Ascendis to Present First Results from Platinum-Resistant Ovarian Cancer (PROC) Cohort of the Phase 1/2 IL-Believe Trial at ESMO 2024

On September 13, 2024 Ascendis Pharma A/S reported initial data showing signs of clinical activity in heavily pre-treated patients with platinum-resistant ovarian cancer (PROC) treated with TransCon IL-2 β/γ in combination with chemotherapy in its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β/γ (Press release, Ascendis Pharma, SEP 13, 2024, View Source [SID1234646562]). First results will be shared in Poster 762P at ESMO (Free ESMO Whitepaper) 2024, the annual meeting of the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) being held in Barcelona from September 13-17, 2024.

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Of the 18 patients (median age 64 years) included in the initial data, 14 were efficacy evaluable patients who had 1 or more post-baseline tumor assessment(s), plus an additional 4 who discontinued treatment before the first post-baseline tumor assessment due to disease progression or death. Anti-tumor clinical responses were observed in 29% (4/14) of the efficacy evaluable patients (2 confirmed and 2 unconfirmed partial responses in patients who had received three to seven prior lines of treatment – including patients whose disease had previously progressed on Elahere, also called mirvetuximab soravtansine-gynx), suggesting clinical activity in heavily pre-treated patients. The data suggest that TransCon IL-2 β/γ was generally well-tolerated: the most common treatment-emergent adverse events related to combination therapy with TransCon IL-2 β/γ plus chemotherapy were fatigue, thrombocytopenia, neutropenia, and anemia. Most TransCon IL-2 β/γ-related TEAEs were grade 1 or 2.

"Building on results announced at ASCO (Free ASCO Whitepaper) 2024 in melanoma, we are excited now to see meaningful signs of anti-tumor activity in combination with chemotherapy in our second indication-specific cohort of heavily pretreated patients who have exhausted standard-of-care options," said Stina Singel, M.D., Ph.D., Executive Vice President, Head of Clinical Development, Oncology, at Ascendis Pharma. "We look forward to providing further updates as patients continue on study treatment."

About TransCon IL-2 β/γ & IL-Believe
TransCon IL-2 β/γ is a novel prodrug with sustained release of an IL-2Rβ/γ-selective IL-2 analogue (IL-2 β/γ). IL-2 β/γ is transiently attached to an inert carrier by a TransCon linker, which under physiological conditions releases active IL-2 β/γ in a predictable, sustained manner. This results in lower Cmax and longer half-life, which is expected to widen the therapeutic index.

TransCon IL-2 β/γ is being investigated in IL Believe, a multicenter Phase 1/2, multi-cohort study in adult patients with locally advanced or metastatic solid tumors. As of the July 29, 2024, data cut off, 42 patients whose disease had progressed within six months after completing platinum-based chemotherapy were enrolled in the PROC dose expansion cohort (Cohort 3 in the trial). Treatment for patients in Cohort 3 included intravenous TransCon IL-2 β/γ at the recommended Phase 2 dose of 120 μg/kg every 3 weeks in combination with the physician’s choice of paclitaxel, docetaxel, or pemetrexed. Disease response was assessed every 9 weeks using RECIST v1.1. and safety, efficacy, and biomarkers were evaluated.

AMGEN TO PRESENT DATA FROM MULTIPLE EARLY-STAGE CLINICAL TRIALS AT ESMO 2024

On September 13, 2024 Amgen reported the presentation of new data across its broad oncology pipeline and portfolio at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024, taking place Sept. 13-17 in Barcelona (Press release, Amgen, SEP 13, 2024, View Source [SID1234646561]). The abstracts showcase data from Amgen-sponsored and investigator-sponsored studies for colorectal, lung, prostate and gastric cancers using molecularly targeted modalities.

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"The breadth of these data reflects our strategy to advance diverse modalities for difficult-to-treat cancers," said Jay Bradner, M.D., executive vice president, Research and Development, and chief scientific officer at Amgen. "These ESMO (Free ESMO Whitepaper) results underscore our leadership in oncology, contributing significant advancements with both investigational and established therapies. Guided by a deep understanding of cancer biology and leveraging incisive therapeutics, we can target dominant drivers of disease with unprecedented precision."

