On September 12, 2024 Immunocore Holdings plc (Nasdaq: IMCR) ("Immunocore" or the "Company"), a commercial-stage biotechnology company pioneering and delivering transformative immunomodulating medicines to radically improve outcomes for patients with cancer, infectious diseases and autoimmune diseases, reported that management will participate in the 2024 Cantor Global Healthcare Conference (Press release, Immunocore, SEP 12, 2024, View Source [SID1234646532]).

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2024 Cantor Global Healthcare Conference
Fireside Chat: Thursday, September 19, 2024, at 12:45 p.m. EDT

The presentation will be webcast live and can be accessed by visiting ‘Events & Presentations’, under ‘Events’, via the ‘Investors’ section of Immunocore’s website at www.immunocore.com. Following the event, a replay of the presentation will be made available for a limited time.

On September 12, 2024 OmRx Oncology, or "OmRx," a new biopharmaceutical venture with investment from Scripps Research, launched with a Phase 2 clinical asset in-licensed from Gilead Sciences, Inc. and a vision for expanding access to immunotherapy in countries where biologic checkpoint inhibitors are often unaffordable and unavailable to patients (Press release, OmRx Oncology, SEP 12, 2024, View Source [SID1234646549]).

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The clinical-stage asset, OX-4224, discovered and initially developed by Gilead Sciences, is an investigational oral immune checkpoint inhibitor that targets the PD-1/PD-L1 pathway and will be evaluated by OmRx for the treatment of patients with non-small cell lung cancer (NSCLC). OmRx in-licensed the Phase 2-ready asset and plans to develop it for patients in countries where an oral small molecule would present a more affordable, accessible, and potentially equally effective option to traditional antibody checkpoint inhibitors such as Keytruda and Opdivo.

OmRx is wholly focused on advancing this clinical-stage program, first as a monotherapy in low and middle-income countries, and eventually, globally for potential all-oral combination therapies. OmRx’s mission to serve patients in low and middle-income countries is shared by Scripps Research, which actively supports a significant number of R&D initiatives addressing global health needs. The company is led by a high-caliber team with expertise in cancer R&D, clinical development, entrepreneurship, and corporate development. The OmRx team includes Dr. William Lee, Ph.D., who serves as Chairman and brings 30 years of experience from his tenure as the former Executive Vice President of Research at Gilead Sciences.

"Immune checkpoint inhibitor monoclonal antibodies represent one of the most important treatment advances in cancer in the past two decades, but they are essentially unavailable to cancer patients outside of the major commercial markets. Oral checkpoint inhibitors, meanwhile, could have important advantages over antibodies – they would not require infusions or infusion centers, dosing and patient exposure could be rapidly modulated in the case of immune-related side effects, and they would be significantly cheaper to manufacture," said Isy Goldwasser, CEO, OmRx. "With the launch of OmRx we can advance a promising oral checkpoint inhibitor that could bring immunotherapy to more people in an affordable way with the goal of reducing the worldwide cancer burden."

OX-4224 will initially be studied as a second-line monotherapy in patients with metastatic NSCLC whose tumors express PD-L1. The Phase 2 randomized, open-label study in India will establish safety and efficacy of OX-4224 in subjects with NSCLC who have not received immune checkpoint inhibitors.

"One of the highlights of my career was overseeing the discovery of a small molecule that blocked the PD-1/PD-L1 immune checkpoint and by doing so, could impact cancer therapy globally," said Dr. William Lee, Chairman, OmRx. "I am extremely grateful to the Scripps team for sharing this vision and investing in OmRx. If OX-4224 demonstrates the safety and efficacy projected, it will make a difference for patients everywhere, and especially in resource-constrained countries like India."

OX-4224 is an investigational product and statements regarding the potential benefits and future development of this investigational product are forward-looking and subject to risks and uncertainties.

On September 12, 2024 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported its consolidated financial results for the six months ended June 30, 2024 (Press release, Innate Pharma, SEP 12, 2024, View Source [SID1234646533]). The consolidated financial statements are attached to this press release.

