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LL 10 november 2021 21:38

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KALVISTA DEVELOPMENT TEAM FINALIZES PHASE 3 TRIAL PROTOCOL
KalVista Pharmaceuticals, Inc. provides an update on the clinical trial progress for KVD900 in development for oral on-demand treatment of HAE.

“We had a productive End-of-Phase 2 meeting with the FDA and recently received meeting minutes which confirmed that our Phase 3 trial design, similar to our recent successful Phase 2 trial, is expected to be appropriate to support an NDA submission,” says Andrew Crockett, CEO of KalVista. “Our development team is finalizing the Phase 3 trial protocol and preparing for study initiation, and we anticipate patients will be dosed during the first quarter of 2022. KalVista is well-capitalized, with funding until at least early 2024, which we expect takes us beyond data from both this Phase 3 trial and the ongoing Phase 2 trial of KVD824.”

The Phase 3 clinical trial of KVD900 is a crossover design evaluating dose levels of 300 mg and 600 mg KVD900 against placebo. The primary endpoint of this Phase 3 trial is time to beginning of symptom relief. The trial is expected to be conducted at more than 50 sites worldwide and recruit approximately 100 patients, consistent with late stage trials of approved on-demand treatments for HAE. The trial is intended to evaluate all HAE attacks, including laryngeal attacks and breakthrough attacks for patients using prophylaxis. Similar to the Phase 2 trial for which positive data was announced earlier in 2021, patients will administer treatment as soon as they recognize the onset of an attack.

LL 12 november 2021 18:35

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Pharvaris Reports Third Quarter 2021 Financial Results and Provides Business Highlights

RAPIDe-1, Phase 2 on-demand study of PHVS416 for the treatment of HAE attacks, proceeding; topline data reaffirmed for 2022

CHAPTER-1, Phase 2 prophylactic study of PHVS416 for the prevention of HAE attacks, recruiting; topline data expected in 2022

PHVS719 Phase 1 pharmacokinetics study initiating this month

Executing from a strong financial position with cash and cash equivalents of €218.6 million as of September 30, 2021

ZUG, Switzerland, Nov. 10, 2021 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a clinical-stage company focused on the development and commercialization of novel oral bradykinin-B2-receptor antagonists for the treatment of hereditary angioedema (HAE) and other bradykinin-B2-receptor-mediated indications, today reported financial results for the third quarter ended September 30, 2021, and provided an update on recent business highlights.

“This quarter we continued to execute on our robust clinical development strategy as we seek to advance novel treatments for HAE patients that offer efficacy without compromising on convenience,” said Berndt Modig, co-founder and chief executive officer of Pharvaris. “We continue enrolling patients in RAPIDe-1, our Phase 2 on-demand study of PHVS416, and have begun recruiting in CHAPTER-1, our Phase 2 prophylactic study of PHVS416 for the prevention of HAE attacks. We expect to report top-line data, including efficacy and safety, for both studies in 2022. This month, in the PHVS719 program for HAE prophylaxis we also expect to initiate dosing in a Phase 1 pharmacokinetic study designed to assess the bioavailability of extended-release formulation.”

Recent Pipeline and Business Highlights and Upcoming Milestones

Phase 2 on-demand study (RAPIDe-1) of PHVS416 proceeding toward data readout in 2022. In February 2021, Pharvaris announced that dosing had commenced in its Phase 2 clinical study of PHVS416 for the on-demand treatment of HAE attacks. The company reaffirms guidance for reporting topline efficacy and safety data from this study in 2022. Pharvaris is conducting RAPIDe-1 at 33 sites in Canada, Europe, Israel, the UK, and the US.

Phase 2 prophylactic study (HAE CHAPTER-1) of PHVS416 recruiting. In addition to developing PHVS416 for the on-demand treatment of HAE attacks, the company plans to investigate the therapeutic potential of the PHVS416 formulation of PHA121 for the prophylactic prevention of HAE attacks. In April 2021, Pharvaris announced that an IND was in effect in the US. Patient recruitment has begun and the study is expanding to Canada, Europe, Israel, and the UK. Pharvaris anticipates reporting topline safety and efficacy data from this study in 2022.

Phase 1 pharmacokinetics study of PHVS719 initiating shortly. PHVS719 is under development as an extended-release formulation of PHA121 intended for use in the prophylactic treatment of HAE. Dosing of a Phase 1 pharmacokinetics study to assess the bioavailability of the extended-release formulation is expected to begin this month.

Expanding corporate capabilities. With Wim Souverijns joining as Chief Community Engagement and Commercial Officer to engage with patient advocacy groups, clinicians, and payers, in the third quarter the company further strengthened capabilities in Community Engagement and Commercialization, as well as CMC, Clinical, and organizational development.

bron:
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LL 21 november 2021 15:03

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Ionis initiates Phase 3 clinical program of donidalorsen in patients with hereditary angioedema
November 18, 2021 at 1:30 PM EST
- OASIS-HAE is the registrational study in Ionis' donidalorsen Phase 3 clinical program and further expands Ionis' late-stage pipeline
- Donidalorsen is one of Ionis' wholly owned medicines the company plans to commercialize

CARLSBAD, Calif., Nov. 18, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today initiation of OASIS-HAE, the registrational study in the donidalorsen (formerly IONIS-PKK-LRx) Phase 3 clinical program. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation of inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). Donidalorsen uses Ionis' LIgand-Conjugated Antisense, or LICA, technology.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

"Initiating the Phase 3 program for donidalorsen moves us one step closer to bringing a potential best-in-class prophylactic treatment to market for people with HAE globally experiencing recurrent painful and severe HAE attacks," said Kenneth Newman, M.D., M.B.A., Ionis' vice president of clinical development and leader of the immunology and pulmonology franchise. "Advancing this program underscores our commitment to the HAE patient community to deliver transformative treatments."

