On May 30, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAN-3110, a next generation oncolytic viral immunotherapy, for the treatment of recurrent high-grade glioma (rHGG) (Press release, Candel Therapeutics, MAY 30, 2024, View Source [SID1234643880]). Glioblastoma (GBM) is the most common and aggressive form of high-grade glioma.

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CAN-3110 was previously granted Fast Track Designation by the FDA for the treatment of rHGG. Candel is currently evaluating CAN-3110 in a multi-institutional phase 1b clinical trial in rHGG. Results from Arm A of the ongoing phase 1b clinical trial in rHGG exploring the clinical and biomarker activity of a single dose administration of CAN-3110 were published in Nature, demonstrating a strong anti-tumoral response associated with extended survival.1 The Company will present data on the feasibility and safety of multiple doses of CAN-3110 in patients with rHGG, supported by the Break Through Cancer Foundation, in a trials-in-progress poster presentation at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.

"Building on the momentum of the FDA’s Fast Track Designation, recently granted to this program, the Orphan Drug Designation for CAN-3110 further reinforces the potential of this therapy and underscores the urgent need for novel and effective treatments for patients with rHGG," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "This designation not only reinforces our commitment to offering new hope and potential patient treatment options, but it also enables us to leverage development incentives and accelerate our efforts to evaluate new indications in the clinic. We are continuing our work in the phase 1b clinical trial of CAN-3110 and look forward to sharing further clinical updates in the second half of 2024."

E. Antonio Chiocca, M.D., Ph.D., Chair of the Department of Neurosurgery at Brigham and Women’s Hospital, Professor at Harvard Medical School, and Principal Investigator on the phase 1b clinical trial, said: "We are grateful to the FDA for recognizing the urgent need for new treatments in rHGG. Patients, and their families, affected by this disease, face immense challenges that the standard of care and conventional therapies have failed to adequately address. The early clinical data suggests that CAN-3110’s unique dual mechanism of action, combining oncolysis and immune activation, has the potential to overcome these challenges for rHGG patients."

About Orphan Drug Designation

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing exclusivity for the product for the designated orphan indication in the United States if the product is ultimately approved for its designated indication.

About CAN-3110

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) oncolytic viral immunotherapy candidate designed with dual activity for oncolysis and immune activation in a single therapeutic. Its activity is designed to be conditional to the expression of Nestin in cancer cells. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with recurrent high-grade glioma (rHGG). In October 2023, the Company announced that Nature published results from this ongoing clinical trial. CAN-3110 was well tolerated with no dose-limiting toxicity reported. In the clinical trial, the investigators observed median overall survival after a single CAN-3110 injection of more than 12 months in this therapy-resistant condition.1 The Company and academic collaborators are currently evaluating the effects of multiple CAN-3110 injections in rHGG, supported by the Break Through Cancer Foundation. CAN-3110 has previously received FDA Fast Track Designation for the treatment of rHGG.

Details on the CAN-3110 ASCO (Free ASCO Whitepaper) poster are as follows:

The trials-in-progress poster presentation will focus on cohort C of the ongoing phase 1b clinical trial of CAN-3110 in patients with rGBM, the most common form of rHGG. Previously presented data showed the ability of a single CAN-3110 injection to double median overall survival (mOS) in the rGBM population, as compared to contemporary control cohorts. Patients presenting with seropositivity to HSV1, reached mOS of 14 months, largely exceeding expected survival of 6 to 9 months or less for this population.

In cohort C, supported by the Break Through Cancer Foundation, two cohorts of 12 patients will receive up to six injections of CAN-3110 over a four-month period. Cohort C is currently exploring the safety and tolerability of CAN-3110 in patients with rGBM. Patients in cohort C are treated with up to six doses of CAN-3110 delivered by stereotactic injections on days 0, 15, 30, 60, 90 & 120, along with concomitant biopsies over the four-month treatment period.

Two sub-cohorts (1&2) of patients who will receive 1×107 pfu or 1×108 pfu per injection of CAN-3110 have been planned for six patients per cohort, using a Bayesian optimal interval (BOIN) design for dose ranging.

Six patients have accrued, completing cohort 1; no dose-limiting toxicities or severe adverse events were observed.
More than 300 core biopsies were obtained from all six patients across the planned time points.
Biopsies were processed for "-omic" analyses, including single-cell RNA sequencing, proteomics/phophoproteomic/immunopeptidomics, metabolomics, spatial transcriptomics, and cell profiling.
ASCO Presentation details are as follows:

Trials-in-Progress Poster Presentation Title: Longitudinal stereotactic injections of oncolytic immunoactivating rQNestin34.5v.2 (CAN-3110) with concomitant biopsies for "-omic" analyses in recurrent glioblastoma (GBM)
Presenter: David A. Reardon, MD, Professor of Medicine at Harvard Medical School; Clinical Director, Center for Neuro-Oncology at Dana Farber Cancer Institute
Session Title: Poster Session – Central Nervous System Tumors
Session Date/Time: Saturday, June 1, 2024; 9:00 AM – 12:00 PM CT

On May 30, 2024 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported that it has entered into a Supply Agreement with SpectronRx for the production of Cu-64 (Press release, Clarity Pharmaceuticals, MAY 30, 2024, View Source [SID1234643849]).