Key presentations include:

First findings from the Phase 1b study of LUMAKRAS plus Vectibix in combination with FOLFIRI in first-line patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC).
Phase 1 dose escalation and initial dose expansion data from AMG 193 selected for a presidential symposium session.
First-in-human study of xaluritamig in men with metastatic castration-resistant prostate cancer (mCRPC).
For more information on the Amgen abstracts, see below.

Abstracts and Presentation Times:

Amgen Sponsored Abstracts

LUMAKRAS (sotorasib) plus Vectibix (panitumumab)

Sotorasib (soto) + panitumumab (pani) and FOLFIRI in the first line (1L) setting for KRAS-G12C mutated metastatic colorectal cancer (mCRC): Safety and efficacy from the phase 1b CodeBreaK 101 study
Abstract #505O, Proffered Paper Oral Session: 2, Madrid Auditorium – Hall 2, Sunday, September 15 from 3:05 – 3:15 p.m. CEST
AMG 193

Phase 1 dose escalation and initial dose expansion results of AMG 193, an MTA-cooperative PRMT5 inhibitor, in patients (pts) with MTAP-deleted solid tumors
Abstract #3482, Proffered Paper Oral Session: Presidential Symposium III: Eyes to the Future, Barcelona Auditorium – Hall 2, Monday, September 16 from 16:30 – 18:15 p.m. CEST
Xaluritamig

Circulating tumor cell (CTC) enumeration and overall survival (OS) in men with metastatic castration-resistant prostate cancer (mCRPC) treated with xaluritamig
Abstract #1610P, Poster, September 15
Xaluritamig, a STEAP1 x CD3 XmAb 2+1 immune therapy, in patients with metastatic castration-resistant prostate cancer (mCRPC): Initial results from dose expansion cohorts in a Phase 1 study
Abstract #1598P, Poster, September 15
LUMAKRAS (sotorasib) for NSCLC

Sotorasib long-term clinical outcomes in pre-treated KRAS G12C-mutated advanced NSCLC: pooled analysis from the CodeBreaK clinical trials
Abstract #1305P, Poster, September 14
Clinical characteristics and therapeutic sequences of KRAS G12C advanced Non-Small Cell Lung Cancer (aNSCLC) patients (pts) treated by sotorasib in the French post-marketing authorization early access (post-MA EA)
Abstract #1307P, Poster, September 14
Bemarituzumab

Fibroblast growth factor receptor 2 isoform IIIb (FGFR2b) protein overexpression and biomarker overlap in patients with advanced gastric or gastroesophageal junction cancer (GC/GEJC)
Abstract #1420P, Poster, September 16
Investigator Sponsored Studies

Vectibix (panitumumab)

**mRNA profiling as a biomarker of prognosis and response to first-line treatment in metastatic colorectal cancer: discovery and validation of a gene expression signature in three randomized trials
Abstract #581P, Poster, September 16
Circulating tumor DNA driving anti-EGFR rechallenge therapy in metastatic colorectal cancer: the RASINTRO prospective multicenter study
Abstract #517P, Poster, September 16
Prospective validation of the metastatic colon cancer score (mCCS) in patients with RAS wild-type metastatic colorectal cancer treated with first-line panitumumab plus FOLFIRI/FOLFOX: Final results of the non-interventional study VALIDATE
Abstract #585P, Poster, September 16
About LUMAKRAS/LUMYKRAS (sotorasib)
LUMAKRAS received accelerated approval from the U.S. Food and Drug Administration (FDA) on May 28, 2021. The U.S. FDA completed its review of Amgen’s supplemental New Drug Application (sNDA) seeking full approval of LUMAKRAS on December 26, 2023, which resulted in a complete response letter. In addition, the FDA concluded that the dose comparison postmarketing requirement (PMR) issued at the time of LUMAKRAS accelerated approval, to compare the safety and efficacy of LUMAKRAS 960 mg daily dose versus a lower daily dose, has been fulfilled. 960 mg once-daily is the indicated dose for patients with KRAS G12C-mutated NSCLC under accelerated approval. The U.S. FDA also issued a new PMR for an additional confirmatory study to support full approval that will be completed no later than February 2028.