OASIS-HAE is a double-blind, randomized, placebo-controlled registrational study in up to 84 patients with hereditary angioedema, Types 1 and 2. Patients will be randomized to receive monthly or bi-monthly subcutaneous donidalorsen for 25 weeks. Following the placebo-controlled portion of the study, patients may enter the 52-week open-label extension study.

New Phase 2 data recently presented at ACAAI highlighted an overall reduction in moderate to severe attacks, and a reduction in these attacks starting with the second dose. For the final month of the study, all donidalorsen treated patients were attack-free. In this study, donidalorsen was safe and well tolerated.

For more information on the OASIS-HAE clinical study please visit www.clinicaltrials.gov.

About Donidalorsen Phase 2 study

In the Phase 2 clinical study, 20 adults with Type 1 or Type 2 HAE were randomized and received either donidalorsen 80mg (n=14) or placebo (n=6) subcutaneously once monthly for 17 weeks. The primary endpoint was the reduction of monthly HAE attacks compared to placebo. Secondary endpoints included the reduction of monthly attacks in weeks five to 17, reduction in the number of moderate or severe attacks in weeks one to 17, the number of moderate or severe attacks in weeks five to 17 and the number of attacks requiring acute therapy in weeks five to 17. The majority of adverse events during the study were mild with a frequency that was similar between groups. The most common treatment-emergent adverse events (TEAEs) were headache and nausea, which were seen more frequently in the placebo arm compared to the active treatment arm.

bron:
ir.ionispharma.com/news-releases/news...

[verwijderd] 25 november 2021 13:36

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quote:
LL schreef op 21 november 2021 15:03:
Ionis initiates Phase 3 clinical program of donidalorsen in patients with hereditary angioedema
November 18, 2021 at 1:30 PM EST
- OASIS-HAE is the registrational study in Ionis' donidalorsen Phase 3 clinical program and further expands Ionis' late-stage pipeline
- Donidalorsen is one of Ionis' wholly owned medicines the company plans to commercialize

CARLSBAD, Calif., Nov. 18, 2021 PRNewswire -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today initiation of OASIS-HAE, the registrational study in the donidalorsen (formerly IONIS-PKK-LRx) Phase 3 clinical program. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation of inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). Donidalorsen uses Ionis' LIgand-Conjugated Antisense, or LICA, technology.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

"Initiating the Phase 3 program for donidalorsen moves us one step closer to bringing a potential best-in-class prophylactic treatment to market for people with HAE globally experiencing recurrent painful and severe HAE attacks," said Kenneth Newman, M.D., M.B.A., Ionis' vice president of clinical development and leader of the immunology and pulmonology franchise. "Advancing this program underscores our commitment to the HAE patient community to deliver transformative treatments."

OASIS-HAE is a double-blind, randomized, placebo-controlled registrational study in up to 84 patients with hereditary angioedema, Types 1 and 2. Patients will be randomized to receive monthly or bi-monthly subcutaneous donidalorsen for 25 weeks. Following the placebo-controlled portion of the study, patients may enter the 52-week open-label extension study.

New Phase 2 data recently presented at ACAAI highlighted an overall reduction in moderate to severe attacks, and a reduction in these attacks starting with the second dose. For the final month of the study, all donidalorsen treated patients were attack-free. In this study, donidalorsen was safe and well tolerated.

For more information on the OASIS-HAE clinical study please visit

About Donidalorsen Phase 2 study

In the Phase 2 clinical study, 20 adults with Type 1 or Type 2 HAE were randomized and received either donidalorsen 80mg (n=14) or placebo (n=6) subcutaneously once monthly for 17 weeks. The primary endpoint was the reduction of monthly HAE attacks compared to placebo. Secondary endpoints included the reduction of monthly attacks in weeks five to 17, reduction in the number of moderate or severe attacks in weeks one to 17, the number of moderate or severe attacks in weeks five to 17 and the number of attacks requiring acute therapy in weeks five to 17. The majority of adverse events during the study were mild with a frequency that was similar between groups. The most common treatment-emergent adverse events (TEAEs) were headache and nausea, which were seen more frequently in the placebo arm compared to the active treatment arm.

bron:

Zowel Ionis als Kalvista inmiddels in Fase III aanbeland!

LL 15 december 2021 17:21

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First patient dosed with Intellia’s NTLA-2002

The first patient has been dosed with NTLA-2002, Intellia Therapeutics, Inc’s in vivo CRISPR/Cas9 genome editing candidate being developed as a single-dose therapy to prevent attacks in people living with HAE. NTLA-2002 is a systemically administered therapy designed to inactivate the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity and thus prevent HAE attacks.

“With the progress of our first-in-human clinical study evaluating NTLA-2002 for people living with HAE, we look forward to beginning clinical testing as we aim to develop a single-dose treatment for these patients”, says Intellia President and CEO John Leonard, M.D.

Intellia’s multi-national Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies.