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"We are focused on our growth strategy as we advance our pipeline," said Hervé Brailly, Chief Executive Officer ad interim of Innate Pharma. "We recently presented Phase 2 results with lacutamab in mycosis fungoides at ASCO (Free ASCO Whitepaper) and are engaged in discussions with the FDA on next steps in its development. We are also progressing towards Phase 1 for our first and differentiated ADC program IPH45, targeting Nectin-4."

Webcast and conference call will be held today at 2:00 p.m. CEST (8:00 a.m. ET)

Access to live webcast: View Source

Participants may also join via telephone by registering in advance of the event at
View Source

This information can also be found on the Investors section of the Innate Pharma website, www.innate-pharma.com.
A replay of the webcast will be available on the Company website for 90 days following the event.

Pipeline highlights:

Lacutamab (anti-KIR3DL2 antibody):

Cutaneous T Cell Lymphoma

TELLOMAK is a global, open-label, multi-cohort Phase 2 clinical trial evaluating lacutamab in patients with Sézary syndrome and mycosis fungoides.

•Favorable results from the Phase 2 TELLOMAK study with lacutamab in mycosis fungoides were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2024 Annual Meeting in June 2024. The data demonstrate that treatment with lacutamab resulted in meaningful antitumor activity, regardless of the KIR3DL2 baseline expression,
1 Developed by Sanofi
2 Including short term investments (€21.8 million) and non-current financial instruments (€10.3 million)

and an overall favorable safety profile. The global objective response rate was 16.8% (Olsen 2011) and 22.4% (Olsen 2022), including 2 complete responses and 16 partial responses.

Peripheral T Cell lymphoma (PTCL)

The Phase 2 KILT (anti-KIR in T Cell Lymphoma) trial, an investigator-sponsored, randomized controlled trial led by the Lymphoma Study Association (LYSA) to evaluate lacutamab in combination with chemotherapy GEMOX (gemcitabine in combination with oxaliplatin) versus GEMOX alone in patients with KIR3DL2-expressing relapsed/refractory PTCL is ongoing and continues to recruit patients.

ANKET (Antibody-based NK cell Engager Therapeutics):

ANKET is Innate’s proprietary platform for developing next-generation, multi-specific NK cell engagers to treat certain types of cancer. Innate’s pipeline includes five public drug candidates born from the ANKET platform: SAR443579/IPH6101 (SAR’579; trifunctional anti-CD123 NKp46-CD16 NKCE), SAR445514/IPH6401 (SAR’514 trifunctional anti-BCMA NKp46-CD16 NKCE), IPH62 (anti-B7-H3), IPH67 (target undisclosed, solid tumors) and tetra-specific IPH6501 (anti-CD20 with IL-2v). Several other undisclosed proprietary preclinical targets are being explored.

IPH6501 (proprietary)

IPH6501 is Innate’s proprietary CD20-targeted IL-2v bearing second-generation ANKET. In March 2024 the first patient was dosed in the Phase 1/2 clinical trial evaluating IPH6501 in B cell Non-Hodgkin’s lymphoma (B-NHL). The study is planned to enroll up to 184 patients.

•Innate presented preclinical data of IPH6501 at the ASCO (Free ASCO Whitepaper) Annual Meeting and European Hematology Association (EHA) (Free EHA Whitepaper) Annual congress in June 2024. Preclinical data showed that IPH6501 depletes autologous CD20+ B cells from healthy donors with greater efficacy and lower induction of pro-inflammatory cytokines than a CD20-T-cell engager. IPH6501 also effectively and preferentially stimulates NK cell proliferation from peripheral blood mononuclear cells of R/R NHL patients.

•The trial-in-progress of the Phase 1/2 study has been presented at the European Hematology Association (EHA) (Free EHA Whitepaper) and ASCO (Free ASCO Whitepaper) in 2024.