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Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are very excited to bring an additional Cu-64 manufacturer to our extensive and reliable network of copper radioisotope suppliers. SpectronRx will be the first private supplier of Cu-64 to join our network in the US.

"Cu-64, with an ideal 12.7-hour half-life, is able to overcome the overwhelming supply restraints of other diagnostic isotopes, specifically Ga-68 with a half-life of ~1 hour and F-18 with a half-life of ~2 hours. Radiopharmaceutical products using these isotopes are severely limited and are associated with significant manufacturing and supply complications, driven by these very short half-lives. This leaves many patients around the world with no option of positron emission tomography (PET) imaging. This is well documented in the United States, the largest oncology market in the world, and the effects of these hurdles more heavily impact vulnerable populations, including African Americans and Veterans, who already experience much higher incidences of prostate cancer than the general population1, 2.

"Our focus at Clarity is to establish the most reliable, scalable, and logistically seamless supply chain in the radiopharmaceutical field to continue delivering our best-in-class products to patients and their clinicians on time. With our TCTs, we can avoid the myriad of challenges associated with the current generation of diagnostic isotopes, such as Ga-68 and F-18, which require local production due to their short half-lives.

"The unique properties of Cu-64 include the ability to produce commercially relevant volumes of this isotope daily, on centrally located cyclotrons. Cu-64 can then be manufactured into ready-to-use products with a shelf life that is measured in days rather than hours and supply the growing demand for PET imaging agents. Should an imaging site require 1, 10 or 100+ patient doses, we see a future where these quantities can be reliably supplied to any zip code in the United States, removing the burden of the current supply issues with Ga-68 and F-18 based products from practices and their patients.

"We are now actively recruiting and imaging patients for our first Phase III trial with 64Cu-SAR-bisPSMA in a pre-prostatectomy setting and planning our second Phase III trial in biochemically recurrent prostate cancer with this optimised product. With outstanding clinical trial data to date, we continue to implement our strategy for the commercial launch of 64Cu-SAR-bisPSMA, developing a seamless supply chain for this potential best-in-class agent."

The overarching Master Service Agreement and associated Cu-64 supply agreement are effective as of 30 May 2024. The initial supply from SpectronRx is expected to launch before the end of calendar year 2024. The Master Services Agreement is for an initial period of five years and the Cu-64 supply agreement is for an initial period of 3 years. Cancellation and extension provisions are aligned with industry standard rates.

On May 30, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, reported that members of management will participate in the following upcoming investor conferences in June (Press release, Precision Biosciences, MAY 30, 2024, View Source [SID1234643865]):

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Jefferies Global Healthcare Conference
Date: Wednesday, June 5, 2024
Time: 1:30 pm – 1:55 pm ET
Format: Fireside Chat
Webcast Registration: Link

Goldman Sachs 45th Annual Global Healthcare Conference 2024
Date: Wednesday, June 12, 2024
Time: 10:00 am – 10:35 am ET
Format: Fireside Chat
Webcast Registration: Link

A live webcast of each presentation will also be accessible on Precision’s website in the Investors section under Events & Presentations at investor.precisionbiosciences.com. An archived replay of the webcasts will be available for approximately 30 days following each event.

On May 30, 2024 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported additional positive data from the ProstACT SELECT trial ("SELECT") of TLX591 (177Lu rosopatamab tetraxetan), a lutetium-labelled rADC therapy for the treatment of adult patients with PSMA-positive metastatic castrate-resistant prostate cancer (mCRPC) (Press release, Telix Pharmaceuticals, MAY 30, 2024, View Source [SID1234643881]). SELECT is a radiogenomics study intended to evaluate lesion concordance between 68Ga (gallium)-based PSMA-PET[4] imaging and TLX591 dosimetry for the purpose of validating PET imaging for patient selection for rADC therapy. The Company has previously reported final safety data from this study3.

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The study has reported a median rPFS of 8.8 months, representing an encouraging signal of the potential efficacy of TLX591 in this patient population. The evaluable sample size for rPFS comprised 23 patients with previously treated, progressive mCRPC and who received two 76 mCi intravenous (IV) infusions of TLX591, 14 days apart[5]. The SELECT trial included a heterogeneous population of low, medium and high disease burden patients to facilitate imaging cross-comparison, with the majority having undergone two prior lines of therapy.

Nat Lenzo, MD, Nuclear Oncologist and General Internal Medicine Physician and lead recruiter onto the SELECT trial, commented, "We are encouraged by this rPFS result, which compares favourably to small molecule radioligand therapy (RLT) Phase I and II studies at similar stages of development[6]. This is a compelling signal of the potential efficacy of TLX591 in this heavily pre-treated population. The results further support the development of this candidate in an earlier mCRPC patient population which is the focus of the ProstACT GLOBAL[7] Phase III trial and where there remains significant unmet need for effective treatment."