LL 8 januari 2022 13:18

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Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2022
Jan 6, 2022

Advance clinical development of NTLA-2001, a potential single-dose therapy for transthyretin (ATTR) amyloidosis; on track to present additional data from Phase 1 study in Q1 2022
Achieve preliminary proof-of-concept for NTLA-2002 in patients with hereditary angioedema (HAE) based on ongoing first-in-human study; anticipate presenting interim data in 2H 2022
Establish initial safety and activity profile of NTLA-5001 for acute myeloid leukemia (AML) in Phase 1/2a study
Progress in vivo and ex vivo research programs and integrate novel platform technologies; expect to nominate multiple new development candidates in 2022
Ended 2021 in strong financial position with $1.1 billion in cash

CAMBRIDGE, Mass., Jan. 06, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, today outlined its expected milestones and the following strategic priorities for 2022:

Accelerating clinical validation of in vivo pipeline: Further characterize the safety and efficacy of NTLA-2001, including in patients with cardiomyopathy and complete enrollment of the Phase 1 study; establish the initial clinical profile of NTLA-2002 as a single-dose therapy for the treatment of hereditary angioedema (HAE).
Strategic pipeline expansion: Significantly progress in vivo and ex vivo pipeline, including determining the initial safety profile of NTLA-5001 for acute myeloid leukemia (AML), advancing in vivo insertion candidates and nominating multiple new development candidates.
Platform innovation: Broaden the Company’s industry-leading platform through expansion of Intellia’s genome editing, delivery and cell engineering capabilities.

“Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases,” said Intellia President and Chief Executive Officer John Leonard, M.D. “As we begin 2022 with great momentum, we are poised to significantly expand our full-spectrum pipeline of potentially curative therapies with the nomination of at least two new in vivo candidates and our first allogeneic development candidate during the year. Importantly, we look forward to sharing additional data from the ongoing study of NTLA-2001 and interim results from the Phase 1/2 study of NTLA-2002, which we expect will further demonstrate the modularity of our genome editing platform.”

Anticipated 2022 Milestones:

In Vivo Programs

NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, and has the potential to be the first single-dose treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Today, Intellia announced that the first patient in the cardiomyopathy arm of the Phase 1 study has been dosed with NTLA-2001. This follows the Company’s recent announcement that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study now includes patients with ATTR-CM enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Intellia expects to complete enrollment of the Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022.
Intellia intends to present additional interim clinical data of NTLA-2001 in ATTRv-PN patients from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four ATTRv-PN dose cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In December 2021, Intellia dosed the first patient in its second clinical study of a CRISPR-based therapeutic candidate evaluating NTLA-2002 for HAE. The first-in-human Phase 1/2 trial is expected to evaluate the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE, and will continue to leverage insights gained from the development of NTLA-2001.
The Company expects to present interim data from the Phase 1/2 study in the second half of 2022. These results are expected to characterize the emerging safety and activity profile of NTLA-2002 and demonstrate preliminary proof-of-concept.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s wholly owned CRISPR-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001 with plans to file an IND or IND-equivalent in 2023. The Company also continues to explore additional editing strategies for AATD.

bron:
ir.intelliatx.com/news-releases/news-...

LL 26 januari 2022 07:10

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KalVista Pharmaceuticals Announces Publication of Oral HAE Therapy KVD900 Phase 1 Data in Journal of Allergy and Clinical Immunology
January 24, 2022 at 7:30 AM EST

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Jan. 24, 2022-- KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of small molecule protease inhibitors, today announced data from the Phase 1 clinical trials of oral, on-demand treatment KVD900 in patients with hereditary angioedema (HAE), have been published online by the Journal of Allergy and Clinical Immunology (JACI).

The objective of the Phase 1 studies was to evaluate the safety, tolerability, pharmacokinetics, and clinical pharmacology of KVD900, an orally administered inhibitor of plasma kallikrein in healthy adults. KVD900 was administered to 98 participants in total, and the data showed that KVD900 achieves near-complete plasma kallikrein inhibition within 30 minutes and was generally safe and well tolerated.

“We are pleased to see these data published in JACI to further describe our ongoing work to bring a safe, oral on-demand treatment option to the market for HAE patients,” said Andrew Crockett, Chief Executive Officer of KalVista. “These data show that KVD900 rapidly suppresses plasma kallikrein activity, a key mediator of HAE attacks, and may provide the early relief from HAE attack progression that represents a currently unmet need in orally administered management of the disease. These findings have since been further validated by the results of our Phase 2 clinical trial for KVD900.”

Additional details can be found in the manuscript, which is available in the “Articles in Press” section of the JACI website, located at: www.jacionline.org. JACI is an official journal of the American Academy of Allergy, Asthma, and Immunology.

bron:
ir.kalvista.com/news-releases/news-re...

[verwijderd] 22 februari 2022 10:53

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Ook Biomarin gaat de gentherapie testen op echte patiënten!

Enrollment Opens in HAERMONY Trial to Test HAE Gene Therapy
Steve Bryson PhD avatar
by Steve Bryson PhD | February 21, 2022

Enrollment is open for the open-label, Phase 1/2 HAERMONY trial to evaluate the safety and effectiveness of BMN 331, an investigational gene therapy for hereditary angioedema (HAE).

The trial’s sponsor, BioMarin Pharmaceutical, plans to recruit about 34 adults with type 1 or type 2 HAE at one site in Missouri. More information about enrollment and contact information can be found here.

HAE is a genetic disorder characterized by sudden and recurrent swelling in the hands, feet, face, upper airways, gastrointestinal tract, and genitals. Most cases are caused by inherited mutations in the SERPING1 gene, which carries instructions for making the C1-inhibitor (C1-INH) protein.

For people with type 1 HAE, these mutations impair C1-INH production, while the protein malfunctions for those with type 2 HAE. Not enough C1-INH activity results in abnormally high levels of a pro-inflammatory protein called bradykinin, which causes swelling attacks.

BMN 331 is designed to provide a healthy copy of the SERPING1 gene to increase levels of C1-INH and reduce swelling attacks or keep them from occurring. The therapy is delivered to liver cells, where C1-INH is produced, by a harmless virus called AAV5 via a single infusion into the vein.

According to BioMarin, preclinical studies have demonstrated that BMN 331 can insert the SERPING1 gene into liver cells and restore C1-INH levels in the bloodstream.

