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Het Grote De Monitor - De Pharming Expert HAE Marktoverzicht

24 Posts, Pagina: 1 2 » | Laatste
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'Het Grote De Monitor - De Pharming Expert HAE Marktoverzicht'

Acuut
  • Ruconest (Pharming) – Humaan Recombinant C1-INH - Intraveneus (FDA & EMA)
  • Firazyr (Takeda/Shire) – Bradykinin receptor antagonist - Subcutaan (FDA & EMA)
  • Kalbitor (Takeda/Shire) – Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Berinert (CSL) – C1-INH - Intraveneus (FDA & EMA)
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (EMA)

Profylaxe
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (FDA & EMA)
  • Haegarda (CSL) – C1-INH - Subcutaan (FDA & EMA)
  • Takhzyro/Lanadelumab (Takeda/Shire) - Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Orladeyo (Biocryst) - Oral capsule (US, China, VK, FR - EMA 03/2021)

In ontwikkeling
  • Ruconest (Pharming) – C1-INH (US) – Profylaxe
    Start in 2021 Start onbekend, tezamen met intradermale toediening

    Micro needle patch on hold, profylaxe uit outlook
    www.pharming.com/pipeline
  • PHVS416 (Pharvaris) – Acuut
    - 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 gestart, eerste patiënt behandeld feb 2021, resultaten 2022
    pharvaris.com/science/#hae
  • PHVS416 (Pharvaris) – Profylaxe
    - 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 in de loop van 2021, resultaten 2022
    - FDA Acceptance IND App april 2021
    pharvaris.com/science/#hae
  • PHVS719 (Pharvaris) – Profylaxe
    - Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
    - Gebaseerd op het al jaren bewezen Icatibant
    - Phase I start in 2021, resultaten 2022
    pharvaris.com/science/#hae
  • KVD900 (Kalvista) - Acuut
    - Orale toediening, kallikrein inhibitor.
    - Eerste Phase II data gepubliceerd
    - Today’s data show that KVD900 halts HAE attack progression and also provides rapid relief by shortening the time to symptom resolution
    www.kalvista.com/products-pipeline/ka...

  • KVD824 (Kalvista) - Profylaxe
    Filed the IND for a Phase 2 clinical trial of KVD824 and expect to initiate that trial in the second quarter of 2021
    www.kalvista.com/products-pipeline
  • IONIS-PKK-LRx (Ionis) - Profylaxe
    - Phase II
    - The study demonstrated a mean reduction of 97% in the number of monthly HAE attacks in weeks five to 17.
    - In weeks five to 17, 92% of patients treated with IONIS-PKK-LRx were attack-free
    ionispharma.com/ionis-innovation/pipe...
  • ATN-XXX (backup) (Attune) - nog onbekend?


Gentherapieën
  • CRISPR/Cas9 NTLA-2002 (Intellia Therapeutics) - Gentherapie
    - Eenmalige toediening
    - IND H2 2021
    - Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment
    angioedemanews.com/2020/02/13/intelli...
  • 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
    ir.regenxbio.com/news-releases/news-r...
voda
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quote:

voda schreef op 6 mei 2021 16:03:


Bedankt. Nu hoef je dit overzicht niet oeverloos x keren op de maandraad te plaatsen.


Dus het is een probleem dat ik dit herplaats als ik de lijst heb aangepast? Het is natuurlijk wel veel interessanter dan lappen van koerslijstjes die je heel gemakkelijk op euronext kunt terugvinden natuurlijk.
CashCow
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quote:

De Monitor - Pharming Professor schreef op 6 mei 2021 16:30:


[...]

Dus het is een probleem dat ik dit herplaats als ik de lijst heb aangepast? Het is natuurlijk wel veel interessanter dan lappen van koerslijstjes die je heel gemakkelijk op euronext kunt terugvinden natuurlijk.


Nou, ik typte ze altijd over en dan maakte ik daar een grafiek van en die hield ik dan naast die van Euronext. En dan waren ze hetzelfde, ik bedoel, dat lijkt me toch belangrijk. Ergens.
LL
0
Over gentechnology voor HAE gesproken Biomarin kwam met zijn Q2 cijfers. Hieronder een snippet van de belangrijkste zaken:

BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin's third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.