About Metastatic Colorectal Cancer and the KRAS G12C Mutation
Colorectal cancer (CRC) is the second leading cause of cancer deaths worldwide, comprising 10% of all cancer diagnoses.1 It is also the third most commonly diagnosed cancer globally.2

KRAS mutations are among the most common genetic alterations in colorectal cancers, with the KRAS G12C mutation present in approximately 3-5% of colorectal cancers.3-5

About Advanced Non-Small Cell Lung Cancer and the KRAS G12C Mutation
Lung cancer is the leading cause of cancer-related deaths worldwide, and it accounts for more deaths worldwide than colon cancer, breast cancer and prostate cancer combined.6

KRAS G12C is the most common KRAS mutation in NSCLC.7 About 13% of patients with non-squamous NSCLC harbor the KRAS G12C mutation.8

About CodeBreaK
The CodeBreaK clinical development program for Amgen’s drug sotorasib is designed to study patients with an advanced solid tumor with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers.

Amgen also has several Phase 1b studies investigating sotorasib monotherapy and sotorasib combination therapy across various advanced solid tumors (CodeBreaK 101) open for enrollment.9 A Phase 2 randomized study evaluating sotorasib in patients with stage IV KRAS G12C-mutated NSCLC in need of first-line treatment is ongoing (CodeBreaK 201).10 A Phase 3 study of LUMAKRAS plus carboplatin and pemetrexed as front-line therapy in KRAS G12C-mutant, programmed death-ligand 1 (PD-L1) negative advanced NSCLC is enrolling patients (CodeBreaK 202). A Phase 3 study of LUMAKRAS in combination with Vectibix and FOLFIRI in first-line KRAS G12C–mutated CRC is also enrolling patients (CodeBreak-301).

LUMAKRAS (sotorasib) U.S. Indication
LUMAKRAS is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.

This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

LUMAKRAS (sotorasib) Important U.S. Safety Information

Hepatotoxicity

LUMAKRAS can cause hepatotoxicity, which may lead to drug-induced liver injury and hepatitis.
Among 357 patients who received LUMAKRAS in CodeBreaK 100, hepatotoxicity occurred in 1.7% (all grades) and 1.4% (Grade 3). A total of 18% of patients who received LUMAKRAS had increased alanine aminotransferase (ALT)/increased aspartate aminotransferase (AST); 6% were Grade 3 and 0.6% were Grade 4. In addition to dose interruption or reduction, 5% of patients received corticosteroids for the treatment of hepatotoxicity.
Monitor liver function tests (ALT, AST and total bilirubin) prior to the start of LUMAKRAS every 3 weeks for the first 3 months of treatment, then once a month or as clinically indicated, with more frequent testing in patients who develop transaminase and/or bilirubin elevations.
Withhold, reduce or permanently discontinue LUMAKRAS based on severity of adverse reaction.
Interstitial Lung Disease (ILD)/Pneumonitis

LUMAKRAS can cause ILD/pneumonitis that can be fatal. Among 357 patients who received LUMAKRAS in CodeBreaK 100, ILD/pneumonitis occurred in 0.8% of patients, all cases were Grade 3 or 4 at onset, and 1 case was fatal. LUMAKRAS was discontinued due to ILD/pneumonitis in 0.6% of patients.
Monitor patients for new or worsening pulmonary symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever). Immediately withhold LUMAKRAS in patients with suspected ILD/pneumonitis and permanently discontinue LUMAKRAS if no other potential causes of ILD/pneumonitis are identified.
Most Common Adverse Reactions

The most common adverse reactions occurring in ≥ 20% were diarrhea, musculoskeletal pain, nausea, fatigue, hepatotoxicity and cough.
Drug Interactions

Advise patients to inform their healthcare provider of all concomitant medications, including prescription medicines, over-the-counter drugs, vitamins, dietary and herbal products.
Inform patients to avoid proton pump inhibitors and H2 receptor antagonists while taking LUMAKRAS.
If coadministration with an acid-reducing agent cannot be avoided, inform patients to take LUMAKRAS 4 hours before or 10 hours after a locally acting antacid.
Please see LUMAKRAS full Prescribing Information.

About Vectibix (panitumumab)
Vectibix is the first and only fully human monoclonal anti-EGFR antibody approved by the FDA for the treatment of mCRC. Vectibix was approved in the U.S. in September 2006 as a monotherapy for the treatment of patients with EGFR-expressing mCRC after disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.