The five-year HAERMONY trial (NCT05121376) will be a two-part study. Part A will assess the preliminary safety of single, escalating doses of BMN 331 and determine whether C1-INH increases in a dose-dependent manner. Part B will be a dose-expansion phase to find up to three safe doses that will sustain clinically meaningful C1-INH levels.

The trial’s primary goal is to assess the number of participants who develop side effects following the infusion. Secondary measures include evaluating HAE attacks’ number and severity, the annualized use of HAE medications, and the levels of antibodies targeting the C1-INH protein and the AAV5 viral delivery system.

Both males and females 18 and older with a genetically confirmed HAE diagnosis are eligible to participate. Patients must be receiving routine prophylactic (preventive) or on-demand therapy six months before enrolling for attacks that occur on average at least once a month.

Participants need to be trained to self-administer acute attack treatments and manage any attacks at home, be willing to abstain from consuming alcohol for at least one year after infusion, and use highly effective contraception, if applicable.

BMN 331 was granted an orphan drug designation by the U.S. Food and Drug Administration. This designation seeks to support the development of rare disease therapies by providing developers with incentives and benefits, including tax credits for clinical trials, exemption of certain fees, and a seven-year market exclusivity period upon approval.

LL 2 maart 2022 08:04

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KalVista Pharmaceuticals Presents Data Showing Rapid Reduction of Plasma Kallikrein Activity After Oral KVD900 Treatment and Early Symptom Relief From HAE Attacks in Patients
February 28, 2022 at 4:15 PM EST

– KVD900 Phase 2 data presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Scientific Meeting –

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Feb. 28, 2022-- KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today presented data for its lead oral drug candidate, KVD900, in development for on-demand treatment of hereditary angioedema (HAE), at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Scientific Meeting. Data presentations included new data from the Phase 2 trial highlighting rapid suppression of plasma kallikrein activity after KVD900 administration and its relationship with symptomatic relief.

“These data from our phase 2 trial in on-demand use for HAE attacks show that oral KVD900 is quickly absorbed, leading to rapid, near-complete suppression of plasma kallikrein activity, a key mediator of HAE attacks,” said Andrew Crockett, Chief Executive Officer of KalVista. “We believe that this rapid absorption followed by rapid suppression of plasma kallikrein activity is the basis for the early symptom relief we observed in the trial.”

The following posters were presented at AAAAI:

Poster Title: Rapid Plasma Kallikrein Inhibition Following Oral KVD900 is Associated with Early Symptom Relief in Patients with Hereditary Angioedema

KVD900 was rapidly absorbed, reaching maximum plasma concentrations within 1 hour
Plasma kallikrein activity was >80% inhibited within 15 minutes with near-complete inhibition (>95%) observed within 1 hour
A significantly shorter median time (1.6 hrs.) to symptom relief was observed in patients receiving KDV900 compared with placebo (9 hrs.)

Poster Title: Agreement of Patient Global Impression of Change (PGI-C) with Attack Resolution or Use of Rescue Mediation in Patients with Hereditary Angioedema

113 HAE attacks were treated in a blinded manner with either KVD900 or placebo
Symptom relief, as captured by improvement on the PGI-C, occurred in 72% of attacks within 24 hours, and among those only 16% of HAE attacks were associated with use of rescue medication, while approximately 60% achieved complete attack resolution without use of rescue
Conversely, for the 28% of attacks where symptom relief was not achieved, 66% were associated with use of rescue medication and less than 4% achieved complete attack resolution without use of rescue
PGI-C is an effective tool to monitor attack symptoms may be an early predictor of attack resolution in patients with HAE

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 has developed a proprietary portfolio of novel, small molecule plasma kallikrein inhibitors initially targeting hereditary angioedema (HAE) and diabetic macular edema (DME). KalVista is developing KVD900 as an oral on-demand therapy for acute HAE attacks, which completed a Phase 2 efficacy trial in February 2021, demonstrating statistical and clinical significance across all endpoints. KVD824 is in development for prophylactic treatment of HAE, with the Phase 2 KOMPLETE clinical trial underway. In addition, KalVista’s oral Factor XIIa inhibitor program represents a new generation of therapies that may further improve the treatment of HAE for patients. In DME, an intravitreally administered plasma kallikrein inhibitor, called KVD001, has completed a Phase 2 clinical trial.

For more information, please visit www.kalvista.com.
For more information on the KVD824 HAE prophylaxis Phase 2 KOMPLETE study, please visit www.kompletestudy.com.

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, including the potential impact of COVID-19, 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 its results, our ability to commence clinical studies or complete ongoing clinical studies, including our Phase 2 KOMPLETE clinical trial, and to obtain regulatory approvals for KVD900, KVD824 and other candidates in development, the ability of KVD900, KVD824 and other candidates in development to treat HAE or DME, 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, 2021, 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.

bron:
ir.kalvista.com/news-releases/news-re...

LL 3 maart 2022 07:28

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Interim Phase 4 Data Support TAKHZYRO® (lanadelumab) as an Effective Treatment to Reduce Attacks in Hereditary Angioedema Patients
February 28, 2022

Interim Real-World Data from Phase 4 EMPOWER Study Show Attack Rate Reduction and Improvement in Treatment Satisfaction and Other Patient-Reported Outcome Scores1
Interim Patient-Reported Outcomes Showed a Reduction of Monthly Attack Rates in New Users and Showed Sustained Angioedema Control in Established Users Over Twelve-Months Using the Angioedema Control Test (AECT)1,2,3
Attack Rate Reduction in Patients Previously on Androgen Treatments Consistent with Study Population in Phase 3 HELP OLE Based on Post-Hoc Analysis4

OSAKA, Japan, and CAMBRIDGE, Massachusetts, February 28, 2022 – Takeda (TSE:4502/NYSE:TAK) today presented four abstracts including interim real-world data from the observational Phase 4 EMPOWER study of TAKHZYRO® (lanadelumab) as a treatment for people with Hereditary Angioedema (HAE) Type I or II in North America, as well as findings from a post-hoc analysis of the HELP Open Label Extension study of long-term safety and efficacy of TAKHZYRO in HAE patients 12 years of age and older at the American Academy of Allergy, Asthma and Immunology (AAAAI) 78th Annual Meeting.