Bron:
finance.yahoo.com/news/biomarin-annou...
LL
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Intellia Therapeutics Announces Second Quarter 2021 HAE Highlights

NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s modular in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce plasma kallikrein protein and activity, a key mediator of HAE. This 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 June, Intellia announced that it had submitted its first Clinical Trial Application (CTA) to the New Zealand Medicines and Medical Devices Safety Authority for NTLA-2002 to initiate a first-in-human study.

The Company expects to enroll the first patient by year-end and is also submitting additional regulatory applications to enable enrollment in other countries. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE, and will continue to leverage insights gained from the development of NTLA-2001.

bron:
finance.yahoo.com/news/intellia-thera...
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Pharvaris:


  • Phase 2 on-demand study (RAPIDe-1) of PHVS416 proceeding toward data readout in 2022. In February 2021, Pharvaris announced that enrollment had commenced in its Phase 2 clinical study of PHVS416 for the on-demand treatment of HAE attacks. An IND for the on-demand RAPIDe-1 study is in effect, expanding enrollment into the United States. Data from this trial is expected to be reported in 2022.
  • Phase 2 prophylactic study (HAE CHAPTER-1) of PHVS416 to begin in 2021. In addition to developing PHVS416 for the on-demand treatment of HAE attacks, the company plans to investigate the therapeutic potential of PHVS416 for the prophylactic prevention of HAE attacks. In April 2021, Pharvaris announced that an IND was in effect in the US and expects to initiate the study in 2021.
  • Phase 1 pharmacokinetics study of PHVS719 to begin by the end of 2021. PHVS719 is an extended-release formulation of PHA121 intended for use in the prophylactic treatment of HAE. The company expects to initiate a Phase 1 pharmacokinetics study by the end of 2021.
  • Presentations of clinical data of PHA121 at medical meetings. Data detailing PHA121’s pharmacokinetic (PK), pharmacodynamic (PD), and safety profiles were presented at the 12th C1 Inhibitor Deficiency and Angioedema Workshop in June 2021 and at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in July 2021. PHA121, the active ingredient in PHVS416 and PHVS719, was well-tolerated and showed a favorable PK profile up to the highest dose tested.
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Intellia

NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s modular in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce plasma kallikrein protein and activity, a key mediator of HAE. This 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 June, Intellia announced that it had submitted its first Clinical Trial Application (CTA) to the New Zealand Medicines and Medical Devices Safety Authority for NTLA-2002 to initiate a first-in-human study.
  • The Company expects to enroll the first patient by year-end and is also submitting additional regulatory applications to enable enrollment in other countries. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE, and will continue to leverage insights gained from the development of NTLA-2001.
LL
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Catabasis

Catabasis expects first results of clinical trial end 2022

Catabasis Pharmaceuticals, Inc. reports financial results for the second quarter ended 30 June 2021 and provides a corporate update.

“We are focused on advancing the development of our lead program, QLS-215, as a differentiated and potentially the most patient-friendly treatment option for the chronic treatment of patients with HAE to prevent attacks,” says Jill C. Milne, Ph.D., CEO of Catabasis. “We anticipate that our first clinical trial with QLS-215 could demonstrate clinical proof of concept of its differentiated profile and long antibody half-life. Initial results from this trial are anticipated by the end of 2022.”

QLS-215 for the Treatment of HAE

The vision for the lead program, QLS-215, is to develop a monoclonal antibody inhibitor of plasma kallikrein for HAE with dosing once every three months or longer and sustained inhibitory blood levels. QLS-215 has the potential to be the most patient-friendly chronic treatment option, based on the data generated to date and the existing HAE treatment landscape.
QLS-215 is a humanized monoclonal antibody targeting plasma kallikrein that has demonstrated potent inhibition of plasma kallikrein as well as a long plasma half-life in non-human primates.
Recent discussions with physicians and patients confirm the need for effective treatments that reduce HAE attacks as well as reduce the burden of treatment.
Catabasis expects to file an Investigational New Drug application for QLS-215 in mid-2022 and plans to initiate a Phase 1 clinical trial with initial results anticipated by year end 2022. Catabasis expects that the results of this trial, if positive, could provide clinical proof of concept for the activity and plasma half-life improvements for QLS-215.
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Update:





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



Acuut
  • Ruconest (Pharming) – Humaan Recombinant C1-INH - Intraveneus (FDA & EMA)
  • Firazyr (Takeda/Shire) – Bradykinin receptor antagonist - Subcutaan (FDA & EMA)
  • Kalbitor (Takeda/Shire) – Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Berinert (CSL) – C1-INH - Intraveneus (FDA & EMA)
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (EMA)