SAR’579, SAR’514, IPH62 and IPH67 (under development by Sanofi)

SAR’579 / IPH6101

The Phase 1/2 clinical trial by Sanofi is progressing well, evaluating SAR’579 / IPH6101, a trifunctional anti-CD123 NKp46-CD16 NK-cell engager and ANKET platform lead asset, in patients with relapsed or refractory acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) or high-risk myelodysplastic syndrome (HR-MDS).

•Updated efficacy and safety results from the dose-escalation part of the Phase 1/2 study with SAR’579 / IPH6101, were shared in an oral presentation at the EHA (Free EHA Whitepaper) 2024 Congress. The data demonstrated that SAR’579 continues to show clinical benefit and durable responses along with a favorable safety profile in patients with R/R AML, with 5 complete remissions (4 CR / 1 CRi) achieved at 1 mg/kg, with durable CR (>10 months) observed in 3 patients.

•In April 2024, Sanofi advanced SAR’579 / IPH6101, to the Phase 2 preliminary dose expansion of the trial. Under the terms of the 2016 research collaboration with Sanofi, the progression to the dose expansion part of the trial has triggered a milestone payment from Sanofi to Innate of €4m.

In July 2024, Sanofi initiated a new Phase 1 / Phase 2, randomized, open label, multi-cohort, multi-center study (NCT06508489) assessing the safety, tolerability and preliminary efficacy of SAR’579 / IPH6101 administered in combination with azacitidine and venetoclax in patients with CD123 expressing hematological malignancies in newly diagnosed AML.

SAR’514/IPH6401

The Sanofi led Phase 1/2 clinical trial with SAR’514 / IPH6401, a trifunctional anti-BCMA Nkp46-CD16 NK-cell engager, in patients with Relapsed/Refractory Multiple Myeloma and Relapsed/Refractory Light-chain Amyloidosis is ongoing.

IPH62, IPH67 and option

•IPH62 is a NK-cell engager program targeting B7-H3 from Innate’s ANKET platform under development. Following a research collaboration period and upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization.

•IPH67 is a NK-cell engager program in solid tumors from Innate’s ANKET platform under development. Following a research collaboration period and upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization.

•Sanofi still retains the option of one additional ANKET target under the terms of the 2022 research collaboration and license agreement.

Antibody Drug Conjugates:

Innate develops different approaches for the treatment of cancer utilizing its antibody engineering capabilities to deliver novel assets, with its innovative ANKET platform and is also exploring Antibody Drug Conjugates (ADC) formats.

IPH45 (Nectin-4 ADC):

IPH45 is Innate’s proprietary and differentiated exatecan-antibody drug conjugate (ADC) targeting Nectin-4.

•First preclinical data were presented in an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024. In preclinical studies, IPH45 shows anti-tumor efficacy in vivo, in Nectin-4 expressing tumors including in enfortumab vedotin (EV) refractory models. Importantly, IPH45 shows stronger activity than EV, in multiple urothelial carcinoma patient-derived xenografted (PDX) mice models, across Nectin-4 high and Nectin-4 low expression levels. In addition, IPH45 has anti-tumor activity in combination with anti-PD1 treatment in PD-1 resistant model in vivo and has a favorable safety profile in relevant animal toxicology models.

•IPH45 continues towards a Phase 1 trial in 2024.

Monalizumab (anti-NKG2A antibody), partnered with AstraZeneca:

•The Phase 3 PACIFIC-9 trial run by AstraZeneca evaluating durvalumab (anti-PD-L1) in combination with monalizumab or AstraZeneca’s oleclumab (anti-CD73) in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who have not progressed following definitive platinum-based concurrent chemoradiation therapy (CRT) is ongoing.

oAfter the period, the Independent Data Monitoring Committee recommended the continuation of the Phase 3 PACIFIC-9 trial based on a pre-planned analysis.