“A big challenge for HAE patients is the unpredictability of attacks and the impact that the attacks have on quality of life. We are encouraged by the initial results of the EMPOWER study, along with the additional evidence presented in the HELP Open Label Extension,” said Associate Professor Paula Busse, MD, Division of Allergy and Clinical Immunology, Icahn School of Medicine at Mount Sinai in New York, NY USA. “Data from both studies show that lanadelumab had marked reduction in attack rates, and that angioedema control was sustained for 12 months.”

Key findings from the data presentations include:

Improvements among new users and sustained outcomes in established users were reported in scores of the Angioedema Quality of Life Questionnaire (AE-QOL), Angioedema Control Test (AECT), and the Treatment Satisfaction Questionnaire for Medication (TSQM-9) when collected every three months, as presented in the EMPOWER IA2 Patient-Reported Outcomes interim data presentation.1
An average of 1 in 5 established TAKHZYRO users were able to extend treatment from every 2 weeks to 4 weeks as shown in interim data shared in the EMPOWER IA2 Treatment Patterns and Subgroups3
Interim real-world data showed marked attack rate reduction of 83% and no new safety signals based on patient self-reporting reduced attack rates in the EMPOWER IA2 Effectiveness and Safety2
A post-hoc analysis of HELP and HELP OLE showed that reduction of attack rates with TAKHZYRO were similar for patients previously on androgen treatments as they were for the wider treatment population in these studies in the Switch from Androgens to TAKHZYRO in HELP 03 and HELP 04.4

“We are pleased to see the real-world data from EMPOWER show improvements in angioedema control and treatment satisfaction. These interim results provide a better understanding of the overall patient experience with TAKHZYRO," said Neil Inhaber, MD, Head, Rare Genetics and Hematology, Global Medical Affairs, Takeda. “With more than a decade of experience and innovation for patients with this devastating condition, Takeda remains committed to continuing our unwavering support for the HAE community.”

The observational Phase 4 EMPOWER study, evaluating real-world HAE attack rates before and after treatment with TAKHZYRO in patients with HAE types I and II, is ongoing. Full results of the EMPOWER study are expected to be published in 2024. HELP OLE is a completed Phase 3 study of the safety and efficacy of TAKHZYRO in patients previously treated with androgens and other therapies for long-term prophylaxis prior to transitioning to TAKHZYRO.

bron:
www.takeda.com/newsroom/newsreleases/...

LL 9 maart 2022 06:59

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KalVista Pharmaceuticals Announces Initiation of KVD900 Phase 3 KONFIDENT Clinical Trial
March 7, 2022 at 6:30 AM EST

-KONFIDENT to Evaluate KVD900 As First Oral On-Demand Therapy for HAE-

-KONFIDENT Designed to Support Broad Label for Treatment of All HAE Attacks-

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Mar. 7, 2022-- 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 initiation of the Phase 3 KONFIDENT clinical trial evaluating the efficacy and safety of KVD900 as the first potential oral, on-demand therapy for hereditary angioedema (HAE) attacks. This worldwide, double-blind, placebo-controlled crossover trial will evaluate the efficacy of two dose levels of KVD900 compared to placebo in adolescents and adults experiencing acute HAE attacks. KVD900 is the most advanced potential oral on-demand therapy for HAE in clinical development, and is intended to provide a substantial improvement over the current on-demand therapies for HAE attacks, which are all delivered by injection.

“Beginning the KONFIDENT trial represents a major milestone for KalVista,” said Andrew Crockett, Chief Executive Officer of KalVista. “We believe that KVD900 has the potential to transform the treatment paradigm for HAE patients experiencing acute attacks, whether they primarily treat with on-demand medications or use long-term prophylaxis. Based upon the results of our Phase 2 study released last year, we expect that KVD900 can provide patients with symptom relief as rapidly as existing therapies, but with an oral tablet that will allow earlier treatment of all patient-recognized HAE attacks. Our goal is to provide patients with the confidence that their attacks will be controlled in the earliest stages and without the associated treatment pain and other challenges of injectable therapies.”

The Phase 3 KONFIDENT trial is a worldwide clinical study being conducted at approximately 60 sites in 20 countries. The trial is intended to enroll a minimum of 84 HAE adolescent and adult patients who will complete treatment of three attacks: one each with 300 mg KVD900, 600 mg KVD900 and placebo in a double-blinded, randomized sequence. The primary endpoint of the trial is time to the beginning of symptom relief, evaluated on a Patient Global Impression of Change (PGI-C) scale, and additional endpoints will evaluate other measures of patient response and attack progression, as well as safety. Patients will dose upon first recognition of an attack, and all attack types including laryngeal attacks will be eligible for treatment. Patients will be permitted to take an additional dose of investigational drug, if symptoms warrant, and will always have access to their conventional injectable therapy. Study participants also will be allowed to maintain their prophylaxis regimen if they were receiving one at study enrollment.

KalVista currently anticipates that data from KONFIDENT will be available in the second half of 2023 and will provide further updates as the trial progresses. Additional information about KONFIDENT can be found at www.konfidentstudy.com and www.clinicaltrials.gov.