Profylaxe
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (FDA & EMA)
  • Haegarda (CSL) – C1-INH - Subcutaan (FDA & EMA & Canada onlangs)
  • Takhzyro/Lanadelumab (Takeda/Shire) - Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Orladeyo (Biocryst) - Oral capsule (US, China, Japan, VK, EMA)

In ontwikkeling
  • Ruconest (Pharming) – C1-INH (US) – Profylaxe
    Start in 2021 Start onbekend, tezamen met intradermale toediening

    Micro needle patch gaat niet meer door, dus profylaxe waarschijnlijk ook niet
    Huidige versie Ruconest niet geschikt voor andere toedieningsvorm, wachten op 'Ruconest-lite'
    www.pharming.com/pipeline
  • PHVS416 (Pharvaris) – Acuut
    - 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 gestart, eerste patiënt behandeld feb 2021, resultaten 2022
    - Studie uitgebreid naar de US
    pharvaris.com/science/#hae
  • PHVS416 (Pharvaris) – Profylaxe
    - 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 start in 2021, resultaten 2022
    - FDA Acceptance IND App april 2021
    pharvaris.com/science/#hae
  • PHVS719 (Pharvaris) – Profylaxe
    - Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
    - Gebaseerd op het al jaren bewezen Icatibant
    - extended release form. PHA121 voor profylaxe gebruik
    - Phase I start in 2021, resultaten 2022
    pharvaris.com/science/#hae
  • KVD900 (Kalvista) - Acuut
    - Orale toediening, kallikrein inhibitor.
    - Eerste Phase II data gepubliceerd
    - Today’s data show that KVD900 halts HAE attack progression and also provides rapid relief by shortening the time to symptom resolution
    www.kalvista.com/products-pipeline/ka...

  • IONIS-PKK-LRx (Ionis) - Profylaxe
    - Phase II
    - The study demonstrated a mean reduction of 97% in the number of monthly HAE attacks in weeks five to 17.
    - In weeks five to 17, 92% of patients treated with IONIS-PKK-LRx were attack-free
    ionispharma.com/ionis-innovation/pipe...
  • QLS-215 (Catabasis)
    NDA mid-2022, Phase I trial end 2022, Phase 1b/2 results end 2023
    - Monoclonal antibody inhibitor of plasma kallikrein for HAE
    - Dosing once every three months or longer and sustained inhibitory blood levels
    - QLS-215 has the potential to be the most patient-friendly chronic treatment option
    www.catabasis.com/our-science/qls-215...
  • ATN-XXX (backup) (Attune) - nog onbekend?


Gentherapieën
  • CRISPR/Cas9 NTLA-2002 (Intellia Therapeutics) - Gentherapie
    - Eenmalige toediening
    - Juni 2021: Clinical Trial Application (CTA) to the New Zealand Medicines and Medical Devices Safety Authority for NTLA-2002 to initiate a first-in-human study
    - Eerste patiënt verwacht eind 2021
    - Meer autoriteiten aanvragen om studie uit te breiden naar meerdere landen
    - Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment
    angioedemanews.com/2020/02/13/intelli...
  • 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
    ir.regenxbio.com/news-releases/news-r...




Mocht er nog wat missen, hoor ik het graag.
LL
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KalVista Pharmaceuticals

CAMBRIDGE, Mass. & SALISBURY, England, August 23, 2021--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of small molecule protease inhibitors, today provided an update on clinical trial progress for KVD824 in development for oral prophylactic treatment of hereditary angioedema (HAE).

"Over the past month we have made substantial progress in commencing KOMPLETE, our worldwide Phase 2 clinical trial of KVD824 as a potential oral prophylactic therapy for HAE," said Andrew Crockett, Chief Executive Officer of KalVista. "The regulatory submissions have been approved in Canada, Australia, and the UK, with patient enrollment expected to begin this quarter. We also submitted our clinical hold response to the FDA related to the US IND filing for KVD824 and will provide further updates once we have additional information."

