Latest news with #hereditaryangioedema
Medscape
a day ago
- Health
- Medscape
EMA Backs First Oral Treatment for Hereditary Angioedema
At its July 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization for sebetralstat (Ekterly, KalVista Pharmaceuticals) for symptomatic treatment of acute attacks of hereditary angioedema in adults and adolescents aged 12 years or older. Hereditary angioedema is a rare, chronic, and potentially life-threatening genetic condition caused by deficiency or dysfunction of C1 esterase inhibitor, leading to excessive bradykinin and unpredictable swelling episodes in the face, abdomen, limbs, and airways. Sebetralstat is a selective oral plasma kallikrein inhibitor designed for administration at the earliest sign of an angioedema attack. It blocks the overactivation of the kallikrein-kinin system, preventing the formation of bradykinin, a key mediator of swelling in the condition. If approved by the European Commission, the drug will be the first oral on-demand treatment for hereditary angioedema in the European Union. The CHMP opinion was based on efficacy and safety data from a phase 3 KONFIDENT trial, which randomized 136 patients with hereditary angioedema. Findings showed that sebetralstat led to significantly faster symptom relief and reduction in attack severity and attack resolution than placebo. It was also well-tolerated with a safety profile similar to placebo. Ekterly will be available in 300-mg film-coated tablets. The most common side effect reported is headache. One of the key advantages of the drug is its oral route of administration, offering patients a more convenient alternative to injectable therapies. This may significantly reduce the time between an angioedema attack onset and treatment administration. Ekterly was granted orphan medicine status during its development due to its targeted use in a rare, life-threatening condition. The EMA noted that the drug would have been unlikely to be developed without such incentives and is now reviewing the available data to determine whether the orphan designation should be maintained.
Yahoo
10-07-2025
- Business
- Yahoo
KalVista Pharmaceuticals Provides Operational Update and Fiscal Year 2025 Financial Results
– Received FDA approval of EKTERLY® (sebetralstat)—the first and only oral on-demand treatment for hereditary angioedema; U.S. launch underway – – Six additional global regulatory submissions under review – – Entered licensing agreements for sebetralstat commercialization in Japan and Canada – – $220.6M in cash, providing runway into 2027 – CAMBRIDGE, Mass. & SALISBURY, England, July 10, 2025--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (Nasdaq: KALV), today provided an operational update and released financial results for the fiscal year ended April 30, 2025. "The FDA approval of EKTERLY represents a major milestone—not only as the first commercial product for KalVista, but more importantly, as the first and only oral on-demand therapy for people living with HAE," said Ben Palleiko, CEO of KalVista Pharmaceuticals. "EKTERLY delivers a long-awaited treatment that is safe, effective, and easy to administer. Our commercial team is actively engaging in the field, leveraging their expertise to educate and activate patients and physicians to enable timely access, drive awareness, and support informed treatment decisions around this important new therapy. With commercial partners now in place in Canada and Japan, and six global regulatory submissions under review, we believe EKTERLY is poised to become the foundational therapy for HAE management worldwide." Recent Business Highlights EKTERLY® (sebetralstat) On July 7, 2025, KalVista announced FDA approval of EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. KalVista further strengthened the robust body of clinical evidence supporting the efficacy and safety of EKTERLY (sebetralstat) for the treatment of HAE. The Company presented new data at the European Academy of Allergy and Clinical Immunology Congress 2025 (EAACI), the 14th C1-inhibitor Deficiency & Angioedema (C1-INH) Workshop in Budapest, Hungary, and the Eastern Allergy Conference (EAC) in Palm Beach, Florida. Key highlights from these medical congresses are outlined below: EAACI: Data showed the efficacy of EKTERLY (sebetralstat) for the on-demand treatment of HAE attacks among patients receiving long-term prophylaxis (LTP). Real world data highlighted significant challenges with LTP adherence, and ongoing reliance on on-demand medications. In KONFIDENT-S, EKTERLY (sebetralstat) delivered rapid, consistent relief for attacks, regardless of LTP mechanism of action with a median time to beginning of symptom relief of 1.3 hours. C1-INH: Analysis from nearly 1,600 attacks in KONFIDENT-S showed a median time to end of attack progression of 19.8 minutes. These results aligned closely