About KalVista Pharmaceuticals, Inc.

KalVista Pharmaceuticals, Inc. is a pharmaceutical company focused on the discovery, development, and commercialization of small molecule protease inhibitors for diseases with significant unmet need. KalVista has developed a proprietary portfolio of novel, small molecule plasma kallikrein inhibitors initially targeting hereditary angioedema (HAE) and diabetic macular edema (DME). KalVista is developing KVD900 as an oral on-demand therapy for acute HAE attacks and has initiated the Phase 3 KONFIDENT clinical trial. KVD824 is in development for prophylactic treatment of HAE, with the Phase 2 KOMPLETE clinical trial underway. In addition, KalVista’s oral Factor XIIa inhibitor program represents a new generation of therapies that may further improve the treatment of HAE for patients. In DME, an intravitreally administered plasma kallikrein inhibitor, called KVD001, has completed a Phase 2 clinical trial.

For more information about KalVista, please visit www.kalvista.com.
For more information on the KVD900 HAE on-demand Phase 3 KONFIDENT study, please visit www.konfidentstudy.com.
For more information on the KVD824 HAE prophylaxis Phase 2 KOMPLETE study, please visit www.kompletestudy.com.

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, including the potential impact of the ongoing COVID-19 pandemic, 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 its results, our ability to commence clinical studies or complete ongoing clinical studies, including our Phase 3 KONFIDENT and Phase 2 KOMPLETE clinical trials, and to obtain regulatory approvals for KVD900, KVD824 and other candidates in development, the ability of KVD900, KVD824 and other candidates in development to treat HAE or DME, 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, 2021, 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: www.businesswire.com/news/home/202203...

KalVista Pharmaceuticals, Inc.
Ben Palleiko
CBO & CFO
857-999-0890
investors@kalvista.com

Source: KalVista Pharmaceuticals, Inc.

bron:
ir.kalvista.com/news-releases/news-re...

[verwijderd] 11 juli 2023 11:50

0

Onderstaande overzicht ook weer verder bijgewerkt. Een aantal belangrijke updates:
- Toevoeging indeling van pathways behandeling reeds op de markt
- Zeer opvallende resultaten bij Donidalorsen
- 3 concurrenten naar Phase III, 1 klaar voor Phase III
- Gentherapieën zeer hoopgevend, zeker tussentijdse resultaten Intellia!
- Kalvista dicht bij markttoetreding, werving fase III konfident afgerond

Verder:
- Geen vermeldingen van preklinische onderzoekjes
- Ruconest profylaxe definitief verwijderd. Onderzoek gefaald.
- Arrowhead en Verseon en Adverum eruit wegens lange tijd geen gemelde progressie
- Alnylam eruit wanneer nog een jaar geen progressie

'Het Grote De Monitor - De Pharming Expert HAE Marktoverzicht'

Acuut

B2 Receptor

Firazyr (Takeda/Shire) – Bradykinin receptor antagonist - Subcutaan (FDA & EMA)

C1-INH

Ruconest (Pharming) – C1-INH - Intraveneus (FDA & EMA)

Berinert (CSL) – C1-INH - Intraveneus (FDA & EMA & Japan)

Cinryze (Takeda/Shire) – C1-INH - Intraveneus (EMA)

Plasma kallikrein

Kalbitor (Takeda/Shire) – Kallikrein inhibitor - Subcutaan (FDA & EMA)

Profylaxe

B2 Receptor
Nog geen

C1-INH

Cinryze (Takeda/Shire) – C1-INH - Intraveneus (FDA & EMA)

Haegarda (CSL) – C1-INH - Subcutaan (FDA & EMA & Canada)

Plasma kallikrein

Takhzyro/Lanadelumab (Takeda/Shire) - Kallikrein inhibitor - Subcutaan (FDA & EMA & Japan)

Orladeyo (Biocryst) - Oral capsule (US, China, Japan, VK, EMA, Chili). Patiëntpopulatie groeit hard (1000+ US)

In ontwikkeling

KVD900/Sebetralstat (Kalvista) - Acuut
- Orale toediening, kallikrein inhibitor
- Phase II liet zeer goede resultaten zien
- Today’s data show that KVD900 halts HAE attack progression and also provides rapid relief by shortening the time to symptom resolution
- Werving patiënten fase III KONFIDENT afgerond (114 patiënten)
- Fase III data voorzien H2/2023
- NDA gepland H1/2024
www.kalvista.com/products-pipeline/ka...

RAPIDe-1 (Pharvaris) – Acuut
- PHVS416 (deucrictibant) Softgel Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
- Gebaseerd op het al jaren bewezen Icatibant
- Versneld traject, afgeleide van de profylaxe toediening
- Phase II lopende, topline data 2023
- Eerste tussentijdse resultaten bij 74 patiënten zéér positief, goede verdraging bij alle doseringen
- Studie lopende in een twintigtal sites te Canada, Europa, Israel en de UK en VS
- Clinical hold opgeheven door FDA
- 2023: top-line Phase 2 CHAPTER-1 data, activation of our first ex-U.S. clinical sites for a Phase 3 on-demand study, and the submission of non-clinical toxicology data to the FDA
pharvaris.com/science/#hae

HAE CHAPTER-1 (Pharvaris) – Profylaxe
- PHVS416 (deucrictibant) Softgel Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
- Gebaseerd op het al jaren bewezen Icatibant
- 1 a 2 maal daags toedienen
- Phase II lopende, resultaten H2/2023
- Eerste B2 receptor op profylaxe markt
- 2023: top-line Phase 2 CHAPTER-1 data, activation of our first ex-U.S. clinical sites for a Phase 3 on-demand study, and the submission of non-clinical toxicology data to the FDA
pharvaris.com/science/#hae