•Updated results from COAST, a Phase 2 study of durvalumab with oleclumab or monalizumab in patients with Stage III unresectable non-small-cell lung cancer were presented at the ASCO (Free ASCO Whitepaper) 2024 Annual Meeting, in June 2024. In this analysis of updated results from COAST, the combination of durvalumab plus oleclumab or monalizumab increased objective response rate, prolonged progression free survival, and trended toward improved overall survival compared to durvalumab alone.

•AstraZeneca presented interim results from the randomized NeoCOAST-2 Phase 2 platform trial during the 2024 World Conference on Lung Cancer in September 2024. In this preliminary analysis on the first 60 of 72 patients randomized to Arm 2, monalizumab added to durvalumab plus platinum-based chemotherapy doublet induced a pathological complete response rate of 26.7% [95% CI; 16.1–39.7] and a major pathological response rate of 53.3% [95% CI; 40.0–66.3] which are numerically higher than the durvalumab plus platinum doublet approved regimen. Treatment in Arm 2 showed manageable safety profile and no impact on surgical rate. The NeoCOAST-2 platform study is intended to assess the safety and efficacy of neoadjuvant durvalumab alone or combined with novel

immuno-oncology agents and chemotherapy in resectable, early-stage NSCLC, followed by adjuvant treatment with durvalumab with or without the novel agents.

IPH5201 (anti-CD39), partnered with AstraZeneca:

The MATISSE Phase 2 clinical trial conducted by Innate in neoadjuvant lung cancer for IPH5201, an anti-CD39 blocking monoclonal antibody developed in collaboration with AstraZeneca, is ongoing and recruitment is on track. Following a pre-planned interim analysis after the period, the MATISSE Phase 2 trial continues according to plans.

IPH5301 (anti-CD73):

The investigator-sponsored CHANCES Phase 1 trial of IPH5301 with Institut Paoli-Calmettes is ongoing. Preliminary results will be presented at the upcoming (European Society of Medical Oncology) ESMO (Free ESMO Whitepaper) Annual Meeting 2024. The abstract, available on the ESMO (Free ESMO Whitepaper) website, states that IPH5301 was safe and well-tolerated with preliminary signals of monotherapy antitumor activity.

Corporate Update:

•In connection with Innate’s previous announcement that it had established an at-the-market ("ATM") program, on January 16, 2024 Innate filed a new Registration Statement on Form F-3 (Registration No. 333-276164). On February 6, 2024, Innate filed a prospectus supplement relating to its previously established ATM program, pursuant to which it may, from time to time, offer and sell to eligible investors a total gross amount of up to $75 million of American Depositary Shares ("ADS"). Each ADS represents one ordinary share of Innate. As of June 30, 2024, no sales have been made under the program.

•Takeda made a strategic decision to terminate the license agreement executed in March 2023 for use of selected Innate antibodies in antibody drug-conjugates. Innate will regain full rights to these antibodies in Q4 2024.
Financial highlights for the first half of 2024:

The key elements of Innate’s financial position and financial results as of and for the six-month period ended June 30, 2024 are as follows:

•Cash, cash equivalents, short-term investments and financial assets amounting to €102.1 million (€m) as of June 30, 2024 (€102.3m as of December 31, 2023).

•Revenue and other income amounted to €12.3m in the first half of 2024 (€40.2m in the first half of 2023) and mainly comprised of:

oRevenue from collaboration and licensing agreements, which mainly resulted from the partial or entire recognition of the proceeds received pursuant to the agreements with AstraZeneca, Sanofi and Takeda. They result from the partial or entire recognition of the proceeds received pursuant to such agreements. They are recognized when the entity’s performance obligation is met. They are recognized at a point in time or spread over time according to the percentage of completion of the work that the Company is committed to carry out under these agreements:

(i) Revenue from collaboration and licensing agreements for monalizumab decreased by €6.5m to €3.0m in the first half of 2024 (€9.5m in the first half of 2023). This change is mainly due to the recognition of increased revenue in the first six months of 2023. Indeed, as of June 30, 2023, the Company had analyzed the cost base used to calculate the percentage of completion of Phase 1/2 trials in connection with their progress. This analysis led to a reduction in the cost base through a re-estimation of projected expenses. As a result, this adjustment on the cost base had a positive impact on the percentage of completion and led to the recognition of additional revenue of €5.9 million for the first half of 2023 which was not replicated in 2024.