KOMPLETE is the Phase 2 clinical trial of KVD824, and is a randomized, double-blind, parallel group design evaluating twice-daily dosing of 300 mg, 600 mg, and 900 mg KVD824 against placebo for 12 weeks. The trial will enroll 48 HAE patients randomized into four equal arms after they report experiencing a minimum of three attacks in an eight-week run-in period. The primary endpoint of the trial is the rate of investigator confirmed HAE attacks during the treatment period. Secondary endpoints include the proportion of participants without investigator confirmed HAE attacks and the rate of investigator confirmed HAE attacks that require conventional treatment. KOMPLETE will be conducted at more than 30 sites in 13 countries.

To date, a total of 121 subjects have been exposed to treatment with KVD824 as single doses up to 1280 mg and up to 14 days of twice-daily dosing of 600 mg and 900 mg. The formulation of KVD824 maintains the plasma concentrations that we believe are required to deliver efficacy consistent with approved injectable therapies. Twice-daily dosing of KVD824 up to 14 days has demonstrated an encouraging safety and tolerability profile.

bron:
finance.yahoo.com/news/kalvista-pharm...
LL
0
BioCryst Announces Acceptance of Regulatory Applications for ORLADEYO® (berotralstat) by Health Canada and Swissmedic

BioCryst Pharmaceuticals, Inc.
Wed, August 25, 2021, 10:01 PM
In this article:

RESEARCH TRIANGLE PARK, N.C., Aug. 25, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the new drug submission for ORLADEYO® (berotralstat) has been accepted for review by Health Canada for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older. The company also announced that Swissmedic has accepted BioCryst’s marketing authorization application for ORLADEYO for review.

“We are making significant progress with our goal to bring ORLADEYO to HAE patients around the world,” said Jon Stonehouse, president and chief executive officer of BioCryst. “Currently there are no targeted oral HAE therapies available in Canada or Switzerland for patients living with HAE. If approved, ORLADEYO has the potential to be an important new treatment option for Canadian and Swiss HAE patients and physicians.”
LL
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KalVista Pharmaceuticals Reports First Fiscal Quarter Results

CAMBRIDGE, Mass. & SALISBURY, England, September 09, 2021--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of small molecule protease inhibitors, today provided an operational update and released financial results for the first fiscal quarter ended July 31, 2021.

"We have made excellent progress in the rollout of our Phase 2 KOMPLETE clinical trial for KVD824," said Andrew Crockett, Chief Executive Officer of KalVista. "Site initiations are underway, and patients are being enrolled in the trial to evaluate KVD824 as a potential oral prophylactic treatment for HAE. We will be having an end-of-Phase 2 meeting with the FDA later this month regarding KVD900, our oral on-demand candidate for treatment of HAE attacks and are ready to initiate the Phase 3 study quickly afterwards. We look forward to advancing both of these compounds as we continue with our strategy of bringing a full spectrum of oral treatment options to HAE patients."

First Fiscal Quarter and Recent Business Highlights:

Presented Phase 2 data for KVD900 in late-breaking session at European Academy of Allergy and Clinical Immunology (EAACI) Congress. The data showed that a single on-demand treatment with orally administered KVD900 significantly slows progression and accelerates resolution of attacks in patients with hereditary angioedema (HAE). KalVista also presented four other posters at EAACI related to the HAE clinical landscape and unmet needs, as well as preclinical data from other oral molecules.

Scheduled an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) to review the proposed development plans for KVD900, KalVista’s oral on-demand candidate for treatment of HAE attacks, expected to take place in September 2021. KVD900 also received Orphan Drug Designation from the FDA.

Presented data at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress showcasing small molecule Factor XIIa inhibitor research.

Provided a progress update on the KVD824 Phase 2 KOMPLETE Clinical Trial. KVD824 is in development for oral prophylactic treatment of HAE. Regulatory submissions have been approved in Canada, Australia, and the UK with patient enrollment now underway. KalVista also submitted a response to the clinical hold related to the U.S. Investigational New Drug (IND) filing for KVD824 and is prepared to initiate the study in the U.S. upon clearance from the FDA.

Presented KVD001 data at American Chemical Society (ACS) Meeting.


finance.yahoo.com/news/kalvista-pharm...
LL
0
BioCryst Announces Approval of ORLADEYO® (berotralstat) in United Arab Emirates

BioCryst Pharmaceuticals, Inc.
Thu, September 9, 2021, 1:00 PM
In this article:

Explore the topics mentioned in this article

Company selects NewBridge Pharmaceuticals as regional distributor in Gulf Cooperation Council

RESEARCH TRIANGLE PARK, N.C., Sept. 09, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Ministry of Health and Prevention (MOHAP) in the United Arab Emirates (UAE) has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older. To support commercialization efforts in the UAE, BioCryst has entered into a supply and distribution agreement with NewBridge Pharmaceuticals (NewBridge), which also covers the Gulf Cooperation Council (GCC) and Iraq.

“As the first targeted oral, once-daily treatment, ORLADEYO provides an important new treatment option for patients and physicians,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE. “While there is still more work to be done to raise awareness to support earlier diagnosis and treatment, the approval of ORLADEYO is an important advancement for HAE patients in the UAE.”

“With many prevalent rare diseases in the MENA region, I am personally inspired, and we at NewBridge are proud, to be part of this partnership with BioCryst for the UAE and a number of other markets in the GCC. This partnership supports our mission by providing access to an important new therapy for HAE patients in a hope that we can help ease their suffering and support them to live better lives,” said Joe Henein, president and chief executive officer of NewBridge Pharma.

“NewBridge is the right partner for BioCryst as they share our vision to bring innovative medicines to patients living with rare diseases,” said Charlie Gayer, chief commercial officer of BioCryst. “With experience across regulatory, medical and commercial, and strong local relationships with key stakeholders, NewBridge will help accelerate our efforts to bring ORLADEYO to patients across the globe by providing a much-needed new option to HAE patients in the UAE.”

NewBridge Pharmaceuticals, headquartered in Dubai, UAE, is a regional specialty company with a comprehensive pharmaceutical platform of services and expertise, established to bridge the access gap and partner with global pharma and biotech companies to in-license and commercialize U.S. Food and Drug Administration or European Medicines Agency approved innovative therapeutics that address unmet medical needs into the Middle East and North Africa (MENA) regions.

finance.yahoo.com/news/biocryst-annou...
LL
0
Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema

NTLA-2002 is the first single-dose genome editing therapeutic candidate designed to prevent attacks in people living with HAE to enter clinical study

NTLA-2002 is Intellia’s second in vivo CRISPR genome editing therapeutic candidate; program to leverage platform insights gained from ongoing development of NTLA-2001 for transthyretin (ATTR) amyloidosis

On track to initiate patient enrollment by year-end

CAMBRIDGE, Mass., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced the authorization of its Clinical Trial Application (CTA) by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with hereditary angioedema (HAE). HAE is a genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. NTLA-2002 is a systemically administered single-dose CRISPR/Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to permanently reduce plasma kallikrein activity and thus prevent HAE attacks.

“We look forward to initiating this year our first-in-human study of NTLA-2002 for people living with HAE, a debilitating disorder that causes frequent, potentially life-threatening attacks,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We believe NTLA-2002 has the potential to be a curative therapy for patients with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and eliminating the significant treatment burden associated with currently available HAE therapies. This study of NTLA-2002 leverages early insights from our ATTR amyloidosis program, where we established proof-of-concept for our modular in vivo genome editing platform with interim Phase 1 data earlier this year. The NTLA-2002 program represents the second systemic in vivo CRISPR genome editing therapy candidate to enter human clinical trials.”

The 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. More information about the study will be available at clinicaltrials.gov.

Beyond New Zealand, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development approach for NTLA-2002.

bron:
finance.yahoo.com/news/intellia-thera...
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Onderstaande overzicht ook weer verder bijgewerkt.

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

Acuut
  • Ruconest (Pharming) – Humaan Recombinant C1-INH - Intraveneus (FDA & EMA)
  • Firazyr (Takeda/Shire) – Bradykinin receptor antagonist - Subcutaan (FDA & EMA)
  • Kalbitor (Takeda/Shire) – Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Berinert (CSL) – C1-INH - Intraveneus (FDA & EMA)
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (EMA)

Profylaxe
  • Cinryze (Takeda/Shire) – C1-INH - Intraveneus (FDA & EMA)
  • Haegarda (CSL) – C1-INH - Subcutaan (FDA & EMA)
  • Takhzyro/Lanadelumab (Takeda/Shire) - Kallikrein inhibitor - Subcutaan (FDA & EMA)
  • Orladeyo (Biocryst) - Oral capsule (US, China, VK, FR - EMA 03/2021)

In ontwikkeling
  • Ruconest (Pharming) – C1-INH (US) – Profylaxe
    Start in 2021 Start onbekend, tezamen met intradermale toediening