In May 2014, the FDA approved Vectibix for use in combination with FOLFOX as first-line treatment in patients with wild-type KRAS (exon 2) mCRC. With this approval, Vectibix became the first-and-only biologic therapy indicated for use with FOLFOX, one of the most commonly used chemotherapy regimens, in the first-line treatment of mCRC for patients with wild-type KRAS mCRC.

In June 2017, the FDA approved a refined indication for Vectibix for use in patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) mCRC.

INDICATION AND LIMITATION OF USE

Vectibix is indicated for the treatment of patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) metastatic colorectal cancer (mCRC): as first-line therapy in combination with FOLFOX, and as monotherapy following disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.

Limitation of Use: Vectibix is not indicated for the treatment of patients with RAS mutant mCRC or for whom RAS mutation status is unknown.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: DERMATOLOGIC TOXICITY

Dermatologic Toxicity: Dermatologic toxicities occurred in 90% of patients and were severe (NCI-CTC grade 3 and higher) in 15% of patients receiving Vectibix monotherapy [see Dosage and Administration (2.3), Warnings and Precautions (5.1), and Adverse Reactions (6.1)].

In Study 20020408, dermatologic toxicities occurred in 90% of patients and were severe (NCI-CTC grade 3 and higher) in 15% of patients with mCRC receiving Vectibix. The clinical manifestations included, but were not limited to, acneiform dermatitis, pruritus, erythema, rash, skin exfoliation, paronychia, dry skin, and skin fissures.
Monitor patients who develop dermatologic or soft tissue toxicities while receiving Vectibix for the development of inflammatory or infectious sequelae. Life-threatening and fatal infectious complications including necrotizing fasciitis, abscesses, and sepsis have been observed in patients treated with Vectibix. Life-threatening and fatal bullous mucocutaneous disease with blisters, erosions, and skin sloughing has also been observed in patients treated with Vectibix. It could not be determined whether these mucocutaneous adverse reactions were directly related to EGFR inhibition or to idiosyncratic immune- related effects (e.g., Stevens Johnson syndrome or toxic epidermal necrolysis). Withhold or discontinue Vectibix for dermatologic or soft tissue toxicity associated with severe or life-threatening inflammatory or infectious complications. Dose modifications for Vectibix concerning dermatologic toxicity are provided in the product labeling.
Vectibix is not indicated for the treatment of patients with colorectal cancer that harbor somatic RAS mutations in exon 2 (codons 12 and 13), exon 3 (codons 59 and 61), and exon 4 (codons 117 and 146) of either KRAS or NRAS and hereafter is referred to as "RAS."
Retrospective subset analyses across several randomized clinical trials were conducted to investigate the role of RAS mutations on the clinical effects of anti-EGFR-directed monoclonal antibodies (panitumumab or cetuximab). Anti-EGFR antibodies in patients with tumors containing RAS mutations resulted in exposing those patients to anti-EGFR related adverse reactions without clinical benefit from these agents. Additionally, in Study 20050203, 272 patients with RAS-mutant mCRC tumors received Vectibix in combination with FOLFOX and 276 patients received FOLFOX alone. In an exploratory subgroup analysis, OS was shorter (HR = 1.21, 95% CI: 1.01-1.45) in patients with RAS-mutant mCRC who received Vectibix and FOLFOX versus FOLFOX alone.
Progressively decreasing serum magnesium levels leading to severe (grade 3-4) hypomagnesemia occurred in up to 7% (in Study 20080763) of patients across clinical trials. Monitor patients for hypomagnesemia and hypocalcemia prior to initiating Vectibix treatment, periodically during Vectibix treatment, and for up to 8 weeks after the completion of treatment. Other electrolyte disturbances, including hypokalemia, have also been observed. Replete magnesium and other electrolytes as appropriate.
In Study 20020408, 4% of patients experienced infusion reactions and 1% of patients experienced severe infusion reactions (NCI-CTC grade 3-4). Infusion reactions, manifesting as fever, chills, dyspnea, bronchospasm, and hypotension, can occur following Vectibix administration. Fatal infusion reactions occurred in postmarketing experience. Terminate the infusion for severe infusion reactions.
Severe diarrhea and dehydration, leading to acute renal failure and other complications, have been observed in patients treated with Vectibix in combination with chemotherapy.
Fatal and nonfatal cases of interstitial lung disease (ILD) (1%) and pulmonary fibrosis have been observed in patients treated with Vectibix. Pulmonary fibrosis occurred in less than 1% (2/1467) of patients enrolled in clinical studies of Vectibix. In the event of acute onset or worsening of pulmonary symptoms interrupt Vectibix therapy. Discontinue Vectibix therapy if ILD is confirmed.
In patients with a history of interstitial pneumonitis or pulmonary fibrosis, or evidence of interstitial pneumonitis or pulmonary fibrosis, the benefits of therapy with Vectibix versus the risk of pulmonary complications must be carefully considered.
Exposure to sunlight can exacerbate dermatologic toxicity. Advise patients to wear sunscreen and hats and limit sun exposure while receiving Vectibix.
Keratitis and ulcerative keratitis, known risk factors for corneal perforation, have been reported with Vectibix use. Monitor for evidence of keratitis or ulcerative keratitis. Interrupt or discontinue Vectibix for acute or worsening keratitis.
In an interim analysis of an open-label, multicenter, randomized clinical trial in the first-line setting in patients with mCRC, the addition of Vectibix to the combination of bevacizumab and chemotherapy resulted in decreased OS and increased incidence of NCI-CTC grade 3-5 (87% vs 72%) adverse reactions. NCI-CTC grade 3-4 adverse reactions occurring at a higher rate in Vectibix-treated patients included rash/acneiform dermatitis (26% vs 1%), diarrhea (23% vs 12%), dehydration (16% vs 5%), primarily occurring in patients with diarrhea, hypokalemia (10% vs 4%), stomatitis/mucositis (4% vs < 1%), and hypomagnesemia (4% vs 0).
NCI-CTC grade 3-5 pulmonary embolism occurred at a higher rate in Vectibix-treated patients (7% vs 3%) and included fatal events in three (< 1%) Vectibix-treated patients. As a result of the toxicities experienced, patients randomized to Vectibix, bevacizumab, and chemotherapy received a lower mean relative dose intensity of each chemotherapeutic agent (oxaliplatin, irinotecan, bolus 5-FU, and/or infusional 5-FU) over the first 24 weeks on study compared with those randomized to bevacizumab and chemotherapy.
Vectibix can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment, and for at least 2 months after the last dose of Vectibix.
In monotherapy, the most commonly reported adverse reactions (≥ 20%) in patients with Vectibix were skin rash with variable presentations, paronychia, fatigue, nausea, and diarrhea.
The most commonly reported adverse reactions (≥ 20%) with Vectibix + FOLFOX were diarrhea, stomatitis, mucosal inflammation, asthenia, paronychia, anorexia, hypomagnesemia, hypokalemia, rash, acneiform dermatitis, pruritus, and dry skin. The most common serious adverse reactions (≥ 2% difference between treatment arms) were diarrhea and dehydration.
To see the Vectibix Prescribing Information, including Boxed Warning visit www.vectibix.com.

Alligator Bioscience and Aptevo Therapeutics present at ESMO Congress 2024: positive interim data from the phase 1 study evaluating ALG.APV-527 as monotherapy in several solid tumor types

On September 13, 2024 Alligator Bioscience AB and Aptevo Therapeutics reported that positive interim data from the dose escalation portion of the companies’ Phase 1 study evaluating ALG.APV-527 will be presented at a poster on Saturday, September 14, 2024, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress, taking place September 13-17, 2024, in Barcelona, ​​Spain (Press release, Alligator Bioscience, SEP 13, 2024, View Source [SID1234646560]).

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Information om presentationen
Posternummer: #668P
Titel: First-in-Human Phase I Dose Escalation Study of ALG.APV-527, a 5T4 Tumor Antigen-Conditional 4-1BB Bispecific Antibody, in Patients with Advanced Solid Tumors, Demonstrates Positive Safety, Signals of Biological Activity and Patients with Lasting Stable Disease
Presentatör: Thomas Marron, MD, PhD
Datum: Lördag, 14 september, 2024

About the study
The Phase 1 study with ALG.APV-527 is an open-label, multicenter study that will include six cohorts in a 3+3 design*. The study will be conducted in adult patients with multiple solid tumor types/histologies likely to express the 5T4 antigen, including (but not limited to) non-small cell lung cancer (NSCLC), gastric/gastroesophageal cancer, and head and neck cancer, in up to 10 clinics in the US. ALG.APV-527 will be administered intravenously every two weeks. The study will evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary anti-tumor activity of ALG.APV-527.

