Hereditary angioedema

BioCryst Presents New Real-world Data Showing Rapid, Substantial and Sustained HAE Attack Rate Reductions After Beginning ORLADEYO® (berotralstat) Treatment

Retrieved on:

Friday, February 23, 2024

“These additional analyses of real-world use of ORLADEYO show that any person living with HAE has the potential to experience a rapid, substantial and sustained reduction in their monthly attack rate with ORLADEYO.

Key Points:

“These additional analyses of real-world use of ORLADEYO show that any person living with HAE has the potential to experience a rapid, substantial and sustained reduction in their monthly attack rate with ORLADEYO.
These analyses present the overall attack rate progression and attack rate progression stratified by severity (i.e., number of attacks at baseline), prior prophylaxis and C1-INH level and function.
These findings further demonstrate that ORLADEYO can help maintain disease control in patients with lower baseline attack rates and further reduce attack rates in patients with more active disease.
Berotralstat Prophylaxis Reduces HAE Attack Rates Regardless of Baseline Attacks: Real-World Outcomes; Poster #012; Friday, February 23, 3:15-4:15 p.m.

Positive Results from CHAPTER-1 Phase 2 Study of Deucrictibant for the Prophylactic Treatment of HAE Attacks to be Presented at AAAAI 2024 Annual Meeting

Retrieved on:

Thursday, February 22, 2024

CHAPTER-1 is a two-part, Phase 2 study evaluating the efficacy, safety, and tolerability of deucrictibant for long-term prophylaxis against angioedema attacks in HAE type 1 and type 2 (HAE-1/2).

Key Points:

CHAPTER-1 is a two-part, Phase 2 study evaluating the efficacy, safety, and tolerability of deucrictibant for long-term prophylaxis against angioedema attacks in HAE type 1 and type 2 (HAE-1/2).
34 participants were treated with double-blinded study drug (placebo or deucrictibant, 20 or 40 mg/day) for 12 weeks of treatment.
“These data validate the mechanism of deucrictibant to provide early and sustained protection from HAE attacks.
By better understanding and addressing these factors, we aspire to make our commitment to ‘pioneering science for patient choice’ a reality.”

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on:

Thursday, February 22, 2024

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.

Key Points:

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.
R&D Expenses: Research and development expenses increased by $9.0 million to $109.0 million during the fourth quarter of 2023, compared to $100.0 million during the fourth quarter of 2022.
G&A Expenses: General and administrative expenses increased by $5.4 million to $29.0 million during the fourth quarter of 2023, compared to $23.6 million during the fourth quarter of 2022.
Net Loss: The Company’s net loss was $132.2 million for the fourth quarter of 2023, compared to $113.4 million during the fourth quarter of 2022.

BioCryst Launches ORLADEYO® (berotralstat) in Italy

Retrieved on:

Monday, February 19, 2024

European Commission, Hereditary angioedema, News, Berotralstat, HAE, Patient, BioCryst Pharmaceuticals

RESEARCH TRIANGLE PARK, N.C., Feb. 19, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Italian Medicines Agency, Agenzia Italiana del Farmaco (AIFA), has finalized reimbursement and recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older.

Key Points:

RESEARCH TRIANGLE PARK, N.C., Feb. 19, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Italian Medicines Agency, Agenzia Italiana del Farmaco (AIFA), has finalized reimbursement and recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older.
With this recommendation, HAE patients in Italy now have access to the first oral, once-daily therapy for the routine prevention of recurrent HAE attacks.
“The positive AIFA recommendation of ORLADEYO® (berotralstat) broadens access to modern prophylaxis in Italy and is great news for Italian clinicians and HAE patients,” said Charlie Gayer, chief commercial officer of BioCryst.
The AIFA decision follows European Commission (EC) marketing authorization of ORLADEYO in April 2021.

KalVista Pharmaceuticals to Present HAE Attack Journey Data at 2024 HAEi Regional Conference Americas

Retrieved on:

Friday, March 8, 2024

Biotechnology, Pharmaceutical, Health, Clinical Trials, EST, Treatment, Quality, Return, Conference, HAE, Quality of life, Hereditary angioedema, Abstract, KALV, Patient, Q&A, Pharmaceutical industry

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 acceptance of multiple abstracts at the 2024 HAEi Regional Conference meeting taking place in Panama City, Panama from March 15-17.

