 million, compared to $2.0 million for the fourth quarter of 2022. Non-cash interest expense was comprised of imputed interest on the liability related to the sales of future royalties and the amortization of the associated issuance costs. Net Loss: Net loss for the fourth quarter of 2023 was $4.8 million, or $0.08 per share of common stock, compared to net loss of $9.7 million, or $0.16 per share of common stock, for the fourth quarter of 2022. The decrease in net loss was primarily attributable to the receipt of $5.0 million in an upfront license fee from BioCryst in the fourth quarter of 2023. Cash Position: As of December 31, 2023, Clearside’s cash and cash equivalents totaled $28.9 million. Subsequent to the quarter end, in February 2024, Clearside completed a registered direct offering of stock and warrants which generated $15.0 million in gross proceeds. The Company believes that with the inclusion of the net proceeds from this offering, it will have sufficient resources to fund its planned operations into the third quarter of 2025. Full Year 2023 Financial Results License Revenue: License and other revenue for the year ended December 31, 2023 was $8.2 million, compared to $1.3 million for the year ended December 31, 2022. The $6.9 million increase was primarily attributable to the receipt of $5.0 million in an upfront license fee from BioCryst and $1.4 million in milestone payments from Aura Biosciences. R&D Expenses: R&D expenses for the year ended December 31, 2023 were $20.8 million, compared to $19.6 million for the year ended December 31, 2022. The increase was primarily due to ODYSSEY clinical trial costs, offset by decreases in other miscellaneous expenses. G&A Expenses: G&A expenses for the year ended December 31, 2023 were $11.9 million, compared to $11.8 million for the year ended December 31, 2022. Other Income: Other income for the year ended December 31, 2023 was $1.7 million, compared to $0.7 million for the year ended December 31, 2022. The increase was due to higher interest rates earned on cash and cash equivalents. Other Expense: Non-cash interest expense for the year ended December 31, 2023 was $9.4 million, compared to $3.3 million for the year ended December 31, 2022. Non-cash interest expense was comprised of imputed interest on the liability related to the sales of future royalties and the amortization of the associated issuance costs. Net Loss: Net loss for the year ended December 31, 2023 was $32.5 million, or $0.53 per share of common stock, compared to net loss of $32.9 million, or $0.55 per share of common stock, for the year ended December 31, 2022. Conference Call & Webcast Details Clearside’s management will host a webcast and conference call today at 4:30 p.m. Eastern Time to discuss the financial results and provide a corporate update. The live and archived webcast may be accessed on the Clearside website under the Investors section: Events and Presentations. The live call can be accessed by dialing 888-506-0062 (U.S.) or 973-528-0011 (international) and entering conference code: 694916. The Company suggests participants join 15 minutes in advance of the event. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®). Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), for the treatment of neovascular age-related macular degeneration (wet AMD), is in Phase 2b clinical testing. Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com and follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of CLS-AX, the expected timing of topline results from the ODYSSEY clinical trial, the potential benefits of CLS-AX, Clearside’s suprachoroidal delivery technology and Clearside’s SCS Microinjector® and Clearside’s ability to fund its operations into the third quarter of 2025. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC) on March 12, 2024 and Clearside’s other Periodic Reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor and Media Contacts: Jenny Kobin Remy Bernarda ir@clearsidebio.com (678) 430-8206 CLEARSIDE BIOMEDICAL, INC. Selected Financial Data (in thousands, except share and per share data) (unaudited) Statements of Operations Data Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 License and other revenue $ 6,345 $ 330 $ 8,226 $ 1,327 Operating expenses: Cost of goods sold — 204 355 204 Research and development 6,313 5,027 20,846 19,630 General and administrative 2,947 3,169 11,869 11,770 Total operating expenses 9,260 8,400 33,070 31,604 Loss from operations (2,915 ) (8,070 ) (24,844 ) (30,277 ) Other income 360 449 1,719 669 Non-cash interest expense on liability related to the sales of future royalties (2,277 ) (2,042 ) (9,360 ) (3,339 ) Net loss $ (4,832 ) $ (9,663 ) $ (32,485 ) $ (32,947 ) Net loss per share of common stock — basic and diluted $ (0.08 ) $ (0.16 ) $ (0.53 ) $ (0.55 ) Weighted average shares outstanding — basic and diluted 62,404,329 60,412,700 61,806,959 60,204,862 Balance Sheet Data December 31, December 31, 2023 2022 Cash and cash equivalents $ 28,920 $ 48,258 Total assets 34,018 51,303 Liabilities related to the sales of future royalties, net 41,988 33,977 Total liabilities 49,930 40,696 Total stockholders’ (deficit) equity (15,912 ) 10,607 Source: Clearside Biomedical, Inc.

The use of Sonrai Discovery streamlines workflows and visualisations, expediting biomarker discovery. Credit: metamorworks / Shutterstock.com. Sonrai Analytics has entered a strategic partnership with Candel Therapeutics to expedite the development of viral immunotherapies for treating cancer leveraging artificial intelligence (AI). The collaboration will add the cloud-based AI technology of Sonrai to Candel’s pipeline. The approach will aid Candel in combining and assessing clinical trial data, potentially enabling the detection of predictive biomarkers for treatment responses. Candel will employ Sonrai’s AI technology platform Sonrai Discovery to consolidate and examine data types comprising multi-omics [a new approach in which the data sets of different omic groups (molecular processes within an organism) are combined during analysis], imaging and clinical information. Sonrai Discovery will streamline workflows and visualisations, expediting the process of biomarker discovery, management and analysis by shortlisting and reporting biomarkers. See Also: Precigen files patent for insect population control using multiple lethal gene expression systems Kalvista Pharmaceuticals files patent for treatment of bradykinin-mediated angioedema with oral plasma kallikrein inhibitor The platform’s ability to aid in the examination and sharing of multi-omics data will permit Candel’s researchers to progress in the development of cancer treatments by overcoming data silos and preventing duplicated efforts. Sonrai Analytics CEO and founder professor Darragh McArt stated: “We are committed to bringing innovation through AI to our partners developing groundbreaking therapeutics to save patients’ lives. The ability to integrate multi-omics data with imaging data is a key innovation in the Sonrai platform, which allows drug developers to maximise outputs from their data and gain new insights.” Sonrai’s AI platform will be instrumental in examining multi-omics data from ongoing clinical trials targeting lung and pancreatic cancer. The integrated analysis will include possible interactions between baseline participant characteristics, post-treatment biological changes and clinical outcomes, offering a comprehensive assessment of the response to Candel’s investigational medicines. Candel Therapeutics chief scientific officer Francesca Barone stated: “At Candel, we have previously implemented AI in our enLIGHTENTM Discovery Platform. By partnering with Sonrai, we are able to expand the scope of computational biology and AI to our clinical data. “Being able to integrate multi-omics data with imaging and clinical data allows us to accelerate our goal to treat cancer patients with precision.”