*In a 3+3 design, cohorts of three patients are treated with increasing dose levels. If at least two out of three or six patients suffer a so-called Dose Limiting Toxicity (DLT), the dose escalation is stopped at the current dose level.

About ALG.APV-527
ALG.APV-527 is a bispecific 4-1BB agonist, which is active only upon simultaneous binding to 4-1BB and 5T4. The molecule has the potential to be of clinical utility as 4-1BB has the ability to stimulate the immune cells (anti-tumor-specific T cells) involved in tumor fighting, making 4-1BB a particularly attractive target for cancer immunotherapy. 5T4 is a tumor-associated antigen that is expressed during fetal development but is also overexpressed on a number of solid tumors, including non-small cell lung cancer (NSCLC), breast, head and neck, cervical, renal, gastric, colon and rectal cancers.

Preclinical studies have been published in the peer-reviewed publication Molecular Cancer Therapeutics , a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The study, which highlights the molecule’s differentiated design that minimizes systemic immune activation, while enabling highly effective tumor-specific responses, demonstrates the drug candidate’s potent activity in preclinical models.

Immuneering Announces Positive Initial Phase 2a Data Including Complete and Partial Responses with IMM-1-104 in Combination with Chemotherapy in First-Line Pancreatic Cancer Patients

On September 12, 2024 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company seeking to develop and commercialize universal-RAS/RAF medicines for broad populations of cancer patients, reported positive initial response data from the first five patients treated with IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel in first line pancreatic cancer as part of its ongoing Phase 2a clinical trial (Press release, Immuneering, SEP 12, 2024, View Source [SID1234647154]).

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"We are delighted to share today’s initial data on IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel. While the initial ORR of 40% and Disease Control Rate of 80% are very encouraging – and both more than would be expected for gemcitabine/nab-paclitaxel alone- we are still in the early stages of this trial, with more scans for all five of these initial patients and for additional patients planned to come. Nevertheless, it was encouraging to see a complete response in the very first pancreatic cancer patient treated with IMM-1-104 in this combination, with the patient now on treatment for over six months," said Ben Zeskind, Ph.D., Co-Founder and CEO of Immuneering. "Looking at the bigger picture, our Phase 2a trial aims to evaluate the efficacy of IMM-1-104 in multiple settings across various tumor types, to identify the highest priority opportunities for future development. If the early trends with IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel continue, we will have an exciting direction for potential future development of IMM-1-104, which could greatly improve the prognosis for a drastically underserved patient population."

Initial Results from Phase 2a Arm Evaluating IMM-1-104 with Modified Gemcitabine/nab-Paclitaxel in First Line Pancreatic Cancer as of September 12, 2024

Patient MAPK
Mutation
Variant Dose (p.o.)
Level for
IMM-1-104 %
Change
in SLD
1st Scan %
Change
in SLD
2nd Scan %
Change
in SLD
3rd Scan %
Change
in SLD
4th Scan %
Change
in SLD
5th Scan ORR/
RECIST
1 GNAS-T105Vfs*3 (*) 240mg QD -100% -100% -100% -100% next
scan CR
2 KRAS-G12V* 240mg QD -8% -10% -40% next
scan uPR°
3 KRAS-G12V* 240mg QD -4% next
scan SD
4 Unk.# 240mg QD +6% next
scan SD
5 KRAS-G12R* 240mg QD -9% next
scan eqPD**
Initial Overall Response Rate
(ORR): 40%
Initial Disease Control Rate (DCR): 80%

* Detected in plasma cfDNA or prior genomic test.
# Unknown (Unk.); MAPK pathway variant not detected in plasma cfDNA or prior genomic test.
° Partial response result classified as "unconfirmed" pending subsequent scan.
**Equivocal (eq); Patient not dosed for over two weeks during hospitalization for a preexisting condition. Scans showed ascites and a pleural effusion categorized by radiology as equivocal new lesions per RECIST 1.1. The investigator determined these to be related to the recent placement of a hepatic stent, not disease progression, and stated that the patient is improving and remains on therapy.

