更新于：2024-06-29
MDNA-11
更新于：2024-06-29
概要
基本信息
最高研发阶段临床申请批准 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评- |
关联
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项与 MDNA-11 相关的临床试验A Phase 1/2 Open Label, Dose Escalation and Expansion Study of MDNA11, IL-2 Superkine, Administered Alone or in Combination With Immune Checkpoint Inhibitor in Patients With Advanced Solid Tumors
This is a Phase 1/2, multi-center, open-label, dose-escalation and expansion study to evaluate safety and tolerability, PK, pharmacodynamic, and early signal of anti-tumor activity of MDNA11 alone or in combination with a checkpoint inhibitor in patients with advanced solid tumors.
开始日期2021-08-27 |
申办/合作机构 |
100 项与 MDNA-11 相关的临床结果
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100 项与 MDNA-11 相关的转化医学
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100 项与 MDNA-11 相关的专利（医药）
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1
项与 MDNA-11 相关的文献（医药）2022-01-01·Journal for immunotherapy of cancer2区 · 医学
Fine-tuned long-acting interleukin-2 superkine potentiates durable immune responses in mice and non-human primate
2区 · 医学
ArticleOA
作者: Lloyd, Peter ; Smith, Paul ; Merchant, Rosemina ; Munegowda, Manjunatha Ankathatti ; To, Minh D ; Pearce, L Bruce ; Merchant, Fahar ; Galligan, Carole
Background:
Recombinant human interleukin-2 (rhIL-2, aldesleukin) is Food and Drug Administration approved for the treatment of metastatic melanoma and renal cell carcinoma and has achieved durable response in a subset of patients. However, its utility as an immunotherapeutic drug is limited by undesirable activation of immune suppressive regulatory T cells (Tregs) and a short half-life requiring frequent high dose administration, leading to unacceptable toxicities. We have engineered MDNA11, a long-acting IL-2 superkine, to overcome these limitations by (1) modifying receptor selectivity in favor of anti-cancer immune cells to increase therapeutic efficacy and (2) fusion to human albumin to extend the pharmacokinetic (PK) profile, circumventing the need for frequent dosing.
Methods:
MDNA11 was evaluated using in vitro and in vivo studies including: binding analyses to measure receptor affinity, IL-2 pathway signaling, PK studies in mice, and efficacy studies in syngeneic tumor models as single agent and in combination with immune checkpoint inhibitors. Finally, the safety and pharmacodynamic profile of MDNA11 was assessed in non-human primate (NHP).
Results:
Binding studies with MDNA11 demonstrated increased affinity for IL-2Rβ (CD122) and no binding to IL-2Rα (CD25). As a result, MDNA11 exhibits reduced/limited Treg stimulation while triggering an enhanced activation of natural killer and naïve CD8 T cells compared with rhIL-2. When administered to animals with pre-established tumors, MDNA11 controlled tumor growth in a monotherapy setting and in combination with anti-PD1 or anti-CTLA4 to induce durable tumor clearance with a once weekly dosing regimen. In a NHP model, MDNA11 was well tolerated while triggering durable and potent immune responses including expansion of lymphocytes without significant effect on Tregs and eosinophils, the latter been linked to an increased risk of vascular leak syndrome.
Conclusion:
MDNA11 is a next generation long-acting IL-2 immunotherapeutic with a highly favorable pharmacodynamic profile that translates to a strong therapeutic efficacy in preclinical tumor models and a strong and durable immune response in NHP.
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项与 MDNA-11 相关的新闻（医药）2024-06-27
Increased cash balance to $37 million following a $20 million investment by RA Capital extending runway into mid-2026
MDNA11 continues to exhibit compelling deep and durable single agent activity and best-in-class potential relative to other IL-2 therapies in clinical development
Results presented at the Annual Meeting of the AACR showed single agent MDNA11 response rate of 29% (4/14) in patients with checkpoint resistant tumors
Combination dose escalation with pembrolizumab (KEYTRUDA) continues to enroll patients at the MDNA11 monotherapy expansion dose (90 µg/kg, Q2W) having cleared safety at 60 µg/kg, Q2W
Approval of Clinical Trial Application by the European Medicines Agency expands the ABILITY-1 study of MDNA11 to Cancer Centers in the EU
Updated MDNA11 monotherapy expansion and combination escalation results to be presented at medical conferences in H2 of 2024
TORONTO and HOUSTON, June 27, 2024 (GLOBE NEWSWIRE) -- Medicenna Corp. (“Medicenna” or the “Company”) (TSX: MDNA, OTCQB: MDNAF), a clinical-stage immunotherapy company focused on the development of Superkines, today reported financial results and corporate highlights for the fiscal year ended March 31, 2024, as well as anticipated corporate milestones.
“Over the past year, we have continued to demonstrate best-in-class potential of MDNA11, having shown durable single-agent efficacy in end-stage cancer patients who have failed immunotherapies while maintaining an acceptable safety profile,” said Fahar Merchant, Ph.D., President and CEO of Medicenna. “We are encouraged by the 29% response rate observed in the study to date, including durable complete regression of target and non-target lesions in a pancreatic cancer patient who remains in remission and a melanoma patient that continues to show durable complete response of the target lesions. On the back of these data, we are delighted with the recent financial backing by RA Capital Management, which has strengthened our balance sheet by $20 million, extending our cash runway into mid-2026. We are also pleased to see acceptable safety pro MDNA11 in combination with pembrolizumab, which continues to enroll patients at the higher dose used in the MDNA11 monotherapy expansion arm as well as the recent approval by EMA of our application to expand the ABILITY-1 study in Europe. We look forward to sharing new clinical data from the monotherapy dose expansion as well as the combination arms of the study at medical conferences during the second half of 2024.”
Program highlights for the fiscal year ended March 31, 2024, along with recent developments, include:
MDNA11: IL-2 Superkine Program
Clinical activity highlights include:
Deep and Durable Anti-tumor Activity with Single-Agent MDNA11
29% response rate (N=4/14) and 50% clinical benefit rate (4 patients with partial responses, 3 patients with stable disease > 24 weeks) in high-dose phase 2 eligible patients who failed checkpoint inhibitor therapy
A pancreatic cancer patient with 100% regression of target and non-target lesions for over 104 weeks and continues to show remission 4 months after stopping treatment
A melanoma patient who is continuing on MDNA11 treatment shows 100% regression of target lesions with continued regression of non-target lesions
2 of 2 MSI-High patients and 2 of 4 evaluable dose expansion patients have had a partial response
Previously reported patients with partial responses and stable disease continue on the study further supporting the durability of MDNA11
The monotherapy expansion arm is enrolling patients with metastatic melanoma, non-melanoma skin cancers and MSI-H/dMMR tumors. Combination escalation arm of the ABILITY-1 study is enrolling patients with advanced solid tumors who progressed following earlier lines of treatment.
Monotherapy: Acceptable Safety Pro >
Complete Phase 1 monotherapy dose escalation data was presented and affirmed that MDNA11 has an acceptable safety profile.
No dose limiting toxicity (DLT) was reported with no evidence of vascular leak syndrome (VLS). Vast majority (95 %) of treatment-related adverse events (TRAEs) were of grade 1-2 and resolved within 48 hours; grade 3 TRAEs mainly constituted asymptomatic transient LFT elevations; no grade 4 or 5 events were reported.
Repeat administration of MDNA11 at the target doses showed further improvement in tolerability.
Monotherapy: Pharmacodynamic Analysis Showed Potent and Durable Systemic Immune Activation
Significant increases in stemness, central and effector memory and markers of enhanced effector function in circulating CD8+ T and NK cells, all of which are critical for achieving meaningful and durable anti-cancer response.
Analysis of gene expression signatures from pre-treatment and on-treatment paired biopsies show that cancer promoting pathways were degraded while immune-related pathways against cancer cells were enhanced following MDNA11 treatment.
Combination with KEYTRUDA®: Enrolling in Cohort 2 in the Absence of DLTs in the First Dose Cohort
In February 2024, the company announced dosing of the first patient in the Phase 1 combination escalation portion of the ABILITY-1 Study
Dose escalation in combination with KEYTRUDA® is now enrolling at the next higher dose of MDNA11 90 µg/kg Q2W and 400 mg pembrolizumab Q6W (priming MDNA11 30 & 60 μg/kg) following absence of any DLT at 60 µg/kg
The company expects to report additional data from the ongoing monotherapy expansion and combination arms of the ABILITY-1 study at medical conferences in the second half of calendar 2024.
Bizaxofusp (formerly MDNA55): Empowered IL-4 Superkine Program
The Company is currently pursuing partnership opportunities for its phase-3 ready IL-4 Superkine for recurrent glioblastoma (rGBM). Bizaxofusp, which holds both FastTrack and Orphan drug status from the FDA and FDA/EMA, respectively, is Medicenna’s Phase 3-ready asset for rGBM which has been tested in 118 patients with high grade gliomas (including 112 patients with rGBM).
On June 1st, 2024, the Company presented survival follow-up, and updated final Phase 2b study results for bizaxofusp at the 2024 ASCO Annual Meeting in Chicago.
Clinical activity highlights include:
In the Phase 2b study, a single treatment with bizaxofusp in unresectable rGBM patients achieved significant survival benefit (mOS of 13.5 vs. 7.2 months, p=0.009) and reduced risk of death by almost half (hazard ratio: 0.54, 95% confidence interval: 0.34-0.83) versus a propensity score (PS) balanced external control arm (ECA).
Bizaxofusp significantly increased median overall survival (mOS) by 88% (p = 0.009) and improved overall survival at 1 and 2 years by 180% and 290%, respectively.
Tumor control was associated with a significant increase in mOS following treatment with bizaxofusp and consequently, may be an early surrogate of survival benefit in future studies.
With the compelling survival benefit with bizaxofusp in rGBM, the most aggressive form of brain cancer which lacks a standard of care, Medicenna is seeking Breakthrough Therapy Designation with the FDA.
The proposed Phase 3 trial design incorporating a hybrid external control arm has been accepted by the FDA. Medicenna is currently working toward securing alignment with the EMA thereby enabling data from a single Phase 3 registrational trial being sufficient to approval in the EU and USA.
Pre-clinical Pipeline Programs
On April 9, 2024, at the 2024 AACR Annual Meeting, the Company presented preclinical data on its first-in-class IL-13R2 targeted candidate, MDNA113, from its T-MASK™ platform, which specifically delivers a masked bispecific anti-PD1-IL2 Superkine to IL-13R2 expressing tumors (affecting over 2 million cancer patients annually) where it is activated by cancer specific enzymes.
These data demonstrated that the T-MASK™ platform exemplified by MDNA113, facilitates tumor targeting and minimizes systemic toxicity while maximizing therapeutic activity at the tumor site.
Operational Highlights
On April 30, 2024, the Company closed a $20 million financing through a private placement with RA Capital Management (“RA”), a multi-stage investment manager based in Boston, MA, by way of a non-brokered private placement (the “Offering”). Pursuant to the terms of the Offering, RA subscribed for 5,141,388 common shares in the capital of the Company (the “Shares”) at a price of CA$1.95 per share and, in lieu of common shares, pre-funded warrants to purchase 5,141,388 common shares at a purchase price of CA$1.94 per pre-funded warrant, for total net proceeds to the Company of approximately CA$20 million.
Expected Upcoming Milestones
Topline MDNA11 monotherapy expansion data to be presented at medical conferences in H2 of calendar 2024.
Clinical update and combination escalation data from the ABILITY-1 Study evaluating MDNA11 with KEYTRUDA expected in H2 of calendar 2024.
Potential for Breakthrough Therapy Designation (BTD) for bizaxofusp. With compelling longer term survival benefit with bizaxofusp in rGBM patients, as presented at the ASCO meeting held in April 2024, Medicenna will seek to apply for BTD with the FDA.
Seek alignment with the European Medicines Agency (“EMA”) for the Phase 3 registration trial of bizaxofusp.
Annual Financial Results
As of March 31, 2024, cash and cash equivalents were $17.0 million, compared to $33.6 million on March 31, 2023. These funds, in combination with the $20 million in proceeds from the Offering, are expected to provide the Company with sufficient capital to execute its current planned expenditures to mid-2026 based on its current plans and projections.
For the year ended March 31, 2024, the Company reported total operating costs of $18.7 million compared to total operating costs of $16.3 million for the year ended March 31, 2023. The increase is related to an increase in general and administrative expenses ($0.7 million) and research and development expenses ($1.5 million) as discussed further below.
Net loss for the year ended March 31, 2024, was $25.5 million or $0.37 per share compared to a loss of $10.0 million or $0.16 per share for the year ended March 31, 2023. The increase in net loss for the year ended March 31, 2024 was primarily a result of an increase in the fair value of the derivative warrant liability of $8.0 million compared to a decrease of $4.3 million in the prior year. The significant increase in fair value of the warrant derivative is due to the 111% increase in the Company’s share price year-over-year, as share price is a key variable in the valuation of the derivative liability.
Research and development expenses of $10.8 million were incurred during the year ended March 31, 2024, compared with $9.3 million incurred in the year ended March 31, 2023. The increase in research and development expenses in the current fiscal year is primarily attributed to increased clinical costs related to the expansion of the MDNA11 ABILITY-1 with new clinical sites added in South Korea, the commencement of the combination arm with Keytruda, and an increase in salaries and benefits due to the addition of research staff during the period.
General and administrative expenses of $7.8 million were incurred during the year ended March 31, 2024, compared with $7.0 million during the year ended March 31, 2023. The increase in G&A expenses in the current year primarily relates to increased salaries and benefits in fiscal 2024 related to the addition and subsequent restructuring of executive staff in the US as the Company refocused its operations to Canada following its delisting from Nasdaq. This transition is anticipated to result in cost savings in future periods.
Medicenna’s financial statements for the year ended March 31, 2024 and the related management’s discussion and analysis (MD&A) will be available on SEDAR at .
About Medicenna
Medicenna is a clinical-stage immunotherapy company focused on developing novel, highly selective versions of IL-2, IL-4 and IL-13 Superkines and first-in-class Empowered Superkines. Medicenna’s long-acting IL-2 Superkine, MDNA11, is a next-generation IL-2 with superior affinity toward CD122 (IL-2 receptor beta) and no CD25 (IL-2 receptor alpha) binding, thereby preferentially stimulating cancer-killing effector T cells and NK cells. Medicenna’s IL-4 Empowered Superkine, bizaxofusp (formerly MDNA55), has been studied in 5 clinical trials enrolling over 130 patients, including a Phase 2b trial for recurrent GBM, the most common and uniformly fatal form of brain cancer. Bizaxofusp has obtained FastTrack and Orphan Drug status from the FDA and FDA/EMA, respectively. Medicenna’s early-stage BiSKITs™ (Bifunctional SuperKine ImmunoTherapies) and the T-MASK™ (Targeted Metalloprotease Activated SuperKine) programs are designed to enhance the ability of Superkines to treat immunologically “cold” tumors.
For more information, please visit , and follow us on Twitter and LinkedIn.
KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
Forward-Looking Statements
This news release contains forward-looking statements within the meaning of applicable securities laws. Forward-looking statements include, but are not limited to, express or implied statements regarding the future operations of the Company, estimates, plans, strategic ambitions, partnership activities and opportunities, objectives, expectations, opinions, forecasts, projections, guidance, outlook or other statements that are not historical facts, such as statements on the Company’s cash runway, the clinical performance and potential, safety pro MDNA11 and bizaxofusp (MDNA55), the reporting of additional results, anticipated corporate milestones, partnership efforts and cost savings following the Nasdaq delisting. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented. Forward-looking statements are often identified by terms such as “will”, “may”, “should”, “anticipate”, “expect”, “believe”, “seek”, “potentially” and similar expressions. Forward-looking statements are based on a number of assumptions believed by the Company to be reasonable at the date of this news release. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, there can be no assurance that such statements will prove to be accurate. These statements are subject to certain risks and uncertainties and may be based on assumptions that could cause actual results and future events to differ materially from those anticipated or implied in such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include the risks detailed in the latest annual information form of the Company and in other filings made by the Company with the applicable securities regulators from time to time in Canada.
The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management, may prove to be incorrect and actual results may differ materially from those anticipated or implied in forward-looking statements. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date hereof and except as required by law, we do not intend and do not assume any obligation to update or revise publicly any of the included forward-looking statements.
This news release contains hyperlinks to information that is not deemed to be incorporated by reference in this news release.
Investor and Media Contact:
Christina Cameron
Investor Relations, Medicenna Therapeutics
ir@medicenna.com
(647) 953-0673
临床结果临床2期突破性疗法临床1期临床3期
2024-06-26
- ABILITY-1 study is currently enrolling patients for the treatment of advanced solid tumors with MDNA11, a novel long-acting IL-2 super-agonist, as a monotherapy or in combination with KEYTRUDA®, at clinical trial sites in U.S.A., Canada, Australia, and Korea
- Monotherapy expansion and combination escalation data of the ABILITY-1 study are expected in H2 2024
TORONTO and HOUSTON, June 26, 2024 (GLOBE NEWSWIRE) -- Medicenna Therapeutics Corp. (“Medicenna” or the “Company”) (TSX: MDNA, OTCQB: MDNAF), a clinical-stage immunotherapy company focused on the development of Superkines, today announced that the European Medicines Agency (“EMA”) has approved the Clinical Trial Application (“CTA”) for the conduct of the Phase 1/2 ABILITY-1 (A Beta-only IL-2 ImmunoTherapY) Study with MDNA11 either alone or in combination with pembrolizumab (KEYTRUDA®) thereby expanding the clinical trial in the European Union (“EU”). MDNA11 is the Company’s long-acting, “beta-enhanced not-alpha” IL-2 super-agonist and is currently enrolling patients with advanced solid tumors in the ABILITY-1 trial at clinical trial sites in U.S.A., Canada, Australia, and Korea.
“We are excited to build on the early success and promising efficacy and safety of our ongoing ABILITY-1 study that is demonstrating MDNA11’s best-in-class potential,” said Fahar Merchant, PhD, President & CEO of Medicenna. “Expanding the clinical trial to various centers in the EU is an important milestone and adds to the positive momentum behind our MDNA11 program. We anticipate that the expansion to Europe will expedite enrolment in the trial and advance the study towards key updates in the monotherapy expansion and combination escalation portions of the ABILITY-1 study which will be presented at medical conferences during H2 2024.”
The ABILITY-1 study is designed to assess the safety, pharmacokinetics, pharmacodynamics, and anti-tumor activity of various doses of intravenously administered MDNA11 in patients with advanced, relapsed, or refractory solid tumors and includes an MDNA11 monotherapy arm, as well as a combination arm designed to evaluate MDNA11 in combination with pembrolizumab (KEYTRUDA®).
About MDNA11
MDNA11 is a long-acting ‘beta-enhanced not-alpha’ interleukin-2 (IL-2) Superkine specifically engineered to overcome the shortcomings of aldesleukin and other next generation IL-2 variants by preferentially activating immune effector cells (CD4+ T, CD8+ T and NK cells) responsible for killing cancer cells, with minimal or no stimulation of immunosuppressive Tregs. These unique proprietary features of the IL-2 Superkine have been achieved by incorporating seven specific mutations and genetically fusing it to a recombinant human albumin scaffold to improve the pharmacokinetic (PK) pro pharmacological activity of MDNA11 due to albumin’s natural propensity to accumulate in highly vascularized sites, in particular tumor and tumor draining lymph nodes. MDNA11 is currently being evaluated in the Phase 1/2 ABILITY-1 study as both a monotherapy and in combination with pembrolizumab (KEYTRUDA®).
About the ABILITY-1 Study
The ABILITY-1 study (NCT05086692) is a global, multi-center, open-label study that assesses the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-tumor activity of MDNA11 as monotherapy or in combination with pembrolizumab (KEYTRUDA®). In the combination dose escalation of the Phase 2 study, approximately 12 patients are expected to be enrolled and administered ascending doses of MDNA11 intravenously once every two weeks in combination with pembrolizumab. This portion of the study includes patients with a wide range of solid tumors with the potential for susceptibility to immune modulating therapeutics. Upon identification of an appropriate dose regimen for combination, the study will proceed to a combination dose expansion cohort.
About Medicenna
Medicenna is a clinical-stage immunotherapy company focused on developing novel, highly selective versions of IL-2, IL-4 and IL-13 Superkines and first-in-class Empowered Superkines. Medicenna’s long-acting IL-2 Superkine, MDNA11, is a next-generation IL-2 with superior affinity toward CD122 (IL-2 receptor beta) and no CD25 (IL-2 receptor alpha) binding, thereby preferentially stimulating cancer-killing effector T cells and NK cells. Medicenna’s IL-4 Empowered Superkine, bizaxofusp (formerly MDNA55), has been studied in 5 clinical trials enrolling over 130 patients, including a Phase 2b trial for recurrent GBM, the most common and uniformly fatal form of brain cancer. Bizaxofusp has obtained FastTrack and Orphan Drug status from the FDA and FDA/EMA, respectively. Medicenna’s early-stage BiSKITs™ (Bifunctional SuperKine ImmunoTherapies) and the T-MASK™ (Targeted Metalloprotease Activated SuperKine) programs are designed to enhance the ability of Superkines to treat immunologically “cold” tumors.
For more information, please visit , and follow us on Twitter and LinkedIn.
KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
Forward-Looking Statements
This news release contains forward-looking statements within the meaning of applicable securities laws. Forward-looking statements include, but are not limited to, express or implied statements regarding the future operations of the Company, estimates, plans, strategic ambitions, partnership activities and opportunities, objectives, expectations, opinions, forecasts, projections, guidance, outlook or other statements that are not historical facts, such as statements on the Company’s clinical potential, of MDNA11 and the ABILITY-1 study and its, safety, enrollment and the reporting of data therefrom. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented. Forward-looking statements are often identified by terms such as “will”, “may”, “should”, “anticipate”, “expect”, “believe”, “seek”, “potentially” and similar expressions. Forward-looking statements are based on a number of assumptions believed by the Company to be reasonable at the date of this news release. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, there can be no assurance that such statements will prove to be accurate. These statements are subject to certain risks and uncertainties and may be based on assumptions that could cause actual results and future events to differ materially from those anticipated or implied in such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include the risks detailed in the latest Annual Report on Form 20-F of the Company and in other filings made by the Company with the applicable securities regulators from time to time in Canada.
The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management, may prove to be incorrect and actual results may differ materially from those anticipated or implied in forward-looking statements. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date hereof and except as required by law, we do not intend and do not assume any obligation to update or revise publicly any of the included forward-looking statements.
This news release contains hyperlinks to information that is not deemed to be incorporated by reference in this news release.
Investor and Media Contact:
Christina Cameron
Investor Relations, Medicenna Therapeutics
ir@medicenna.com
(647) 953-0673
临床2期免疫疗法孤儿药临床研究临床申请
2024-06-09
·药明康德
▎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。
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参考资料（可上下滑动查看）
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[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
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临床结果基因疗法免疫疗法临床2期
100 项与 MDNA-11 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 |
|---|---|---|---|---|
| 基底细胞癌 | 临床2期 | 美国 | 2021-08-27 | |
| 基底细胞癌 | 临床2期 | 澳大利亚 | 2021-08-27 | |
| 基底细胞癌 | 临床2期 | 加拿大 | 2021-08-27 | |
| 膀胱癌 | 临床2期 | 美国 | 2021-08-27 | |
| 膀胱癌 | 临床2期 | 澳大利亚 | 2021-08-27 | |
| 膀胱癌 | 临床2期 | 加拿大 | 2021-08-27 | |
| 结直肠癌 | 临床2期 | 美国 | 2021-08-27 | |
| 结直肠癌 | 临床2期 | 澳大利亚 | 2021-08-27 | |
| 结直肠癌 | 临床2期 | 加拿大 | 2021-08-27 | |
| 皮肤鳞状细胞癌 | 临床2期 | 美国 | 2021-08-27 |
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临床结果
临床结果
适应症
分期
评价
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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 |
|---|
临床1/2期 | 14 | 鏇糧獵醖齋繭淵窪窪齋(齋範獵構選窪淵襯製遞) = 廠積製獵繭壓窪網觸夢 衊簾齋顧膚襯鏇醖獵憲 (鬱膚蓋願壓鹹鏇廠夢願 ) 更多 | 积极 | 2024-05-13 | |||
临床1/2期 | 30 | 廠鑰網網鑰鑰範鏇艱齋(鹹簾範夢艱觸壓鹽鏇鹽) = none 鏇觸構積範壓襯膚獵齋 (築鹹顧壓簾獵壓衊壓鏇 ) 更多 | - | 2024-04-05 | |||
临床1/2期 | 20 | 鬱構製繭憲構鑰窪壓遞(憲憲憲願範簾襯製網窪) = 廠艱顧衊積顧衊鬱構壓 鏇範糧蓋夢築壓糧窪窪 (蓋蓋鬱鏇獵糧蓋顧窪夢 ) 更多 | 积极 | 2023-10-31 | |||
临床1/2期 | 20 | 製鹹選蓋艱願餘範膚襯(觸構鑰憲膚築廠選窪淵) = MDNA11 was generally well tolerated across cohorts, with the majority of adverse events (AEs) being grade 1 or 2, with no grade 4 or 5 AEs. 積構窪壓餘製襯鹹網範 (積獵簾觸積憲廠醖壓廠 ) | 积极 | 2023-08-09 | |||
临床1/2期 | 20 | 齋壓夢鬱範廠積鑰願齋(淵簾製鑰簾夢憲獵餘顧) = MDNA11 was generally well tolerated across cohorts, with the majority of adverse events (AEs) being grade 1 or 2, with no grade 4 or 5 AEs. 廠夢糧範獵築鹽蓋繭繭 (蓋製築夢齋選艱淵鹽顧 ) | 积极 | 2023-08-09 | |||
临床1/2期 | 14 | 齋願構蓋顧鹹壓窪鑰製(艱壓艱鏇淵鏇築蓋網鏇) = The most common drug-related AEs were infusion related reactions (71%), pyrexia (35%), diarrhea (28%) and nausea (28%), and with a majority of these being Grade 1 or 2 and lasted less than 24 hours. 網蓋廠遞遞簾網壓鑰願 (獵製襯壓糧艱鹽鑰鹽窪 ) | 积极 | 2022-11-01 | |||
临床1/2期 | - | 鬱顧壓衊選憲淵鑰顧襯(夢蓋範蓋繭鹽壓糧艱糧) = dose dependent activation of various biomarkers on effector cells without concomitant stimulation of immune suppression 鏇鬱繭鑰繭襯製選鹹艱 (製積糧選築遞遞構築網 ) | 积极 | 2022-11-01 | |||
临床1/2期 | 14 | 鏇繭淵憲願壓艱憲構夢(蓋鏇觸鹽繭鑰構艱築膚) = 糧廠糧憲簾鹹獵製積選 遞艱蓋製選糧膚壓製糧 (糧構齋願繭廠願積鏇簾 ) 更多 | 积极 | 2022-09-28 | |||
临床1/2期 | 肿瘤 末线 | 14 | (dose escalation) | 夢築夢襯獵夢鏇鏇繭廠(鏇鏇製醖網糧遞蓋鏇窪) = 蓋廠鹹鏇淵範鹹鹽蓋鏇 壓鏇獵鬱齋糧簾繭築艱 (顧鏇餘醖壓範蓋鑰範積 ) | 积极 | 2022-07-27 |
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