(ii) Revenue related to the license and collaboration agreement signed with Sanofi in 2016 increased by €2.0m, to €4.0m for the six months ended June 30, 2024, as compared to €2.0m for the six months ended June 30, 2023. On April 15, 2024, the Company announced the treatment of the

first patient in the Phase 2 dose expansion part of the Sanofi-sponsored clinical trial evaluating NK Cell Engager SAR443579/ IPH6101 in various blood cancers. Under the terms of the 2016 agreement, this trial progress triggered a milestone payment of €4.0 million fully recognized in revenue during the first quarter of 2024. This amount was received by the Company on May 17, 2024.As a reminder, the Company announced that, in June 2023, the first patient was dosed in a Sanofi-sponsored Phase 1/2 clinical trial evaluating IPH6401/SAR’514 in relapsed or refractory Multiple Myeloma. As provided by the licensing agreement signed in 2016, Sanofi made a milestone payment of €2.0 million, fully recognized in revenue as of June 30, 2023. This amount was received by the Company on July 21, 2023.

(iii) Revenue related to the research collaboration and licensing agreement signed with Sanofi in 2022 decreased by €18.3m, to €0.4m for the six months ended June 30, 2024, as compared to €18.7m for the six months ended June 30, 2023. As previously announced, on January 25, 2023, the Company announced the expiration of the waiting period under the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 and the effectiveness of the licensing agreement as of January 24, 2023. Consequently, the Company received an upfront payment of €25.0 million in March 2023, including €18.5 million for the exclusive license, €1.5 million for the research activities and €5.0 million for the option on two additional targets. The €18.5 million upfront payment relating to the exclusive license was fully recognized in revenue as of June 30, 2023. The €1.5 million upfront payment is recognized on a straight-line basis over the duration of the research activities that the Company has agreed to carry out. As a result, a €0.2 million has been recognized in revenue as of June 30, 2024 and June 30, 2023. Then, on December 19, 2023, the Company announced that Sanofi had exercised an option for one of the two targets. As a consequence, the Company recognized related income of €2.5 million as of December 31, 2023. This option exercise also resulted in a milestone payment of €15.0 million, including €13.3 million in respect of the exclusive license, which was fully recognized in income as of December 31, 2023, and €1.7 million in respect of research activities to be carried out by the Company, which will be recognized in income on a straight-line basis over the duration of the research activities that the Company has agreed to carry out. Sanofi and Innate will collaborate and work on the research activities defined in the contractual research program. These activities began during the first half of 2024. An amount of €0.2 million has been recognized in revenue as of June 30, 2024. Amounts not recognized in revenue are classified as deferred revenue.

(iv) Revenues under the license agreement signed with Takeda in 2023 are nil for the first half of 2024, compared to €4.6 million for the first half of 2023. On April 3, 2023, the Company announced that it has entered into an exclusive license agreement with Takeda under which Innate grants Takeda exclusive worldwide rights to research and develop antibody drug conjugates (ADC) using a panel of selected Innate antibodies against an undisclosed target, with a primary focus in Celiac disease. Takeda will be responsible for the future development, manufacture and commercialization of any potential products developed using the licensed antibodies. As such, the Company considers that the license granted is a right to use the intellectual property, which is granted fully and perpetually to Takeda. The agreement does not stipulate that Innate’s activities will significantly affect the intellectual property granted during the life of the agreement. Consequently, the $5.0 million (or €4.6 million) initial payment, received by the Company in May 2023, was fully recognized in revenue as of June 30, 2023.

oGovernment funding for research expenditures of €4.1m in the first half of 2024 (€4.9m in the first half of 2023).