Key Points:

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 acceptance of multiple abstracts at the 2024 HAEi Regional Conference meeting taking place in Panama City, Panama from March 15-17.
KalVista is a Silver-level sponsor of the meeting.
Results shared as an oral poster presentation and Q&A
Delayed On-Demand Treatment of Hereditary Angioedema Attacks: Patient Perceptions and Associated Barriers: Sandra Christiansen, Maeve O’Connor, Julie Ulloa, Sherry Danese, Vibha Desai, Paul Audhya, Paula Busse.
Results shared as an oral poster presentation and Q&A
Characteristics of Hereditary Angioedema Attacks Among Long-Term Prophylaxis Users: Bob Geng, Vibha Desai, Julie Ulloa, Sherry Danese, Paul Audhya, Timothy Craig, Maeve O’Connor (presenter-only).

KalVista Pharmaceuticals Announces Appointment of Benjamin L. Palleiko as Chief Executive Officer

Retrieved on:

Thursday, March 7, 2024

Biotechnology, Pharmaceutical, Health, Clinical Trials, CFO, Hereditary angioedema, University, Growth, MBA, United States Navy, Tufts University, HAE, Naval aviation, KALV, CBO, NDA, Navy, Quantitative Economics, Pharmaceutical industry

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 that Benjamin L. Palleiko, the Company’s current President, CBO and CFO, has been appointed as Chief Executive Officer and a director of the Company, effective immediately.

Key Points:

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 that Benjamin L. Palleiko, the Company’s current President, CBO and CFO, has been appointed as Chief Executive Officer and a director of the Company, effective immediately.
In conjunction, Andrew Crockett has submitted his resignation as CEO and a director of the Company.
His long tenure with the Company positions us for continued success and allows us to make this seamless transition,” said Brian J.G.
He holds an MBA in Finance and MA in International Relations from the University of Chicago, and a BA in Quantitative Economics from Tufts University.

Astria Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides a Corporate Update

Retrieved on:

Monday, March 4, 2024

Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update.

Key Points:

Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update.
The trial is evaluating safety and tolerability, changes in HAE attack rate, pharmacokinetics (PK), pharmacodynamics (PD), and quality-of-life assessments.
Pending proof-of-concept results from the ALPHA-STAR trial, Astria expects to progress directly to a pivotal Phase 3 program which is anticipated to initiate in the first quarter of 2025.
Cash Position: As of December 31, 2023, Astria had cash, cash equivalents and short-term investments of $246.5 million, compared to $188.8 million as of September 30, 2023.

KalVista Pharmaceuticals Presents Data on Unmet Needs in HAE from a Patient Perspective at the 2024 American Academy of Allergy, Asthma & Immunology Annual Meeting

Retrieved on:

Tuesday, February 27, 2024

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 that it presented real-world data from US patient surveys that assessed the experience of HAE patients using injectable on-demand treatments at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting that took place in Washington, DC.

Key Points:

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 that it presented real-world data from US patient surveys that assessed the experience of HAE patients using injectable on-demand treatments at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting that took place in Washington, DC.
The following presentations occurred at AAAAI 2024:
Characteristics of Hereditary Angioedema Attacks Among Long-Term Prophylaxis Users: Bob Geng, Allergy and Immunology, University of California, San Diego, California, United States (Poster Presentation)
Among HAE patients who had treated a recent attack, the location and duration of the most recent attacks were similar between long-term prophylaxis (LTP) and on-demand only users
Earlier on-demand treatment was associated with a shorter attack duration, regardless of LTP use
Delayed On-demand Treatment of Hereditary Angioedema Attacks: Patient Perceptions and Associated Barriers: Sandra Christiansen, University of California San Diego, La Jolla, CA, United States (Poster Presentation)
The most common barriers to earlier treatment were uncertainty if attack was real, thinking the attack would be mild, and wanting to save treatment for a severe attack
Anxiety Associated with On-Demand Treatment for Hereditary Angioedema (HAE) Attacks: James Wedner, Washington University School of Medicine, St Louis, MO, United States (Poster Presentation)
Both adults and adolescents with HAE reported moderate to extreme anxiety when anticipating use of parenteral on-demand treatment, irrespective of use of on-demand only or on-demand plus LTP
The results of this study highlighted the association between delayed treatment and treatment-related anxiety, with a higher proportion of patients who delayed treatment experiencing moderate to extreme anxiety
Anxiety Associated with Refilling On-demand Therapy for HAE Attacks Contributes to Treatment Delay and Non-Treatment: Autumn Burnette, Division of Allergy and Immunology, Howard University Hospital, Washington, DC, United States (Poster Presentation)
Anxiety associated with not being able to refill on-demand treatment quickly impacted treatment decisions, which contributed to treatment delay or resulted in non-treatment of HAE attacks
Characterizing the Negative Impact of Delayed On-Demand Treatment of HAE Attacks: Princess Ogbogu, Division of Pediatric Allergy, Immunology, and Rheumatology, University Hospitals Rainbow Babies and Children's Hospital, Cleveland, Ohio, United States (Poster Presentation)
The Impact of On-demand Treatment on Quality of Life of People with HAE: Paula Busse, Department of Medicine, Division of Clinical Immunology, Mount Sinai, New York, United States (Poster Presentation)
Characterizing the Perspective of Patients With HAE on Prophylactic Treatment: Stephen Betschel, Division of Allergy and Immunology, Department of Medicine, St. Michael’s Hospital, University of Toronto, Toronto, Ontario, Canada (Poster Presentation)
Only 35% of LTP patients always carried on-demand treatment when away from home, while 43% of prophylaxis patients cited avoiding potential triggers as a reason for not carrying on-demand treatment at all times
Treatment Patterns of Patients Requiring Redosing of an On-demand Treatment After the Return of an HAE Attack: Constance Katelaris, Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney, NSW, Australia (Poster Presentation)
“The results of these surveys clearly conveyed the challenges faced by patients trying to manage their HAE attacks with injectable on-demand treatments.
The resulting non-compliance with treatment guidelines may lead to poor clinical outcomes, even among patients receiving LTP,” said Andrew Crockett, Chief Executive Officer of KalVista.
“We believe the efficacy and safety data from our phase 3 trial for sebetralstat show a potential path forward to address these persisting unmet needs.”

KalVista Pharmaceuticals Presents Additional Phase 3 KONFIDENT Data at the 2024 American Academy of Allergy, Asthma & Immunology Annual Meeting

Retrieved on:

Monday, February 26, 2024

Additional safety analyses demonstrated that the safety profiles associated with one dose or two doses of sebetralstat 300 mg or 600 mg were comparable to placebo.

Key Points:

Additional safety analyses demonstrated that the safety profiles associated with one dose or two doses of sebetralstat 300 mg or 600 mg were comparable to placebo.
Data presented as a supplement also showed that the median time to all endpoints was shorter for attacks with higher initial severity, at both dose levels.
“If approved, we believe that a single dose of sebetralstat 300 mg would appear to be appropriate for most HAE attacks.
“We believe that these additional efficacy and safety data only strengthen the case for sebetralstat to become the first, oral on-demand treatment available to the HAE community.”

KalVista Pharmaceuticals Awarded UK Innovation Passport for Sebetralstat

Retrieved on:

Tuesday, February 20, 2024

Biotechnology, Health, Genetics, Pharmaceutical, Cardiology, AAAAI, Hereditary angioedema, Annual general meeting, Safety, Health care, HAE, KALV, MHRA, American Academy, Pharmaceutical industry

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 Innovation Passport for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).

Key Points:

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 Innovation Passport for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).
The Innovation Passport is the first step in the UK’s Innovative Licensing and Access Pathway (ILAP), which is designed to accelerate a product’s time to market and facilitate patient access to innovative medicines.
“As a company which has its roots in the UK, we are pleased to receive the ILAP designation, which will enable us to further accelerate our regulatory submission for sebetralstat,” said Andrew Crockett, Chief Executive Officer of KalVista.
The Company will be presenting late-breaking KONFIDENT trial data on February 25, 2024, at the upcoming American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting.