This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

开始日期2021-12-10

申办/合作机构

Intellia Therapeutics, Inc.

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100 项与 Lonvoguran 相关的专利（医药）

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项与 Lonvoguran 相关的文献（医药）

2024-04-01·The journal of allergy and clinical immunology. In practice

Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema

Article

作者: Riedl, Marc A ; Bordone, Laura ; Newman, Kenneth B ; Revenko, Alexey ; Cohn, Danny M

Hereditary angioedema (HAE) is typically caused by a deficiency of the protease inhibitor C1 inhibitor (C1INH). The absence of C1INH activity on plasma kallikrein and factor XIIa leads to overproduction of the vasoactive peptide bradykinin, with resulting angioedema. As the primary site of C1INH and prekallikrein production, the liver is recognized as an important therapeutic target in HAE, leading to the development of hepatic-focused treatment strategies such as GalNAc-conjugated antisense technology and gene modification. This report reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials. Donidalorsen is an investigational GalNAc₃-conjugated antisense oligonucleotide that binds to prekallikrein mRNA in the liver and reduces the expression of prekallikrein. Phase 2 data with subcutaneous donidalorsen demonstrated a significant reduction in HAE attack rate compared with placebo. Phase 3 trials are underway. ADX-324 is a GalNAc₃-conjugated short-interfering RNA being investigated in HAE. BMN 331 is an investigational AAV5-based gene therapy vector that expresses wild-type human C1INH and is targeted to hepatocytes. A single intravenous dose of BMN 331 is intended to replace the defective SERPING1 gene and enable patients to produce functional C1INH. A first-in-human phase 1/2 study is ongoing with BMN 331. NTLA-2002 is an investigational in vivo clustered regularly interspaced short palindromic repeats/Cas9-based therapy designed to knock out the prekallikrein-coding KLKB1 gene in hepatocytes; a phase 1/2 study is ongoing. Findings from these and other ongoing studies are highly anticipated with the expectation of expanding the array of treatment options in HAE.

2024-02-01·The New England journal of medicine1区 · 医学

CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema

1区 · 医学

Article

作者: Lindsay, Karen ; Gurugama, Padmalal ; Lebwohl, David ; Soukamneuth, Samantha ; Han, Bo W ; Shah, Mrinal Y ; Golden, Adele ; McKee, Mark D ; Sepp-Lorenzino, Laura ; Petersen, Remy S ; Xu, Yuanxin ; Leonard, John ; Maitland, Michael L ; Boiselle, Carri ; Longhurst, Hilary J ; Maag, David ; Vogel, Joseph D ; Butler, James S ; Fijen, Lauré M ; Cohn, Danny M ; Abdelhady, Ahmed M ; Clark, Eliana D ; Seitzer, Jessica

BACKGROUND:

Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (KLKB1), with the goal of lifelong control of angioedema attacks after a single dose.

METHODS:

In this phase 1 dose-escalation portion of a combined phase 1-2 trial of NTLA-2002 in adults with hereditary angioedema, we administered NTLA-2002 at a single dose of 25 mg, 50 mg, or 75 mg. The primary end points were the safety and side-effect profile of NTLA-2002 therapy. Secondary and exploratory end points included pharmacokinetics, pharmacodynamics, and clinical efficacy determined on the basis of investigator-confirmed angioedema attacks.

RESULTS:

Three patients received 25 mg of NTLA-2002, four received 50 mg, and three received 75 mg. At all dose levels, the most common adverse events were infusion-related reactions and fatigue. No dose-limiting toxic effects, serious adverse events, grade 3 or higher adverse events, or clinically important laboratory findings were observed after the administration of NTLA-2002. Dose-dependent reductions in the total plasma kallikrein protein level were observed between baseline and the latest assessment, with a mean percentage change of -67% in the 25-mg group, -84% in the 50-mg group, and -95% in the 75-mg group. The mean percentage change in the number of angioedema attacks per month between baseline and weeks 1 through 16 (primary observation period) was -91% in the 25-mg group, -97% in the 50-mg group, and -80% in the 75-mg group. Among all the patients, the mean percentage change in the number of angioedema attacks per month from baseline through the latest assessment was -95%.

CONCLUSIONS:

In this small study, a single dose of NTLA-2002 led to robust, dose-dependent, and durable reductions in total plasma kallikrein levels, and no severe adverse events were observed. In exploratory analyses, reductions in the number of angioedema attacks per month were observed at all dose levels. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830.).

101

项与 Lonvoguran 相关的新闻（医药）

2024-08-08

·GlobeNewswire-Health Care

Intellia Therapeutics Announces Second Quarter 2024 Financial Results and Highlights Recent Company Progress

Phase 2 study of NTLA-2002 for hereditary angioedema (HAE) met its primary and all secondary endpoints; plan to present detailed results at an upcoming medical meeting in the fourth quarterSelected the 50 mg dose of NTLA-2002 for the pivotal Phase 3 trial on track to begin in 2H 2024Rapid enrollment continues in the Phase 3 MAGNITUDE trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathyOn track to initiate the Phase 3 study of NTLA-2001 for the treatment of hereditary ATTR amyloidosis with polyneuropathy by year-endPlan to present new clinical data from the ongoing NTLA-2001 Phase 1 in 2H 2024 Expect to dose the first patient in the Phase 1/2 study of NTLA-3001, an in vivo gene insertion candidate for the treatment of alpha-1 antitrypsin deficiency (AATD) in 2H 2024Ended the second quarter of 2024 in a strong financial position with approximately $940 million in cash CAMBRIDGE, Mass., Aug. 08, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the second quarter ended June 30, 2024. “We are delighted to report the Phase 2 study of NTLA-2002 met its primary efficacy and all secondary endpoints at both dose levels and, importantly, provided clear support for advancing the 50 mg dose into the pivotal Phase 3 trial in patients with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Based on these positive results and our recent successful end-of-Phase 2 meeting with the FDA, we see a clear path to initiating the Phase 3 trial in the coming months. We look forward to presenting the detailed Phase 2 results at a medical meeting in the fourth quarter as we continue to advance what we believe could be a functional cure for hereditary angioedema. With three pivotal Phase 3 trials and our first gene insertion trial expected to be active by year-end, Intellia is closer than ever to transforming the future of medicine with our one-time, in vivo gene editing therapies.” Second Quarter 2024 and Recent Operational Highlights Hereditary Angioedema (HAE) NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose. Intellia announced today positive topline results from the Phase 2, randomized, double-blind, placebo-controlled study of NTLA-2002 in patients with HAE. The clinical trial met its primary efficacy and all secondary endpoints in the 16-week primary observation period, with a single 25 mg or 50 mg dose leading to deep reductions in attacks. No new safety findings were observed. Intellia has selected the 50 mg dose for further evaluation in the global pivotal Phase 3 trial based upon the greater number of patients with complete attack elimination and greater kallikrein protein reduction compared to the 25 mg dose observed in the Phase 2 study, which is consistent with the previously reported Phase 1 results. The Company plans to present the detailed Phase 2 data at an upcoming medical meeting in the fourth quarter of this year.The Company announced today the successful completion of an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) supporting its Phase 3 plans for NTLA-2002. The Phase 3 study is on track to initiate in the second half of 2024, subject to regulatory feedback.In June, Intellia presented positive long-term data from the ongoing Phase 1 study. Eight of 10 patients remained completely attack-free following the 16-week primary observation period. These patients have experienced ongoing attack-free durations of greater than 18 months after a single-dose treatment, with the longest ongoing individual attack-free duration reaching over 26 months. Across all patients, NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate. Consistent with previously reported results, NTLA-2002 was well-tolerated, with the majority of adverse events being mild in severity through the latest follow-up. These interim data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024 in Valencia, Spain. Transthyretin (ATTR) Amyloidosis NTLA-2001: NTLA-2001, now known as nexiguran ziclumeran (nex-z), is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Intellia leads development and commercialization of NTLA-2001 in collaboration with Regeneron. ATTR Amyloidosis with Cardiomyopathy (ATTR-CM): The pivotal Phase 3 MAGNITUDE trial is enrolling rapidly and continues to track ahead of the Company’s initial projections. During the second quarter, the Company received approval for its application under the new European Union Clinical Trials Regulation, which enables the Phase 3 trial to proceed in Denmark, Germany, France, Italy, Spain and Sweden. The MAGNITUDE trial of NTLA-2001 is now cleared by regulatory agencies in over 12 countries and actively enrolling at over 35 sites globally. Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN): The Company plans to initiate a pivotal Phase 3 trial of NTLA-2001 as a single-dose treatment for ATTRv-PN by year-end. As previously announced, the study is expected to be a small, placebo-controlled trial conducted at ex-U.S. sites with approximately 50 ATTRv-PN patients. Intellia plans to present updated data from the ongoing Phase 1 study in the second half of 2024. Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease NTLA-3001: NTLA-3001 is a first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert the wild-type SERPINA1 gene, which encodes the alpha-1 antitrypsin (AAT) protein, with the potential to restore permanent expression of fully functional AAT protein to normal levels after a single dose. This is Intellia’s first wholly owned gene insertion program. In July, Intellia announced the authorization of its Clinical Trial Application by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a first-in-human study of NTLA-3001. Intellia expects to dose the first patient in the Phase 1/2 study of NTLA-3001 in the second half of 2024. In Vivo Platform Expansion In June, Intellia presented positive clinical proof-of-concept data that redosing with CRISPR, utilizing the Company’s proprietary non-viral, LNP-based delivery platform, enabled an additive pharmacodynamic effect. In the three patients who previously received the lowest dose of 0.1 mg/kg in the Phase 1 dose-escalation study of NTLA-2001, follow-on dosing with a 55 mg dose of NTLA-2001 led to a deeper protein reduction. Median reduction in serum TTR was 90% at day 28 after redosing. The corresponding reduction from original baseline levels was a 95% median reduction in serum TTR. NTLA-2001 was generally well tolerated across all patients after receiving the follow-on dose. While redosing is not planned for the NTLA-2001 program in ATTR amyloidosis, a redosing option could be an important advantage of Intellia’s LNP-based delivery platform for future investigational therapies where a target additive effect is desired. These data were presented at the Peripheral Nerve Society (PNS) Annual Meeting in Montreal, Canada.Intellia is expanding the range of diseases that can be targeted with its CRISPR-based technologies by deploying new editing and delivery innovations. This includes advancing gene editing programs in five different tissues outside the liver, either independently or in collaboration with partners. These research and preclinical programs are targeting diseases that originate in the bone marrow, brain, muscle, lung and eye, which, if successful, could dramatically expand the opportunities for CRISPR-based treatments. Ex Vivo Program Updates Intellia is advancing multiple programs, wholly owned and in collaboration with partners, utilizing its allogeneic platform for the treatment of immuno-oncology and autoimmune diseases. The Company’s proprietary allogeneic cell engineering platform avoids both T cell- and NK cell-mediated rejection in preclinical models, a key unsolved challenge with other investigational allogeneic approaches. Cell therapies engineered with Intellia’s allogeneic platform, combined with edits to enhance cell function, offer a new approach to target both hematological and solid tumors. Corporate Updates In June, Intellia announced the appointment of Brian Goff to its board of directors. Mr. Goff joins the board of directors with over three decades of commercialization, operations and sales and marketing experience at leading biopharmaceutical companies.In June, Intellia announced the appointment of Edward Dulac as Executive Vice President, Chief Financial Officer, and Treasurer, effective July 22, 2024. Mr. Dulac succeeds Glenn Goddard, who stepped down from his role effective June 30, 2024. Mr. Dulac joins Intellia with more than 20 years of combined finance, business development and corporate strategy experience. Upcoming Events The Company will participate in the following events during the third quarter of 2024: Morgan Stanley 22nd Annual Global Healthcare Conference, September 4, New YorkWells Fargo Healthcare Conference, September 4, BostonCantor Global Healthcare Conference, September 17, New York Second Quarter 2024 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $939.9 million as of June 30, 2024, compared to $1.0 billion as of December 31, 2023. The decrease was driven by cash used to fund operations of $234.4 million. The Company’s investments were offset in part by $96.4 million of net equity proceeds from the Company’s “At the Market” (ATM) program, $35.9 million of reimbursement from collaborators, including a one-time $30.0 million payment received in April 2024 related to the Company’s technology collaboration with Regeneron, $25.1 million of interest income and $4.8 million in proceeds from employee-based stock plans. The cash position is expected to fund operations into late 2026.Collaboration Revenue: Collaboration revenue was $7.0 million during the second quarter of 2024, compared to $13.6 million during the second quarter of 2023. The $6.6 million decrease was mainly driven by a reduction in revenue related to the AvenCell license and collaboration agreement.R&D Expenses: Research and development (R&D) expenses were $114.2 million during the second quarter of 2024, compared to $115.3 million during the second quarter of 2023. The $1.1 million decrease was primarily driven by a decrease in employee-related expenses. Stock-based compensation expense included in R&D expenses was $25.4 million for the second quarter of 2024.G&A Expenses: General and administrative (G&A) expenses were $31.8 million during the second quarter of 2024, compared to $30.7 million during the second quarter of 2023. The $1.1 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in G&A expenses was $15.4 million for the second quarter of 2024.Net Loss: Net loss was $147.0 million for the second quarter of 2024, compared to $123.7 million during the second quarter of 2023. Conference Call to Discuss Second Quarter 2024 Results The Company will discuss these results on a conference call today, Thursday, August 8 at 8 a.m. ET. To join the call: U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.Please visit this link for a simultaneous live webcast of the call. A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on August 8 at 12 p.m. ET. About Intellia TherapeuticsIntellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx. Forward-Looking Statements This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations concerning: the safety, efficacy, success and advancement of its clinical programs for NTLA-2001, now known as nexiguran ziclumeran or “nex-z”, for transthyretin (“ATTR”) amyloidosis, NTLA-2002 for the treatment of hereditary angioedema (“HAE”), and NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (“AATD”)-associated lung disease, pursuant to its clinical trial applications (“CTA”) and investigational new drug (“IND”) submissions, including the expected timing of data releases, regulatory feedback, regulatory filings, and the initiation, enrollment, dosing and completion of clinical trials, such as the presentation of additional data from the NTLA-2001 and NTLA-2002 clinical trials in 2024, its ability to rapidly enroll the Phase 3 MAGNITUDE study, the planned initiation of its Phase 3 trial of NTLA-2001 for the treatment of hereditary ATTR amyloidosis with polyneuropathy by year-end, the planned initiation of a global pivotal Phase 3 study of NTLA-2002 in 2H 2024, its ability to dose the first patient in its NTLA-3001 Phase 1 study in 2024, the potential of NTLA-2001 to halt and reverse disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose, the potential of NTLA-2002 to be a functional cure for patients with HAE and to demonstrate lifelong control of HAE attacks after a single dose, and the potential of NTLA-3001 to restore permanent expression of functional alpha-1 antitrypsin protein to normal levels after a single dose; the expansion of its CRISPR/Cas9 technology and related novel technologies, including advancing gene editing programs in tissues outside of the liver, such as bone marrow, brain, muscle, lung and eye, and the potential of those programs to dramatically expand the opportunities for CRISPR-based treatments; its ability to advance multiple ex vivo programs utilizing its allogeneic platform, which is designed to avoid both T cell- and NK cell-mediated rejection, for the treatment of immuno-oncology and autoimmune diseases, and to combine its allogeneic platform with edits to enhance cell function and offer a new approach to target both hematological and solid tumors; its ability to advance additional in vivo and ex vivo development candidates and timing expectations of advancing such development candidates and releasing data related to such technologies and development candidates; its ability to optimize the impact of its collaborations on its development programs, including, but not limited to, its collaboration with Regeneron Pharmaceuticals, Inc. (“Regeneron”) and their co-development programs for ATTR amyloidosis; and its growth as a Company and expectations regarding its uses of capital, expenses, future accumulated deficit and financial results. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of preclinical and clinical studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the ability to develop and commercialize any one or more of Intellia’s product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; risks related to the potential delay of planned clinical trials due to regulatory feedback or other developments; and risks related to Intellia’s collaborations with Regeneron, or its other collaborations not continuing or not being successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. INTELLIA THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) (Amounts in thousands, except per share data) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Collaboration revenue $6,957 $13,594 $35,892 $26,200 Operating expenses: Research and development 114,207 115,276 226,054 212,392 General and administrative 31,793 30,652 62,884 58,100 Total operating expenses 146,000 145,928 288,938 270,492 Operating loss (139,043) (132,334) (253,046) (244,292) Other (expense) income, net: Interest income 12,422 12,653 25,054 24,633 Change in fair value of investments, net (20,354) - (26,419) - Loss from equity method investment - (4,000) - (7,048) Change in fair value of contingent consideration - - - (100) Total other (expense) income, net (7,932) 8,653 (1,365) 17,485 Net loss$(146,975) $(123,681) $(254,411) $(226,807) Net loss per share, basic and diluted$(1.52) $(1.40) $(2.64) $(2.58) Weighted average shares outstanding, basic and diluted 96,975 88,185 96,238 87,979 INTELLIA THERAPEUTICS, INC. CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) (Amounts in thousands) June 30, 2024 December 31, 2023 Cash, cash equivalents and marketable securities $939,873 $1,012,087 Total assets 1,191,536 1,300,977 Total liabilities 220,474 250,808 Total stockholders’ equity 971,062 1,050,169 Intellia Contacts: Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communicationsian.karp@intelliatx.com Lina LiSenior Director, Investor Relations and Corporate Communicationslina.li@intelliatx.com Media:Matt CrensonTen Bridge Communicationsmedia@intelliatx.com mcrenson@tenbridgecommunications.com

临床结果临床2期临床3期临床1期财报

2024-08-08

·BioPharmaDive

Merck TIGIT drug fails another trial; Intellia therapy succeeds in HAE study

Today, a brief rundown of news from Merck & Co. and Cabaletta Bio, as well as updates from Intellia Therapeutics, Vertex Pharmaceuticals and Novartis that you may have missed.An experimental Merck & Co. drug aimed at the cancer immunotherapy target TIGIT has failed another clinical trial, the company said Thursday. Independent trial monitors advised Merck to stop a Phase 3 study testing a co-formulation of the medicine, vibostolimab, and its drug Keytruda in advanced small-cell lung cancer because treatment was unlikely to extend survival compared to an immunotherapy and chemotherapy regimen and was associated with higher rates of side effects, Merck said. The vibostolimab-Keytruda combination has already fallen short in studies for lung and skin cancer. Ben FidlerShares of cell therapy developer Cabaletta Bio fell 34% Thursday after the company disclosed a clinical trial participant with lupus who received its autoimmune disease cell therapy experienced a life-threatening neurological side effect. Cabaletta said the adverse event, a known cell therapy complication called ICANS, resolved quickly following treatment with an antiseizure medication. A committee of investigators has recommended the study continue at the current dose level. Nonetheless, the incident will fuel the commercial debate on the feasibility of broadly using cell therapies on autoimmune conditions, wrote Stifel analyst Benjamin Burnett. Ben FidlerA gene editing therapy from Intellia Therapeutics met the primary and secondary goals of a Phase 2 trial testing it in people with hereditary angioedema, the company said Thursday, without disclosing specific data. According to Intellia, both tested doses of its medicine, dubbed NTLA-2002, led to deep reductions in the swelling attacks that characterize HAE. The data build on prior results from Phase 1 testing, and led Intellia to choose a 50 milligram dose for a Phase 3 study it plans to start later this year. The company will disclose the full Phase 2 findings in the fourth quarter. Ned PagliaruloVertex Pharmaceuticals and the National Health Service in England have reached an agreement on reimbursement for the companys CRISPR therapy Casgevy in people with transfusion-dependent beta thalassemia. Vertex did not disclose the agreed-upon price, but noted the funding for patient access comes from NHS Englands Innovative Medicines Fund. Discussions on reimbursement of Casgevy for sickle cell disease are ongoing, the company said. Casgevy has a $2.2 million list price in the U.S. Ned PagliaruloThe Food and Drug Administration has granted accelerated approval to Novartis drug Fabhalta to treat people with primary IgA nephropathy, a rare disease in which the bodys immune system attacks the kidneys. Its the second U.S. approval for Fabhalta, which secured an initial clearance in paroxysmal nocturnal hemoglobinuria late last year. The OK in IgAN was based on data showing that treatment lowered urine protein levels, a surrogate marker of organ benefit. Novartis is now evaluating whether that finding translates to a slower decline in kidney function. Ned Pagliarulo '

细胞疗法上市批准临床2期临床3期免疫疗法

2024-06-09

·药明康德

疾病控制率达100%的免疫联合疗法；使月度发作率下降98%的基因编辑疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 新型口服双重抑制剂TU2218联用PD-1抑制剂pembrolizumab治疗晚期实体瘤患者，在一项1b期临床试验中的疾病控制率（DCR）达100%。 2. 用于治疗遗传性血管水肿（HAE）的在研体内CRISPR基因编辑疗法NTLA-2002早期临床数据亮眼，单次给药后使患者的月度发作率平均下降了98%。 3. 现货型基因疗法PRGN-2012治疗复发性呼吸乳头状瘤病（RRP）患者的1/2期临床试验达到主要终点，有望成为首个获得FDA批准的用于治疗RRP的疗法。 4. 男性无激素避孕药YCT-529完成了1a期临床试验，在临床前研究中，该候选疗法预防小鼠妊娠的有效率为99%，且100%可逆。药明康德内容团队整理 TU2218：公布1b期临床试验的中期数据 TiumBio公司公布了新型口服双重抑制剂TU2218联用PD-1抑制剂pembrolizumab治疗晚期实体瘤患者1b期临床试验的中期结果。TU2218靶向转化生长因子β受体1（TGFR1）和血管内皮生长因子受体2（VEGFR2）。肿瘤微环境中的TGF-ß和VEGF通路会干扰免疫检查点抑制剂的抗肿瘤活性，因此TU2218有望通过抑制这两种通路最大限度地提高免疫疗法的疗效。此次公布的结果显示，5名接受2期推荐剂量（RP2D）TU2218联用pembrolizumab治疗的患者的DCR为100%，其中两名患者达到了部分缓解（PR），3名患者达到了疾病稳定（SD）。所有剂量组中，12名接受联合治疗的患者中有2人达到了PR，6人达到了SD。在这项研究中，TU2218联用pembrolizumab的耐受性良好，没有观察到剂量限制性毒性（DLT），这与之前的单药治疗试验一致。 NTLA-2002：公布1期临床试验的长期数据 Intellia Therapeutics公司宣布其用于治疗遗传性血管水肿的在研疗法NTLA-2002的1期临床试验的长期数据。NTLA-2002是一种体内CRISPR基因编辑候选疗法，通过脂质纳米颗粒（LNP）以mRNA形式递送CRISPR-Cas9基因编辑系统，靶向敲除血浆前激肽释放酶（KLKB1）基因，以永久性降低血浆中激肽释放酶活性，从而防止遗传性血管水肿的发作。此次公布的结果显示，中位随访时间为20.1个月时，单次NTLA-2002治疗让患者的月均疾病发作率平均减少了98%，中度至重度发作平均减少了99%。在测试的每个剂量水平上，HAE发作率均显著减少。单次输注后，患者最长的无发作期超过26个月，并且仍在持续。此外，100%接受NTLA-2002治疗的患者在停止其他预防性治疗后无需再进行预防性治疗。 PRGN-2012：公布1/2期临床试验的新数据 Precigen公司公布了其现货型基因疗法PRGN-2012治疗复发性呼吸乳头状瘤病患者的1/2期临床试验数据。PRGN-2012旨在激发对抗受HPV6或HPV11感染细胞的免疫反应，以治疗RRP。此前，PRGN-2012已获得FDA和欧盟委员会授予的孤儿药资格，还获得了FDA授予的突破性疗法认定。此次公布的结果显示，该1/2期临床试验达到了主要的安全性和疗效终点。中位随访时间为20个月时，51%（18/35）接受PRGN-2012治疗的患者达到了完全缓解（CR），且不再需要进行手术治疗，CR持续时间超过12个月。86%的患者在接受PRGN-2012治疗后的一年内需要进行的手术次数较治疗前一年减少，从治疗前一年的中位次数4次减少到接受治疗后的0次。此外，PRGN-2012显著改善了达到CR患者的Derkay评分（一种用于测量RRP生长程度的量表）和生活质量评分。安全性方面，PRGN-2012的耐受性良好，无DLT，也没有＞2级的治疗相关不良事件。 ▲PRGN-2012的1/2期临床试验结果（图片来源：参考资料[9]）该公司计划在2024年下半年通过加速批准途径提交PRGN-2012的滚动生物制品许可申请（BLA）。新闻稿指出，如果获得批准，PRGN-2012有可能成为首个获得FDA批准用于治疗RRP的疗法。 YCT-529：公布1a期临床试验数据 YourChoice Therapeutics公司公布其男性无激素避孕药YCT-529的1a期临床试验结果。YCT-529是一种视黄酸受体-α（RAR-a）抑制剂，通过阻断RAR-α来阻止睾丸中精子的产生和释放。临床前研究表明，YCT-529在预防小鼠妊娠方面有99%的有效性，且100%可逆。该研究的结果表明，YCT-529是安全的。 C-CAR031：公布1期临床试验的初步数据阿斯利康（AstraZeneca）公布其GPC3靶向CAR-T疗法C-CAR031的最新临床试验结果。GPC3是一种在肝癌中过表达的表面抗原，该蛋白在健康组织中几乎不存在。C-CAR031是一种GPC3靶向的自体CAR-T疗法，这款CAR-T疗法还同时“装备”了TGFβ以抵抗免疫抑制微环境，以增强其效力。此次公布的结果显示，中位随访时间为5.82个月时，22例可评估疗效的GPC3阳性晚期肝细胞癌（HCC）患者的DCR为90.9%，客观缓解率为50.0%，且该疗法的安全性可控。阿斯利康的早期肿瘤学开发负责人Matt Hellmann博士在接受行业媒体STAT采访时表示：“这是首个在肝癌治疗上显示具疗效的CAR-T疗法。这也是GPC3靶点的首次临床验证。” ▲C-CAR031的疗效结果摘要（图片来源：参考资料[1]） CB-010：公布1期临床试验数据 Caribou Biosciences公司公布了其同种异体CAR-T细胞疗法CB-010治疗复发/难治性B细胞非霍奇金淋巴瘤患者的新数据。CB-010利用基因编辑技术生成，除了表达靶向CD19的嵌合抗原受体，还利用基因编辑技术敲除了免疫检查点蛋白PD-1的表达，旨在提高抗肿瘤活性的持久性。截至2024年4月1日的数据，CB-010的疗效和安全性可与已获批的自体CAR-T细胞疗法相媲美。13名部分人类白细胞抗原（HLA）匹配（≥4个等位基因）患者的总缓解率达92%，CR率为46%，中位无进展生存期（PFS）获得改善，为14.4个月。 ▲接受CB-010治疗患者的无进展生存曲线（图片来源：参考资料[4]） ▲接受CB-010治疗患者的疗效结果（图片来源：参考资料[4]） INB-200：公布1期临床试验的新数据 IN8bio公司公布其耐化疗的转基因自体γδ T细胞疗法INB-200用于治疗新确诊的多形性胶质母细胞瘤的积极早期数据。INB-200是一种强大的协同治疗方法，能在患者接受化疗时持续存在，并保持其识别、参与和杀死癌细胞的自然能力。截至2024年5月30日的数据，中位随访时间为11.7个月，92%接受INB-200治疗的可评估患者的中位PFS超过了7个月（即接受标准治疗方案的患者的中位PFS）。一名患有IDH突变神经胶质瘤的患者在接受治疗后的34.9+个月时仍然存活，且疾病没有进展。近期发表的一项IDH抑制剂治疗IDH突变脑胶质瘤患者的临床试验中，对照组患者的中位PFS为11.1个月，试验组患者的中位PFS为28.5个月。安全性方面，在任何队列中均未报告与治疗相关的严重不良事件、DLT、细胞因子释放综合征、输注反应或免疫效应细胞相关神经毒性综合征。 IFx-2.0：公布1b期临床试验数据 TuHURA Biosciences公司公布了其个体化的候选癌症疫苗IFx-2.0治疗对免疫检查点抑制剂（ICI）耐药的晚期Merkel细胞癌（MCC）和皮肤鳞状细胞癌（cSCC）患者的1b期试验的初步结果。该候选疗法旨在克服患者对ICI的原发性耐药。此次公布的结果显示，连续三周每周给药一次IFx-2.0的耐受性良好。5名此前接受ICI治疗后在3.8个月内疾病就发生进展的晚期MCC患者中，有4人在接受IFx-2.0治疗后重新对ICI治疗产生了持久的应答，包括1例CR、1例病理完全缓解（pCR）和两例PR。患者的平均缓解时间为25个月，其中两名患者已分别持续缓解19个月和23个月，并且仍在持续缓解中。 MDNA11：公布1/2期临床试验的新数据 Medicenna Therapeutics公司公布了其下一代长效IL-2超级激动剂MDNA11治疗晚期实体瘤的新临床结果。MDNA11具有优秀的CD122（IL-2受体β）结合作用，而没有CD25（IL-2受体α）亲和力，从而能优先刺激癌症杀伤效应T细胞和NK细胞。此次公布的结果显示，MDNA11的耐受性良好，没有报告DLT和血管渗漏综合征。MDNA11在胰腺癌患者中表现出持久的缓解，1例转移性胰腺导管腺癌患者在基线时存在的所有病灶均完全消退，已维持超过104周，在停止治疗4个月后仍然保持着缓解。一例双检查点抑制剂治疗后进展的黑色素瘤患者的靶病灶在第28周时完全消退，在第44周的扫描时靶病灶仍保持完全消退。目前，该患者仍在接受MDNA11的治疗，非靶病灶也在继续消退。 WTX-124：公布1/1b期临床试验数据 Werewolf Therapeutics公司公布了其条件激活的白介素-2（IL-2）候选疗法WTX-124治疗接受过ICI治疗的局部晚期或转移性实体瘤患者的1/1b期临床试验数据。结果显示，WTX-124在此类患者中具有临床活性，且耐受性良好。截至2024年5月1日的数据，接受WTX-124单药治疗的患者中有1人获得了持久并经过确认的CR，2人达到了PR。获得缓解的患者在治疗的前两个治疗周期内就出现了缓解，并且靶病灶实现了100%的消退。此外，没有发现WTX-124联用pembrolizumab出现新的安全信号。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Phase I study of C-CAR031, a GPC3-specific TGFβRIIDN armored autologous CAR-T, in patients with advanced hepatocellular carcinoma (HCC). 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Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603691086/en [25] Cyclacel Pharmaceuticals Reports New Clinical Data at 2024 ASCO Annual Meeting Highlighting Oral Fadraciclib’s Potential as a Precision Medicine for Cancer. Retrieved June 7, 2024, from https://investor.cyclacel.com/news-releases/news-release-details/cyclacel-pharmaceuticals-reports-new-clinical-data-2024-asco [26] Obsidian Therapeutics Announces Additional OBX-115 Safety and Efficacy Data in Oral Presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603008329/en [27] Replimune Presents RP1 Data from the IGNYTE anti-PD1 Failed Melanoma Cohort and RP2 Data in Uveal Melanoma at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://ir.replimune.com/news-releases/news-release-details/replimune-presents-rp1-data-ignyte-anti-pd1-failed-melanoma [28] RS Oncology Announces Positive Data from a Phase 1 Clinical Trial of RSO-021, a First-in-Class Therapeutic for Malignant Pleural Mesothelioma. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603559175/en [29] Ascendis Pharma Presents New Data and Updated Results from Phase 1/2 IL-Believe Trial at ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892565/0/en/Ascendis-Pharma-Presents-New-Data-and-Updated-Results-from-Phase-1-2-IL-Believe-Trial-at-ASCO-2024.html [30] Scholar Rock Presents New Data from SRK-181 Phase 1 DRAGON Trial at ASCO 2024 Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603004778/en [31] invoX Pharma Presents Positive Clinical Data from Phase 1 Study of FS222 in Patients with Advanced Solid Tumours at the 2024 American Society of Clinical Oncology Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603068133/en [32] Eledon Presents Updated Data from Ongoing Phase 1b Trial Evaluating Tegoprubart for Prevention of Rejection in Kidney Transplantation. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892558/0/en/Eledon-Presents-Updated-Data-from-Ongoing-Phase-1b-Trial-Evaluating-Tegoprubart-for-Prevention-of-Rejection-in-Kidney-Transplantation.html [33] ImmuneOncia Announces Biomarker Results from Phase 1 Clinical Trial of CD47 Antibody at ASCO. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603017832/en [34] Entera Bio Reports Phase 1 Clinical Data of First-in-Class, Oral PTH(1-34) Peptide Candidate (EB612) for Patients with Hypoparathyroidism at ENDO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892199/0/en/Entera-Bio-Reports-Phase-1-Clinical-Data-of-First-in-Class-Oral-PTH-1-34-Peptide-Candidate-EB612-for-Patients-with-Hypoparathyroidism-at-ENDO-2024.html [35] Enlivex Receives Regulatory Authorization for the Initiation of a Placebo-Controlled Phase I/II Trial Evaluating Allocetra in Up To 46 Patients with Thumb Osteoarthritis. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892206/0/en/Enlivex-Receives-Regulatory-Authorization-for-the-Initiation-of-a-Placebo-Controlled-Phase-I-II-Trial-Evaluating-Allocetra-in-Up-To-46-Patients-with-Thumb-Osteoarthritis.html [36] Indaptus Therapeutics Announces New Positive Data from Ongoing Phase 1 Trial of Decoy20. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892212/0/en/Indaptus-Therapeutics-Announces-New-Positive-Data-from-Ongoing-Phase-1-Trial-of-Decoy20.html [37] Alkermes Presents Data From Phase 1b Study of ALKS 2680 Demonstrating Improved Wakefulness in Patients With Narcolepsy Type 1 at SLEEP 2024. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/alkermes-presents-data-from-phase-1b-study-of-alks-2680-demonstrating-improved-wakefulness-in-patients-with-narcolepsy-type-1-at-sleep-2024-302161336.html [38] IDRx Reports Updated Preliminary Phase 1 Data from Ongoing Phase 1/1b StrateGIST 1 Trial Supporting Best-in-Class Potential for IDRX-42 in Patients with GIST. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240531639390/en [39] Century Therapeutics Presents Interim Results from Phase 1 ELiPSE-1 Study at ASCO 2024 Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892097/0/en/Century-Therapeutics-Presents-Interim-Results-from-Phase-1-ELiPSE-1-Study-at-ASCO-2024-Annual-Meeting.html [40] MediciNova Announces Data from Phase 1b/2a Clinical Trial of MN-166 (ibudilast) in Glioblastoma Patients at the American Society of Clinical Oncology (ASCO) Annual Meeting 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892064/7767/en/MediciNova-Announces-Data-from-Phase-1b-2a-Clinical-Trial-of-MN-166-ibudilast-in-Glioblastoma-Patients-at-the-American-Society-of-Clinical-Oncology-ASCO-Annual-Meeting-2024.html [41] FibroGen Announces FDA Clearance of Investigational New Drug Application for FG-3165, a Galectin-9 Targeting Monoclonal Antibody, for the Treatment of Patients with Solid Tumors. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892568/0/en/FibroGen-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-FG-3165-a-Galectin-9-Targeting-Monoclonal-Antibody-for-the-Treatment-of-Patients-with-Solid-Tumors.html [42] STORM Therapeutics Presented Interim Phase 1 Clinical Data on its METTL3 RNA Methyltransferase Inhibitor STC-15 at ASCO 2024. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/storm-therapeutics-presented-interim-phase-1-clinical-data-on-its-mettl3-rna-methyltransferase-inhibitor-stc-15-at-asco-2024-302160864.html [43] Black Diamond Therapeutics Presents Promising BDTX-1535 Clinical Data in Patients with Recurrent Glioblastoma at 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://investors.blackdiamondtherapeutics.com/news-releases/news-release-details/black-diamond-therapeutics-presents-promising-bdtx-1535-clinical [44] Immunocore reports updated Phase 1 data of brenetafusp (IMC-F106C), an ImmTAC bispecific targeting PRAME, in immune checkpoint pre-treated cutaneous melanoma patients at ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/05/31/2891735/0/en/Immunocore-reports-updated-Phase-1-data-of-brenetafusp-IMC-F106C-an-ImmTAC-bispecific-targeting-PRAME-in-immune-checkpoint-pre-treated-cutaneous-melanoma-patients-at-ASCO-2024.html [45] Kymera Therapeutics Presents New Clinical Data from Ongoing Phase 1 Trial of MDM2 Degrader KT-253 at ASCO Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/01/2891812/0/en/Kymera-Therapeutics-Presents-New-Clinical-Data-from-Ongoing-Phase-1-Trial-of-MDM2-Degrader-KT-253-at-ASCO-Annual-Meeting.html [46] Accutar Biotechnology Presents Phase 1 Data of AC699 Monotherapy in Patients with ER+ / HER2- Breast Cancer at ASCO 2024. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240601126229/en [47] Molecular Partners Presents Positive Data From Completed Phase 1 Trial Of MP0317 (FAP X CD40 DARPin) Monotherapy In Patients With Advanced Solid Tumors At ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/01/2891809/0/en/Molecular-Partners-Presents-Positive-Data-From-Completed-Phase-1-Trial-Of-MP0317-FAP-X-CD40-DARPin-Monotherapy-In-Patients-With-Advanced-Solid-Tumors-At-ASCO-2024.html [48] Updated Data from the Phase 1/2 Study of Olomorasib in KRAS G12C-Mutant Advanced Solid Tumors Presented at the 2024 ASCO® Annual Meeting. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/updated-data-from-the-phase-12-study-of-olomorasib-in-kras-g12c-mutant-advanced-solid-tumors-presented-at-the-2024-asco-annual-meeting-302160829.html [49] ALX Oncology Presents First Evorpacept Combination Data with an Antibody-Drug Conjugate from Phase 1 ASPEN-07 Clinical Trial in Patients with Advanced Bladder Cancer. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/02/2891851/0/en/ALX-Oncology-Presents-First-Evorpacept-Combination-Data-with-an-Antibody-Drug-Conjugate-from-Phase-1-ASPEN-07-Clinical-Trial-in-Patients-with-Advanced-Bladder-Cancer.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床结果基因疗法免疫疗法临床2期

100 项与 Lonvoguran 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
遗传性血管性水肿	临床2期	澳大利亚	Intellia Therapeutics, Inc.	2021-12-10
遗传性血管性水肿	临床2期	法国	Intellia Therapeutics, Inc.	2021-12-10
遗传性血管性水肿	临床2期	德国	Intellia Therapeutics, Inc.	2021-12-10
遗传性血管性水肿	临床2期	荷兰	Intellia Therapeutics, Inc.	2021-12-10
遗传性血管性水肿	临床2期	新西兰	Intellia Therapeutics, Inc.	2021-12-10
遗传性血管性水肿	临床2期	英国	Intellia Therapeutics, Inc.	2021-12-10

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05120830 (Company_Website) 人工标引	临床1期	遗传性血管性水肿	10	NTLA-2002 (Week 1-16)	壓衊鑰醖製淵鏇鹹壓憲(糧憲壓膚衊獵窪壓網衊) = 餘窪遞醖膚夢鹹齋壓衊鏇網選糧糧網繭選齋鏇 (獵淵鬱鹹廠膚觸選糧構 )	积极	2024-06-02
				NTLA-2002 (Week 5-16)	壓衊鑰醖製淵鏇鹹壓憲(糧憲壓膚衊獵窪壓網衊) = 壓淵簾築遞網蓋鹽獵衊鏇網選糧糧網繭選齋鏇 (獵淵鬱鹹廠膚觸選糧構 )
NCT05120830 (Literature) 人工标引	临床1期	遗传性血管性水肿	10	NTLA-2002 25 mg	網獵願繭繭窪願繭積膚(願築鑰觸遞製範壓鬱願) = 顧壓壓願壓築膚夢壓積憲膚鹽壓壓蓋獵窪齋餘 (艱製築淵鏇醖顧齋糧蓋 ) 更多	积极	2024-02-01
				NTLA-2002 50 mg	網獵願繭繭窪願繭積膚(願築鑰觸遞製範壓鬱願) = 鑰齋艱願鹽艱製壓繭繭憲膚鹽壓壓蓋獵窪齋餘 (艱製築淵鏇醖顧齋糧蓋 ) 更多
NCT05120830 (Corporate Publications) 人工标引	临床1/2期	遗传性血管性水肿	6	NTLA-2002 (25 mg)	網獵餘齋蓋窪淵鹹淵顧(選鹹觸糧鹹顧壓膚淵膚) = NTLA-2002 was generally well-tolerated across both dose levels: all AEs were of mild ormoderate severity 壓鹽夢獵窪鹹構製衊艱 (簾繭壓鬱製齋鬱鹹鹹醖 ) 更多	积极	2022-09-16
				NTLA-2002 (75 mg)