with KONFIDENT, reinforcing the rapid effect of EKTERLY (sebetralstat) after absorption. Interim data from KONFIDENT-S highlighted the role of EKTERLY (sebetralstat) in treating mucosal attacks with a median time to beginning of symptom relief of 1.3 hours for both abdominal and laryngeal attacks. EAC: In KONFIDENT-S, EKTERLY (sebetralstat) was used to treat 76 attacks that had progressed to severe or very severe after a median of 2.16 hours from attack onset, demonstrating its utility in more advanced stages of HAE attacks. The median time to beginning of symptom relief for these attacks was 1.36 hours, with reduction in attack severity and substantial reduction of symptom burden in a median of 1.77 hours and 9.15 hours, respectively. Organizational In April, KalVista entered into an exclusive agreement with Kaken Pharmaceutical, Co., Ltd. to commercialize sebetralstat in Japan. Under the terms of the deal, in June 2025 KalVista received an upfront payment of $11 million, and an additional $11 million will be paid upon achieving a regulatory milestone anticipated in early 2026. This agreement also includes potential commercial milestone payments of up to $2 million and royalties based on the Japan National Health Insurance (NHI) price, with royalties expected to be in the mid-twenties as a percentage of sales. In June, KalVista granted Pendopharm, a division of Pharmascience Inc., the exclusive rights to manage the regulatory approval process and commercialization of sebetralstat in Canada. Financial Results for Fiscal Year Ended April 30, 2025: Research and development expenses were $71.7 million and $86.2 million for the fiscal years ended April 30, 2025, and 2024, respectively. The decrease in R&D was primarily attributable to reduced clinical trial expenses, preclinical activities and recognizing expense associated with EKTERLY (sebetralstat) pre-commercial awareness within General & Administrative. General and administrative expenses were $116.3 million and $54.3 million for the fiscal years ended April 30, 2025, and 2024, respectively. The increase in G&A expenses was primarily due to pre-commercial planning expenses related to EKTERLY (sebetralstat). Cash, cash equivalents and marketable securities were $220.6 million on April 30, 2025, compared to $210.4 million on April 30, 2024. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY®, the first and only oral on-demand treatment for hereditary angioedema (HAE). The Company has multiple regulatory applications under review in key global markets. For more information about KalVista, please visit and follow us on LinkedIn, X, Facebook and Instagram. 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, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, our expectations about the safety and efficacy of sebetralstat and our other product candidates, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE , 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, 2025, 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. KALVISTA PHARMACEUTICALS, INC. Consolidated Balance Sheets April 30, 2025 and 2024 (in thousands except share and per share amounts) (Unaudited) 2025 2024 Cash, cash equivalents & Marketable securities $ 220,617 $ 210,401 Other current assets 21,073 15,289 Total current assets 241,690 225,690 Other assets 9,080 9,714 Total assets $ 250,770 $ 235,404 Current liabilities $ 45,167 $ 22,807 Long-term liabilities 110,212 6,015 Total Liabilities 155,379 28,822 Stockholders' equity 95,391 206,582 Total liabilities and stockholders' equity $ 250,770 $ 235,404 KALVISTA PHARMACEUTICALS, INC. Consolidated Statements of Operations and Comprehensive Loss Years Ended April 30, 2025 and 2024 (in thousands, except share and per share amounts) (Unaudited) 2025 2024 Research and development $ 71,709 $ 86,167 General and administrative 116,286 54,278 Total operating expenses 187,995 140,445 Operating loss (187,995 ) (140,445 ) Other income: Interest income 6,435 3,896 Interest (expense) (5,785 ) — Foreign currency exchange gain (loss) 2,481 138 Other income (expenses), net 4,812 9,767 Total other income 7,943 13,801 Loss before income taxes (180,052 ) (126,644 ) Income tax (benefit) expense 3,392 — Net loss $ (183,444 ) $ (126,644 ) Net loss per share, basic and diluted $ (3.69 ) $ (3.44 ) Weighted average common shares outstanding, basic and diluted 49,652,878 36,786,575 View source version on Contacts Investors: Ryan BakerHead, Investor Relations(617) Media: Molly CameronDirector, Corporate Communications(857) Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
09-07-2025
- Business
- Yahoo
KalVista price target raised to $37 from $22 at BofA