•Operating expenses are €38.7m in the first half of 2024 (€40.6m in the first half of 2023), of which 75.2% (€29.1m) are related to R&D.

oR&D expenses decreased by €2.4m to €29.1m in the first half of 2024 (€31.5m in the first half of 2023). This change is mainly explained by lower personnel and other R&D expenses by €2.2millions (-15.4%). This decrease is due to an non-recurring amortization charge in the first half of 2023, related to the IPH5201 rights (full amortization of the additional €2.0 million invoice from Orega Biotech following the dosing of the first patient in the Phase 2 MATISSE clinical trial in June 2023). Additionally, direct R&D expenses, which slightly decreased by €0.2 million, remained at €17.1 million, with an acceleration in preclinical

spending related to the Antibody-Drug Conjugates (ADC) program offsetting the decrease in expenses related to certain more mature clinical-stage programs.

oGeneral and administrative (G&A) expenses increased by €0.4m to €9.6m in the first half of 2024 (€9.1m in the first half of 2023) mainly resulting from (i) a €0.4m decrease of personnel expenses mainly due to a reduction of administrative staff, offset by (ii) a €0.3m increase in non-scientific advisory and consulting fees resulting from greater reliance on recruitment agencies, and finally (iii) a €0.5 million increase in other expenses, mainly due to the derecognition of returned spaces in the first half of 2023 (as a reminder, on March 13, 2023, the Company signed an amendment to the lease of the ‘Le Virage’ building, aimed at reducing the area of leased premises) and the sale of related furniture during the same period, which led to an exceptional reduction in these charges in the first half of 2023.

•A net financial gain of €1.5m in the first half of 2024 (€2.1m in the first half of 2023). This variance mainly results from the unfavorable evolution of the dollar exchange rate and its impact on foreign exchange recorded during the first half of 2024. The negative currency impact was offset by an increase in the fair value of certain financial instruments.

•A net loss of €24.8m for the first half of 2024 (net income of €1.7m for the first half of 2023).

The table below summarizes the IFRS consolidated financial statements as of and for the six months ended June 30, 2024, including 2023 comparative information.

On September 12, 2024 REVEAL GENOMICS, S.L., a Barcelona-based biotechnology start-up seeking to revolutionize precision oncology through biomarker innovation, reported both the development and the clinical validation of TNBCDX, the first genomic test designed specifically for patients with early-stage TNBC (Press release, REVEAL GENOMICS, SEP 12, 2024, View Source [SID1234646550]). This dual milestone will be presented at the ESMO (Free ESMO Whitepaper) Congress 2024 in Barcelona (Spain).

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TNBCDX represents a significant advancement in the management of TNBC, a subtype of breast cancer that is particularly challenging to treat due to its lack of hormone receptors and HER2 expression. This disease is typically treated with neoadjuvant multi-agent chemotherapy regimens that include taxanes, anthracyclines, cyclophosphamide, carboplatin, and pembrolizumab. However, these therapies often pose significant toxicities for patients and can lead to a considerable financial burden and increased use of healthcare resources.

Meanwhile, less intensive systemic therapies involving taxanes and carboplatin without anthracyclines and pembrolizumab are showing outstanding outcomes in selected patients. In this context, a biomarker such as TNBCDX was needed to guide treatment decisions and safely identify patients who could benefit from de-escalation of therapy.

Across two independent validation studies with 418 patients, the TNBCDX scores showed a strong association with pathological complete response (pCR), distant disease-free survival (DDFS), and overall survival (OS), providing a more refined risk stratification. Notably, the TNBCDX test outperformed tumor-infiltrating lymphocytes (TILs) in predicting both pCR and survival outcomes.