    Micro needle patch gaat niet meer door, dus profylaxe waarschijnlijk ook niet
    Wachten op 'Ruconest-Lite', waarvoor 8000 vials nodig zijn om te verifiëren
    www.pharming.com/pipeline
  • PHVS416 (Pharvaris) – Acuut
    - 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 gestart, eerste patiënt behandeld feb 2021, resultaten 2022
    pharvaris.com/science/#hae
  • PHVS416 (Pharvaris) – Profylaxe
    - 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 in de loop van 2021, resultaten 2022
    - FDA Acceptance IND App april 2021
    pharvaris.com/science/#hae
  • PHVS719 (Pharvaris) – Profylaxe
    - Orale toediening, klein molekuul en daardoor snel werkzaam (15-30min).
    - Gebaseerd op het al jaren bewezen Icatibant
    - Phase I start in 2021, resultaten 2022
    pharvaris.com/science/#hae
  • KVD900 (Kalvista) - Acuut
    - Orale toediening, kallikrein inhibitor.
    - Eerste Phase II data gepubliceerd
    - Today’s data show that KVD900 halts HAE attack progression and also provides rapid relief by shortening the time to symptom resolution
    www.kalvista.com/products-pipeline/ka...

  • KVD824 (Kalvista) - Profylaxe
    Filed the IND for a Phase 2 clinical trial of KVD824 and expect to initiate that trial in the second quarter of 2021
    www.kalvista.com/products-pipeline
  • IONIS-PKK-LRx (Ionis) - Profylaxe
    - Phase II
    - The study demonstrated a mean reduction of 97% in the number of monthly HAE attacks in weeks five to 17.
    - In weeks five to 17, 92% of patients treated with IONIS-PKK-LRx were attack-free
    ionispharma.com/ionis-innovation/pipe...
  • ATN-XXX (backup) (Attune) - nog onbekend?
  • STAR-0215 (Astria)
    Voorheen Catabasis, hernoemd naar Astria
    - STAR-0215, voorheen QLS-215
    - NDA Mid/2022, Phase I results end 2022, Phase 1b/2 results end 2023
    - Therapy has a similar mechanism of action to Takhzyro, but more potent and greater stability in the bloodstream.
    - Binding to kalikrein is 10 times stronger, therapy 10 times more potent than Takhzyro at supressing the enzyme's activity by 90%.
    - Half-time was much longer than Takhzyro (33.6 vs 10.5 days)
    - STAR-0215 is predicted to remain above the minimum therapeutic levels for more than 84 days, while Takhzyro’s levels are expected to fall below this therapeutic threshold after approximately 10 days
    www.catabasis.com/our-science/qls-215...
    angioedemanews.com/2021/09/27/catabas...


Gentherapieën
  • CRISPR/Cas9 NTLA-2002 (Intellia Therapeutics) - Gentherapie
    - Eenmalige toediening
    - Okt 21: Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema
    - Aims to reduce plasma kallikrein activity to prevent excess bradykinin production leading to HAE attacks after a single course of treatment
    angioedemanews.com/2020/02/13/intelli...
  • 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
    ir.regenxbio.com/news-releases/news-r...
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BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BioMarin is leveraging its broad expertise in gene therapy to improve efficiencies in the development process of BMN 331. The IND for BMN 331 has been allowed by FDA and the first patient is expected to be enrolled around the end of 2021. Patients with HAE may experience benefit from the constitutive expression and stable C1-INH protein levels potentially provided by gene therapy, thus reducing the frequency and severity of attacks and reducing the burden associated with current standard of care.

bron:
investors.biomarin.com/2021-10-27-Bio...
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Phase 2 study results of Ionis' novel antisense treatment for hereditary angioedema to be presented at ACAAI annual meeting
November 7, 2021 at 7:05 AM EST
- Positive Phase 2 study results demonstrate significant efficacy of donidalorsen (formerly IONIS-PKK-LRx) in the reduction of hereditary angioedema attacks
- Based on the results of the Phase 2 study, Ionis plans to initiate a Phase 3 program of donidalorsen in people with HAE

CARLSBAD, Calif., Nov. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today that positive results from the Phase 2 study of its investigational antisense medicine, donidalorsen (formerly IONIS-PKK-LRx), will be presented at the American College of Asthma, Allergy & Immunology (ACAAI) Annual Scientific Meeting in New Orleans and via livestream, November 4-8. The Phase 2 study results support the clinical profile of donidalorsen as a potential, best-in-class prophylactic treatment for patients with hereditary angioedema (HAE), and underscore Ionis' commitment to advancing antisense technology to target the root cause of diseases.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

HAE is a rare and potentially fatal autosomal dominant disease that results in recurrent, painful attacks of swelling affecting the hands, feet, limbs, face, abdomen, larynx and trachea. 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 HAE. Donidalorsen was developed using Ionis' advanced LIgand-Conjugated Antisense (LICA) technology.