BofA raised the firm's price target on KalVista (KALV) to $37 from $22 and keeps a Buy rating on the shares after the company announced the approval of Ekterly for on-demand treatment of hereditary angioedema attacks with a broad label and no restrictions on type of HAE attack or background LTP usage for patients 12 and older. The firm, which says the approval was in-line with its expectations and it is 'encouraged by the broad label,' remains 'bullish' on Ekterly's differentiation as the only oral on-demand treatment on the market, the analyst tells investors. Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week. Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on KALV: Disclaimer & DisclosureReport an Issue KalVista price target raised to $27 from $19 at Citizens JMP KalVista price target raised to $20 from $18 at Leerink Buy Rating for KalVista Pharmaceuticals Driven by FDA Approval and Strategic Market Entry of Ekterly KalVista price target raised to $40 from $32 at Jefferies Musk to launch new political party, Trump threatens BRICS tariff: Morning Buzz Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Medscape
07-07-2025
- Health
- Medscape
US FDA Approves KalVista's Oral Swelling Disorder Drug, Shares Rise
(Reuters) -KalVista Pharmaceuticals said on Monday the U.S. Food and Drug Administration has approved its drug, the first on-demand oral treatment for a type of hereditary swelling disorder, sending its shares up more than 18%. The FDA's decision follows an extended review of the drug last month due to heavy workloads and limited resources at the regulator. Ekterly, which offers a convenient alternative to injectable treatments, is expected to be available by mid-July, KalVista's financial chief Nicole Sweeny said. The wholesale acquisition cost for the drug will be $16,720 per dose compared to other on-demand treatments that cost between $11,000 and $17,000 per dose. Other on-demand treatments for hereditary angioedema (HAE), which affects about 8,000 people in the United States, include CSL's Berinert, Takeda's Cinryze, and Pharming's Ruconest, which are administered intravenously, as well as injectables such as Takeda's Kalbitor and Firazyr. The life-threatening condition causes sudden, dangerous swelling in the body, including the skin, digestive tract and upper respiratory system, due to deficiency in a protein known as C1 inhibitor. KalVista CEO Ben Palleiko said the company anticipates U.S. sales of "the on-demand segment of the HAE market will grow by 70% (to) $1.2 billion by 2030 ... fueled by the introduction of Ekterly." TD Cowen analyst Stacy Ku estimates peak U.S. sales of the drug at over $750 million, adding the brokerage believes "there should be high demand for Ekterly's convenience." Approval of the drug was based on a trial in which it achieved the beginning of symptom relief in 1.6 hours, while a later study showed it could treat attacks in 10 minutes, the company said. (Reporting by Puyaan Singh, Padmanabhan Ananthan and Siddhi Mahatole in Bengaluru; Editing by Arun Koyyur and Tasim Zahid)
Yahoo
07-07-2025
- Business
- Yahoo
US FDA approves KalVista Pharma's swelling disorder drug, shares rise
By Puyaan Singh (Reuters) -KalVista Pharmaceuticals said on Monday the U.S. Food and Drug Administration has approved its drug, the first on-demand oral treatment for a type of hereditary swelling disorder, sending its shares up more than 17% in premarket trading. The regulator's decision follows an extended review of the drug last month due to heavy workloads and limited resources. Ekterly, which offers a convenient alternative to injectable treatments, will be launched immediately, the company said. KalVista CEO Ben Palleiko said patients find it 'really burdensome' to carry injectable treatments, which are 'almost twice the size of an iPhone,' while Ekterly is packaged 'in a little cardboard wallet, about the size of a credit card.' Other on-demand treatments for hereditary angioedema, which affects about 8,000 people in the United States, include CSL's Berinert, Takeda's Cinryze, and Pharming's Ruconest, which are administered intravenously, as well as injectables such as Takeda's Kalbitor and Firazyr. Needham analyst Serge Belanger said KalVista previously indicated it would likely price Ekterly at a slight premium to Firazyr, the most commonly used on-demand treatment, which costs $11,000 per unit. The life-threatening condition causes sudden, dangerous swelling in the body, including the skin, digestive tract and upper respiratory system, due to deficiency in a protein known as C1 inhibitor. Citizens Bank analyst Jonathan Wolleben estimates peak U.S. sales of Ekterly at around $250 million, adding the early launch could exceed expectations due to potential premium pricing and rapid adoption. Approval of the drug was based on a trial in which Ekterly achieved the beginning of symptom relief in 1.6 hours, while a later trial showed the drug could treat attacks in 10 minutes, the company said. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