The first validation study, ADAPT-TN, led by Dr. Nadia Harbeck and Dr. Oleg Gluz of the West German Study Group, was a phase II prospective neoadjuvant trial (WSG-ADAPT TN Trial, NCT01815242) that enrolled patients diagnosed with stage I–III TNBC. The study involved a treatment regimen of nab-paclitaxel combined with either gemcitabine or carboplatin. Patients who did not achieve a pCR were given additional anthracycline-based chemotherapy. The second validation study, MMJ-CAR-2014-01 (NCT01560663), led by Dr. Miguel Martin, is an ongoing prospective, multicenter, nonrandomized trial in Spain investigating the antitumor activity of neoadjuvant carboplatin and docetaxel in early-stage TNBC. This study includes patients with stage I–III TNBC, treated with six cycles of carboplatin and docetaxel.

Dr. Miguel Martin, Chair of Medical Oncology at the Hospital General Universitario Gregorio Marañón in Madrid, and a recognized leader in breast cancer, will present the findings during the ESMO (Free ESMO Whitepaper) session scheduled for 10:50 a.m. on September 14th, 2024, at Barcelona Auditorium.

"The development and validation of TNBCDX are significant achievements in the field of precision oncology for TNBC. This test will enable oncologists to tailor treatments more effectively, offering new hope for individuals managing this aggressive form of breast cancer," commented Dr. Miguel Martin. He added, "The ability of TNBCDX to provide critical genomic insights, with a special focus on the immune system, marks an important step forward in the treatment of TNBC."

Dr. Patricia Villagrasa, CEO and Co-founder of REVEAL GENOMICS, added, "The introduction of TNBCDX into clinical practice will transform the treatment landscape for those diagnosed with TNBC. We are committed to ensuring global access to this important diagnostic tool."

About the TNBCDX Test
TNBCDX is the first genomic test designed specifically for patients with early-stage TNBC. The test integrates clinical variables with key tumor and immune biological factors across 15 genes to provide a comprehensive risk assessment, guiding treatment decisions in a clinical setting. TNBCDX includes two scores: a risk score and a pCR (pathological complete response) likelihood score. Additionally, the test measures the levels of ERBB2 to ensure that the tumor is accurately classified as TNBC.

TNBCDX is currently available in a Research Use Only (RUO) format. The company is in the process of standardizing the assay, with plans to offer it through the Biomedical Diagnostic Center (CDB) at Hospital Clínic by 2025.

About TNBC
TNBC accounts for approximately 15-20% of all breast cancer diagnoses. Unlike other breast cancer subtypes, TNBC does not express estrogen receptor, progesterone receptor, or HER2, making it more difficult to treat with conventional therapies. TNBC is known for its aggressive nature and poor prognosis; however, it also exhibits substantial biological and clinical heterogeneity, underscoring the need for more precise diagnostic and therapeutic approaches.

On September 12, 2024 Vironexis Biotherapeutics, focused on transforming the future of cancer treatment by pioneering AAV-delivered T-cell immunotherapy, reported the company launched from stealth, unveiling its TransJoin AAV Gene Therapy Platform and a pipeline of more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine (Press release, Vironexis Biotherapeutics, SEP 12, 2024, View Source [SID1234646797]). Vironexis’s $26 million seed financing was led by Drive Capital and Future Ventures, with participation from Moonshots Capital and Capital Factory. The company has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for VNX-101, its first gene therapy product candidate, for the treatment of CD19+ acute lymphoblastic leukemia. Vironexis anticipates initiating patient enrollment of a Phase 1/2 trial of VNX-101 in the fourth quarter of 2024, which will mark the first-ever clinical trial of an AAV-delivered cancer immunotherapy. VNX-101 has received both Fast Track Designation and Rare Pediatric Disease Designation from the FDA.

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"We’re excited to launch Vironexis from stealth and reveal our noteworthy progress advancing AAV-delivered T-cell immunotherapy," said Samit Varma, co-founder and CEO of Vironexis. "Our novel technology builds on the power of T-cell immunotherapy while overcoming key shortcomings and challenges of existing approaches such as CAR-T and bispecific antibodies. We believe we have the opportunity to dramatically improve upon the safety, efficacy and durability of these drug classes, while streamlining manufacturing and significantly lessening the burden of treatment for patients. Our focus on execution has yielded an expansive pipeline and a clinic-ready lead program in just three years. We’re working as quickly as possible to transform the future of cancer treatments for patients."