Source: Immuneering Corporation

To date, the first two patients in the Phase 2a arm evaluating IMM-1-104 with modified gemcitabine/nab-paclitaxel in first-line pancreatic cancer have recorded complete or partial responses for an initial response rate of 40% (2/5) and disease control rate of 80% (4/5), with the other three patients earlier in the course of treatment and all five continuing on treatment.
Benchmarks for gemcitabine/nab-paclitaxel alone in first-line pancreatic cancer patients were established by the Phase 3 MPACT study, which included 1 Complete Response (CR) out of 431 patients, a 23% Overall Response Rate, and a 48% Disease Control Rate1. Benchmarks for modified (m) Gemcitabine/nab-Paclitaxel include an 18.6% ORR2.
To date, the combination of IMM-1-104 plus modified gemcitabine/nab-paclitaxel was observed to be well tolerated, with an emerging safety profile in line with known data for both therapeutics respectively.
Based on safety data to date, the trial’s Data and Safety Monitoring Board (DSMB) has approved enrolling additional patients into this arm at 320mg QD p.o., the first of which have already been dosed and are awaiting first scans.
[1] Von Hoff, et al. N Engl J Med. 2013;369:1691-1703, [2] Ahn DH, et al. Therapeutic Advances in Medical Oncology. 2017;9(2):75-82

"These exciting early clinical findings are consistent with the preclinical data we shared at AACR (Free AACR Whitepaper) earlier this year, which pointed to synergies between IMM-1-104 and chemotherapeutics – driving deeper more durable responses than either can achieve alone," said Brett Hall, Ph.D., Chief Scientific Officer, Immuneering Corporation. "If the combination data for IMM-1-104 continues to be positive – and taking into account the excellent emerging safety profile for IMM-1-104 – one can imagine the drug’s potential inclusion in various vertical drug combinations, immune-modifying combinations, and orthogonal combinations with therapeutics with non-overlapping mechanisms of action, which Immuneering may in the future develop both on its own and in partnership with third parties."

"There is a high unmet need in pancreatic cancer for novel therapies that meaningfully improve outcomes. With current therapies in pancreatic cancer, we rarely see complete responses, and as such any treatment that leads to one is exciting and deserves further investigation, particularly when observed in the setting of a well-tolerated agent such as IMM-1-104," said Tanios Bekaii-Saab, M.D., Leader of the Gastrointestinal Cancer Disease Group for the Mayo Clinic Cancer Center enterprise wide and Medical Oncology consultant in Mayo Clinic in Phoenix, Arizona.

In the Phase 2a portion of Immuneering’s ongoing IMM-1-104 Phase 1/2a clinical trial, IMM-1-104 is being evaluated as both monotherapy and in combination with approved chemotherapeutic agents. The Phase 2a portion includes five arms, one of which focuses on patients with RAS mutant melanoma, another on patients with RAS mutant non-small cell lung cancer (NSCLC), and three arms focused on patients with pancreatic cancer. Immuneering previously announced that IMM-1-104 received fast track designation for the treatment of first- and second-line pancreatic cancer.

Near-Term Milestone Expectations

IMM-1-104

Initial data from at least one additional arm of the Phase 2a portion of the Company’s Phase 1/2a trial is expected by year end.

IMM-6-415

Initial pharmacokinetic (PK), pharmacodynamic (PD) and safety data from the Phase 1 portion of the Company’s Phase 1/2a trial is expected by year end.

Conference Call

Immuneering will host a conference call and live webcast at 4:30 p.m. ET / 1:30 p.m. PT on September 12, 2024, to discuss the results and provide a business update. Individuals interested in listening to the live conference call may do so by dialing (800) 715-9871 for U.S callers and (646) 307-1963 for other locations and reference conference ID 8890310, or from the webcast link in the "investors" section of the company’s website at www.immuneering.com A webcast replay will be available in the investor relations section on the company’s website for 90 days following the completion of the call.

About IMM-1-104

IMM-1-104 aims to achieve universal-RAS activity that selectively impacts cancer cells to a greater extent than healthy cells, through Deep Cyclic Inhibition of the MAPK pathway with once-daily dosing. IMM-1-104 is currently being evaluated in a Phase 1/2a study in patients with advanced solid tumors harboring RAS mutations (NCT05585320).

Vironexis Biotherapeutics Launches with FDA Clearance of IND Application for First-Ever Clinical Trial of an AAV-delivered Cancer Immunotherapy

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.