Topline results of the Phase 2 study, reported earlier this year, showed that donidalorsen met its primary and all secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with hereditary angioedema (HAE) compared to placebo. These data support advancing donidalorsen into Phase 3 development, which Ionis plans to initiate this year.

bron:
ir.ionispharma.com/news-releases/news...
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BioCryst Presents New Data Showing Sustained Reduction of HAE Attack Rates and Improved Patient Satisfaction After Patients Switch to ORLADEYO® (berotralstat)


Data from APeX-S showed patients had more than 80 percent attack free months after switching to ORLADEYO from injectable prophylactic therapies

RESEARCH TRIANGLE PARK, N.C., Nov. 05, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new long-term efficacy and safety data from studies evaluating oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE), including results showing sustained reduction of HAE attack rates and improved patient satisfaction after patients switched to ORLADEYO monotherapy from injectable prophylactic therapies (lanadelumab and C1 inhibitors).

The data are being presented at the 2021 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted in New Orleans, Louisiana, and online, from November 4-8, 2021.

“We continue to see improvement in key indicators that demonstrate the value of oral, once-daily ORLADEYO as an excellent option for patients who want alternatives to injectable prophylactic therapies, which carry a high treatment burden,” said Dr. William Sheridan, chief medical officer of BioCryst. “The data we are presenting at ACAAI further support ORLADEYO as a transformative therapy for HAE patients, including those who are already well-controlled on other therapies.”

“The data from APeX-S showed that HAE patients who switched to ORLADEYO from an injectable prophylactic therapy had more than 80 percent attack free months,” said Marc Riedl, M.D., clinical director of the US HAEA Angioedema Center at the University of California San Diego. “These data show that, regardless of which therapy patients switched from, or when they switched, ORLADEYO provided consistently low attack rates when used as a monotherapy. These important findings add to real-world evidence that this oral, once-daily therapy is a beneficial treatment option for many HAE patients.”

“Increased satisfaction and improvement in quality of life continue to be major driving factors for patients who decide to switch to an oral prophylactic option, as shown in our analysis of patients who switched from injectable prophylaxis to ORLADEYO in APeX-2,” said Jonathan Bernstein, M.D., professor of medicine, department of internal medicine, division of allergy & immunology at the University of Cincinnati and partner of the Bernstein Allergy Group and Bernstein Clinical Research Center. “These results underscore what I see every day in clinical practice, that HAE patients want a more convenient treatment option to control their HAE attacks and reduce their overall burden of therapy.”

BioCryst ACAAI 2021 Presentation Highlights

Consistently Low Hereditary Angioedema (HAE) Attack Rates Observed in US Patients Treated with Berotralstat (Distinguished Industry Oral Presentation)

Highlights safety, effectiveness and patient-reported outcomes of ORLADEYO 150 mg in U.S. patients from APeX-S who completed 12 months of treatment (n=71).

Following initiation of ORLADEYO, patients experienced low HAE attack rates that were sustained throughout the treatment period (median attack rate of 0.0 attacks per month through month 12), consistent with previously reported data.

ORLADEYO was associated with prompt, sustained, statistically significant and clinically meaningful improvements in quality of life (-10.8 change from baseline at month 1 and -13.6 change from baseline at months 6 and 12; p<0.001) as measured by the AE-QoL (angioedema quality of life questionnaire) total score. These improvements exceeded the minimal clinically important difference (-6 change).

ORLADEYO was generally well tolerated, with no drug-related serious adverse events reported.

Berotralstat Demonstrates Low Hereditary Angioedema (HAE) Attack Rates in Patients Switching from Injectable Prophylaxis (poster #P045)

Patients at U.S. sites in APeX-S who switched to ORLADEYO (n=34) had more than 80 percent attack free months for up to 12 months after switching from long-term prophylactic treatment (LTP) (lanadelumab and C1 inhibitors).