PHVS719 (Pharvaris) – Profylaxe
- XR Tablet - Orale toediening. PHA121 maar in langzaam afgevende tabletvorm voor lange werking
- Gebaseerd op het al jaren bewezen Icatibant
- Phase I lopende
pharvaris.com/science/#hae

Oral Factor XIIa inhibitor (Kalvista) - Profylaxe
IND 2023
www.kalvista.com/products-pipeline

Donidalorsen (Ionis) - Profylaxe
- Phase II resultaten; significant sustained protection
- Two year open label extension study consistent efficacy and safety, overall reduction rates of 96%
- Overall sustained mean reduction of 95% attack rates, 99,6% of study days were HAE attack-free
- Hoge AE-QoL score
- Zeer veilig bleek na gebruik van meer dan een jaar
- Phase III enrollment completed, topline data H1/2024
- Market preperation ongoing
ionispharma.com/ionis-innovation/pipe...

STAR-0215 (Astria)
- ALPHA-STAR Phase 1b/2 loopt; resultaten mid 2024
- Monoclonal antibody inhibitor of plasma kallikrein for HAE
- Toediening eenmaal per drie maanden
- STAR-0215 heeft de potentie om de meest patiënt-vriendelijke optie te worden voor chronische behandeling
- Positieve Fase 1a resultaten
- Uitbreiding 1a Q1/2023 naar een halfjaarlijkse toediening, resultaten mid 2024
astriatx.com/our-science/star-0215/

ATN-249 (Attune) – Profylaxe
Orale toediening, kallikrein inhibitor. Phase I
attunepharma.com/assets/Attune-Phase-...

Garadacimab (CSL Behring) - Profylaxe - Recombinant monoclonal antibody
- Subcutane toediening om de 4 weken
- Garadacimab is a first-in-class, fully human, immunoglobulin G4 monoclonal antibody
- Phase III lopende
- Mean reduction number of attacks by 88.68% at a 75 mg dose, by 98.94% in those receiving 200 mg, and by 90.50% in those receiving 600 mg, compared with a placebo.
www.clinicaltrials.gov/ct2/show/NCT03...

ALN-F12 (Alnylam) - Profylaxe
Subcutane toediening
www.ncbi.nlm.nih.gov/pubmed/30463937

ARC-F12 (Arrowhead Pharmaceuticals) - Profylaxe
Subcutane toediening
ir.arrowheadpharma.com/static-files/9...

ADX-324 (ADARx Pharmaceuticals) - Profylaxe
Subcutane toediening - RNA technologie
Phase I is gestart (later meer uitleg)

Gentherapieën

CRISPR/Cas9 NTLA-2002 (Intellia Therapeutics) - Gentherapie
- Eenmalige toediening
- Tussentijdse resultaten eerste patiënten: aanvalvrij tot 5.5-10.6 maanden na eenmalige toediening
- Zeer zware gevallen laten zelfs tot 4 maanden aanvalvrije perioden zien
- Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment
- Regenerative Medicine Advanced Therapy (RMAT) designation
- Eerste patiënt behandeld in fase II studie, trial recruiting in Netherlands, UK, and New Zealand, with more sites to come
- FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002
ir.intelliatx.com/news-releases/news-...

BMN-331 (Biomarin) - Gentherapie
- Lopende studie open-label, Phase 1/2 HAERMONY trial to evaluate the safety and effectiveness of BMN 331
- Eenmalige toediening

RGX-314 (Regenxbio) - Gentherapie
- Eenmalige toediening - samenwerking met Neurimmune bouwt voort op veelbelovende resultaten met ons klinische RGX-314-programma
- A gene therapy product candidate utilizing NAV Vectors designed to deliver a gene encoding a therapeutic antibody targeting and binding to plasma kallikrein
- Veilig gebleken bij 85 patiënten in wet AMD studie
ir.regenxbio.com/news-releases/news-r...

[verwijderd] 26 september 2023 17:58

0

Een aantal bijzondere updates:
- Zeer opvallende resultaten bij Donidalorsen en Garadacimab
- Gentherapieën zeer hoopgevend, zeker tussentijdse resultaten Intellia!
- Opvallende progressie Intellia met gentherapie, mede door RMAT designation FDA
- Sebetralstat, Doniladorsen en Garadacimab dicht bij markttoetreding
- 2024 het jaar van duidelijkheid en 2024/2025 het jaar van introducties

'Het Grote De Monitor - De Pharming Expert HAE Marktoverzicht'

Acuut

B2 Receptor

Firazyr (Takeda/Shire) – Bradykinin receptor antagonist - Subcutaan (FDA & EMA)

C1-INH

Ruconest (Pharming) – C1-INH - Intraveneus (FDA & EMA)

Berinert (CSL) – C1-INH - Intraveneus (FDA & EMA & Japan)

Cinryze (Takeda/Shire) – C1-INH - Intraveneus (EMA)

Plasma kallikrein

Kalbitor (Takeda/Shire) – Kallikrein inhibitor - Subcutaan (FDA & EMA)

Profylaxe

B2 Receptor
Nog geen

C1-INH

Cinryze (Takeda/Shire) – C1-INH - Intraveneus (FDA & EMA)

Haegarda (CSL) – C1-INH - Subcutaan (FDA & EMA & Canada)

Plasma kallikrein

Takhzyro/Lanadelumab (Takeda/Shire) - Kallikrein inhibitor - Subcutaan (FDA & EMA & Japan)

Orladeyo (Biocryst) - Oral capsule (US, China, Japan, VK, EMA, Chili). Patiëntpopulatie groeit hard (1000+ US)