TransJoin enables a patient’s body to express an engineered transgene that redirects T cells throughout the body to tumor cells. The proprietary technology requires only a single dose and provides a bridge that joins together T cells and tumor cells to promote long-term, continuous T cell-mediated tumor killing. The foundational technology for TransJoin was licensed from Nationwide Children’s Hospital. In 2022, research led by Timothy Cripe, M.D., Ph.D., Chief of the Division of Pediatric Hematology/Oncology/Bone and Marrow Transplant, describing the TransJoin technology was published in Science Advances [link here to paper]. Dr. Cripe is one of Vironexis’s co-founders, along with Mr. Varma, a seasoned biotechnology and gene therapy company entrepreneur, and Brian Kaspar, Ph.D., a notable gene therapy scientist-entrepreneur who founded AveXis, Inc. (acquired by Novartis) and pioneered the AAV gene therapy ZOLGENSMA.

"AAV is a proven delivery technology with multiple approvals since 2017. Recognizing the pivotal impact of AAV delivery for the treatment of rare diseases, we believed its unique ability to enable long-term, continuous expression of a therapeutic protein could be the missing link to overcome the myriad challenges associated with first-generation T-cell immunotherapies like CAR-T. We subsequently demonstrated the potential of this approach in the preclinical setting," said Dr. Cripe. "It’s thrilling to now be on the cusp of seeing how this technology translates in the clinical setting. Our ultimate goal is to help a vastly broader population of patients realize the tremendous benefits of T-cell immunotherapy."

Vironexis is in the process of obtaining pre-IND input from the FDA for its second program, VNX-202, as a treatment for the prevention of metastatic HER2+ cancer (including breast cancer and other tumor types), and plans to start dosing patients in a Phase 1/2 clinical trial in 2025. The company’s other current product candidates include treatments for BCMA/GPRC5D+ multiple myeloma, CD19/20+ B-cell lymphoma; treatments to prevent metastases in GD2+ neuroblastoma, HER2+ gastric cancer, PSMA+ and MSLN+ pancreatic cancer, B7H3+ ostersarcoma and GP350+ nasopharyngeal cancer; a cancer vaccine for GP350+ nasopharyngeal cancer; and a treatment for CD19+ systemic lupus erythematosus, which Vironexis plans to partner for further development.

Molly Bonakdarpour, Partner at Drive Capital commented, "As a portfolio investment, Vironexis offers an ideal blend of groundbreaking technology, impressive preclinical data, the potential for vast patient impact, and a founding team with deep, relevant expertise and a proven track record of company formation, strategic thinking, and successful execution. Vironexis’s notable productivity in a very short timeframe has been remarkable. We look forward to its upcoming transition to a clinical-stage company."

Steve Jurvetson, Co-Founder of Future Ventures, added, "We were drawn not only to the novelty of the TransJoin technology but also to its broad applicability, spanning treatments for blood-based cancers, solid tumor metastasis prevention, and cancer vaccines, as well as immune disorders. The versatility of this platform is truly standout, and the Vironexis team’s progress in rapidly building a pipeline that explores the expanse of these opportunities is extraordinary."

How TransJoin Works
Vironexis’s TransJoin technology is designed to enable the expression of a secreted T cell engager that binds the tumor cell on one side (changeable depending on the product candidate indication target) and T cells via CD3 on the other side. CD3 is a protein that helps promote T-cell recognition of and activation against cancer cells. Following a single, one-time intravenous infusion, the TransJoin technology instructs the liver to continuously secrete the bispecific protein into the bloodstream to redirect T cells to tumor cells. TransJoin provides a bridge that joins T cells and tumor cells, resulting in long-term, consistent serum levels of the therapy and, thus, long-term, consistent T-cell-mediated tumor cell killing. TransJoin’s extremely low-dose AAV delivery minimizes toxicity and adverse events.