After switching from prior injectable prophylaxis, mean monthly attack rates were consistently low (< 0.5 attacks per month) and median attack rates remained at 0.0 attacks per month throughout 12 months of treatment with ORLADEYO monotherapy.

The transition from injectable LTP to ORLADEYO was generally well tolerated with no additional safety signals.

Improved Patient Satisfaction with Berotralstat in Patients Switching from Injectable Hereditary Angioedema (HAE) Prophylactic Treatments (poster #P048)

Treatment satisfaction in patients who switched to ORLADEYO monotherapy from prior injectable LTP at U.S. sites in APeX-S was reported as assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM).

Statistically significant improvements were observed in convenience and global satisfaction scores in patients who switched from lanadelumab or a subcutaneous C1 inhibitor to ORLADEYO (n=34), consistent with a positive experience using an oral HAE prophylactic therapy.

For all patients who switched to ORLADEYO, the most significant improvement was observed in convenience, with scores of more than 90 points at month 12 (33.4 point improvement; p<0.001).

From baseline to month 12, effectiveness scores remained consistently high, supporting patients’ perception that ORLADEYO is as effective as their prior prophylactic therapy.

Sustained Reduction in Hereditary Angioedema (HAE) Attack Rates Following Switch to Berotralstat: Subgroup Analysis from APeX-2 (poster #P053)

Long-term efficacy and safety data were analyzed in patients who switched to ORLADEYO 150 mg for parts 2 and 3 of the APeX-2 pivotal clinical trial after receiving placebo in part 1 of the trial (n=17).

A rapid reduction in attack rates was observed following the switch to ORLADEYO, with median attack rates of 0.0 attacks per month at more than 75 percent of all timepoints.

Sustained reductions in mean and median HAE attack rates were observed after patients switched to ORLADEYO.

ORLADEYO was generally well tolerated in patients who switched from placebo; safety data in this switch subset were consistent with those previously reported for the overall study patient population.

All e-posters are available online at epostersonline.com/acaai2021 and will be on display in the exhibit hall during the meeting.

bron:
ir.biocryst.com/news-releases/news-re...
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KalVista Pharmaceuticals Presents Data Demonstrating KVD900 Achieves Rapid Exposure and Improves Outcomes as Oral On-Demand Treatment of HAE
November 8, 2021 at 6:30 AM EST


– KVD900 Phase 2 data presented at American College of Allergy, Asthma & Immunology Annual Scientific Meeting –

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Nov. 8, 2021-- 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 presented at American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting. Data presentations included an oral presentation and poster presentation on Phase 2 data for KVD900, KalVista’s lead program for oral on-demand treatment of hereditary angioedema (HAE) attacks.

“Current guidelines recommend effective on-demand therapy for every patient with HAE to reduce symptom severity and attack duration,” said Jonathan Bernstein, FAAAAI, FACAAI, FACP, M.D., University of Cincinnati College of Medicine and Bernstein Clinical Research Center, LLC. “Treatment of HAE attacks with KVD900 achieved rapid plasma exposure which was associated with faster improvements in initial symptom relief compared with placebo. As the first oral on-demand treatment to demonstrate this early therapeutic effect for patients, KVD900 may represent a remarkable advancement for management of the disease.”

Oral Presentation: On-Demand Oral Treatment with KVD900 for HAE Attacks Achieves Rapid Exposures and Improves Patient Outcomes

KVD900 was rapidly absorbed, with measurable concentrations detected within 15 minutes
Plasma levels reached peak concentration within 1 hour of administration
Median time to symptom improvement was significantly shorter with KVD900 than with placebo (1.6 vs 9.0 hours, p<0.0001), as indicated by the Patient Global Impression of Change (PGI-C) scale
A significantly higher percentage of patients also rated their HAE attack symptoms as improved within 12 and 24 hours with KVD900 compared with placebo

Poster Title: Relationship Between PGI-C Scale and Other Patient Reported Outcomes (PROs) in KVD900 Trial in HAE

In a Phase 2 trial, several PROs were collected to capture the patient experience
60 patients completed treatment for at least one attack (n=113 attacks). PGI-C scoring of “a little better” or higher at two consecutive timepoints had 97% sensitivity for composite visual analogue scale (VAS) and Patient Global Impression of Severity (PGI-S) improvement.
Moderate to substantial agreement between PGI-C and the other measures suggests that improvement on PGI-C was clinically significant from the patients’ perspective.

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