Dr David N. Cade, MD, Group Chief Medical Officer at Telix, stated, "TLX591 is a radio-ADC with significant potential advantages compared to small molecule radiopharmaceuticals in treating prostate cancer. TLX591 is differentiated by a patient-friendly dosing regimen with far lower cumulative radiation exposure compared to small molecule radioligand therapies[8]. This positive signal of efficacy from SELECT builds on prior studies that demonstrated the potential for TLX591 to deliver improved quality of life and durable tumour control in this advanced patient population[9]."

TLX591 is being further evaluated in the Phase III ProstACT GLOBAL trial in first and second line mCRPC, which is now preparing to enrol patients at its first U.S. sites. This innovative trial design allows physicians a choice of androgen receptor inhibition or docetaxel chemotherapy, thus integrating with real-world standard of care, reflective of Telix’s continued innovation in prostate cancer care and commitment to patient outcomes.

About TLX591

TLX591 (INN: lutetium Lu 177 rosopatamab tetraxetan) is Telix’s lead investigational radio antibody-drug conjugate (rADC) for the treatment of mCRPC, composed of a high-specificity PSMA-targeting antibody, chelator linker, and cytotoxic lutetium (177Lu) payload. TLX591 is administered intravenously under a two-dose fractionated regimen, potentially enabling the delivery of a highly targeted and potent dose with improved off-target organ radiation exposure. The mAb-based approach may offer distinct advantages in selectivity, internalisation, and retention time over small molecule RLTs for the treatment of mCRPC.

A total of 242 patients have been treated with TLX591 across eight Phase I and Phase II trials9 including a previously published Phase II (open-label, single-arm) trial, which reported a 42.3 month OS in 17 patients with advanced mCRPC when TLX591 was delivered under a fractionated dosing regimen[10].

About ProstACT SELECT

The purpose of the ProstACT SELECT trial is to evaluate the utility of PSMA-PET imaging with Illuccix to select patients for TLX591 rADC therapy. The primary objectives are to determine whole body biodistribution and organ radiation dosimetry, and assess the safety and tolerability of TLX591 in patients with advanced mCRPC. Radiographic progression-free survival (rPFS) is a secondary study objective.

Previously reported data from the SELECT trial includes3:

Confirmation of biodistribution and safety profile with a low rate of off-target side effects.
Confirmation of internalisation and long retention, delivering a payload to the tumour, potentially maximising cell killing effect.
Lower rates of haematologic toxicity than prior, later-line studies of TLX591.

On May 30, 2024 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN), a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported it is entitled to receive a milestone payment from AstraZeneca (LSE/STO/Nasdaq: AZN) triggered by the dosing of the first patient in a Phase 3 trial evaluating rilvegostomig, AstraZeneca’s PD-1/TIGIT bispecific antibody (Press release, Compugen, MAY 30, 2024, View Source [SID1234643850]). The TIGIT component of rilvegostomig is derived from Compugen’s clinical-stage anti-TIGIT antibody, COM902. Both rilvegostomig and COM902 are designed to have reduced Fc effector function.

The trial, called TROPION-Lung10, is evaluating the efficacy and safety of rilvegostomig as monotherapy and in combination with datopotamab deruxtecan (Dato-DXd), AstraZeneca and Daiichi Sankyo’s (TSE: 4568) TROP2-directed antibody drug conjugate versus pembrolizumab as first-line treatment for patients with locally advanced or metastatic non-squamous non-small cell lung cancer with high PD-L1 expression (TC ≥ 50%) and without actionable genomic alterations. The trial is sponsored by AstraZeneca in collaboration with Daiichi Sankyo and is expected to enrol approximately 675 patients in more than 14 countries. Further details about TROPION-Lung10 are available on ClinicalTrials.gov, identifier: NCT06357533.

"We are very excited to see the advancement of rilvegostomig into its second Phase 3 trial by AstraZeneca in collaboration with Daiichi Sankyo, two global leaders in oncology," said Anat Cohen-Dayag, Ph.D., President, and Chief Executive Officer of Compugen. "TROPION-Lung10 follows the start of the ARTEMIDE-Biliary01 Phase 3 trial evaluating rilvegostomig in biliary tract cancer, for which we received a $10 million milestone payment. Now, after dosing the first patient in this lung cancer trial, we are eligible to receive a $5 million milestone payment from AstraZeneca. Broadening the assessment of rilvegostomig reinforces our partnering strategy to expand opportunities for our pipeline and brings us closer to realizing potential future milestone payments and royalties."

The ARTEMIDE-Biliary01 Phase 3 trial is evaluating the efficacy and tolerability of rilvegostomig compared to placebo in combination with investigator’s choice of chemotherapy in patients with biliary tract cancer after surgical resection with curative intent. Further details about the ARTEMIDE-Biliary01 trial are available on ClinicalTrials.gov, identifier: NCT06109779.

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