In ontwikkeling

KVD900/Sebetralstat (Kalvista) - Acuut
- Orale toediening, kallikrein inhibitor
- Phase II liet zeer goede resultaten zien
- Today’s data show that KVD900 halts HAE attack progression and also provides rapid relief by shortening the time to symptom resolution
- Werving patiënten fase III KONFIDENT afgerond (114 patiënten)
- Fase III data voorzien H2/2023
- NDA gepland H1/2024
- Continued to advance plans for the eventual worldwide launch of sebetralstat (bijzonder in Japan)

RAPIDe-1 (Pharvaris) – Acuut
- PHVS416 (deucrictibant) Softgel Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
- Gebaseerd op het al jaren bewezen Icatibant
- Versneld traject, afgeleide van de profylaxe toediening
- Phase II lopende, topline data 2023
- Eerste tussentijdse resultaten bij 74 patiënten zéér positief, goede verdraging bij alle doseringen
- Studie lopende in een twintigtal sites te Canada, Europa, Israel en de UK en VS
- Clinical hold opgeheven door FDA
- 2023: top-line Phase 2 CHAPTER-1 data, activation of our first ex-U.S. clinical sites for a Phase 3 on-demand study, and the submission of non-clinical toxicology data to the FDA

HAE CHAPTER-1 (Pharvaris) – Profylaxe
- PHVS416 (deucrictibant) Softgel Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
- Gebaseerd op het al jaren bewezen Icatibant
- 1 a 2 maal daags toedienen
- Enrollment completed in Phase 2 CHAPTER-1 prophylactic study; top-line data anticipated by YE2023
- Eerste B2 receptor op profylaxe markt
- 2023: top-line Phase 2 CHAPTER-1 data, activation of our first ex-U.S. clinical sites for a Phase 3 on-demand study, and the submission of non-clinical toxicology data to the FDA
- RAPIDe-3 Phase III anticipated initiation by YE2023

PHVS719 (Pharvaris) – Profylaxe
- XR Tablet - Orale toediening. PHA121 maar in langzaam afgevende tabletvorm voor lange werking
- Gebaseerd op het al jaren bewezen Icatibant
- Phase I lopende

Oral Factor XIIa inhibitor (Kalvista) - Profylaxe
Bringing the first candidate to clinical trials in 2023

Donidalorsen (Ionis) - Profylaxe
- Phase II resultaten; significant sustained protection
- Two year open label extension study consistent efficacy and safety, overall reduction rates of 96%
- Overall sustained mean reduction of 95% attack rates, 99,6% of study days were HAE attack-free
- Hoge AE-QoL score
- Zeer veilig bleek na gebruik van meer dan een jaar
- Completed enrollment of the Phase 3 OASIS-HAE study of donidalorsen in patients with hereditary angioedema; on track for data readout in H1:2024
- Reported positive topline Phase 2 open label extension data of donidalorsen in patients with hereditary angioedema treated for two years
- Market preperation ongoing

Garadacimab (CSL Behring) - Profylaxe - Recombinant monoclonal antibody
- Subcutane toediening om de 4 weken
- Garadacimab is a first-in-class, fully human, immunoglobulin G4 monoclonal antibody
- Phase III lopende
- Mean reduction number of attacks by 88.68% at a 75 mg dose, by 98.94% in those receiving 200 mg, and by 90.50% in those receiving 600 mg, compared with a placebo.
- Garadacimab (Anti-FXIIa), is being prepared for global regulatory submission.

STAR-0215 (Astria)
- ALPHA-STAR Phase 1b/2 loopt; resultaten mid 2024
- FDA Fasttrack Designation
- Monoclonal antibody inhibitor of plasma kallikrein for HAE
- Toediening eenmaal per drie maanden
- STAR-0215 heeft de potentie om de meest patiënt-vriendelijke optie te worden voor chronische behandeling
- Positieve Fase 1a resultaten
- Uitbreiding 1a Q1/2023 naar een halfjaarlijkse toediening, resultaten mid 2024

ATN-249 (Attune) – Profylaxe
- Orale toediening, kallikrein inhibitor. Phase I

ALN-F12 (Alnylam) - Profylaxe
- Subcutane toediening

ARC-F12 (Arrowhead Pharmaceuticals) - Profylaxe
- Subcutane toediening

ADX-324 (ADARx Pharmaceuticals) - Profylaxe
- Subcutane toediening - RNA technologie
- Phase I is gestart
- The early phase 1 clinical data strongly supports the potential of ADX-324 for patients suffering from hereditary angioedema.

Gentherapieën

CRISPR/Cas9 NTLA-2002 (Intellia Therapeutics) - Gentherapie
- Eenmalige toediening
- Tussentijdse resultaten eerste patiënten: aanvalvrij tot 5.5-10.6 maanden na eenmalige toediening
- Zeer zware gevallen laten zelfs tot 4 maanden aanvalvrije perioden zien
- Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment
- Regenerative Medicine Advanced Therapy (RMAT) designation
- Eerste patiënt behandeld in fase II studie, trial recruiting in Netherlands, UK, and New Zealand, with more sites to come
- FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002

BMN-331 (Biomarin) - Gentherapie
- Lopende studie open-label, Phase 1/2 HAERMONY trial to evaluate the safety and effectiveness of BMN 331
- Eenmalige toediening

RGX-314 (Regenxbio) - Gentherapie
- Eenmalige toediening - samenwerking met Neurimmune bouwt voort op veelbelovende resultaten met ons klinische RGX-314-programma
- A gene therapy product candidate utilizing NAV Vectors designed to deliver a gene encoding a therapeutic antibody targeting and binding to plasma kallikrein
- Veilig gebleken bij 85 patiënten in wet AMD studie

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