On April 23, 2024 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported positive topline data from the investigator sponsored Phase 1 trial conducted by collaborators at Sant Joan de Déu-Barcelona Children’s Hospital (SJD) (Press release, Theriva Biologics, APR 23, 2024, View Source [SID1234642250]). The Phase 1 trial was designed to evaluate the safety and tolerability of two intravitreal injections of Theriva’s investigational oncolytic adenovirus VCN-01 in patients (n=9) with intraocular retinoblastoma that is refractory to chemotherapy or radiotherapy, and for whom enucleation was the only recommended treatment.

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"Results from the investigator sponsored trial further validate VCN-01’s unique mechanism of action and therapeutic potential to improve patient outcomes in otherwise refractory cancers," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We look forward to building on the encouraging safety profile and antitumor activity, which further supports and informs the design of our proposed Phase 2 clinical trial. The Monitoring Committee determined that the trial results were positive, and therefore, Theriva will receive an exclusive, worldwide license, and related patents from SJD for the treatment of pediatric patients with advanced retinoblastoma. The positive completion of this trial is an important step in refining our clinical strategy for VCN-01 as an adjunct to chemotherapy to address the high unmet need in this underserved indication."

Key Takeaways: Patients received two intravitreal injections of VCN-01, 14 days apart, at a dose of either 2 x 109 vp/eye (n=1) or 2 x 1010 vp/eye (n=8). The data for 9 evaluable patients were reviewed by the study Monitoring Committee who agreed that the trial had a positive outcome:

Safety: VCN-01 was well tolerated after intravitreal administration at the 2 doses and the most frequently reported treatment-related adverse events were Grade 1 or 2. There were no dose limiting toxicities and no ocular or systemic toxicities equal to or greater than Grade 3 during the evaluation period.
Some degree of ocular inflammation and associated turbidity was observed after VCN-01 injection. Inflammation was managed, and vitreous haze improved in some cases, by local and systemic administration of anti-inflammatory drugs.
Antitumor effects: intravitreal VCN-01 demonstrated promising antitumor activity and did not appear to change the retinal function.
Four patients presented a response characterized by unequivocal improvement in vitreous seed density.
Eye enucleation was avoided in 3 patients to date, one of whom has retained their eye after 4 years of follow-up.
About Retinoblastoma

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 – 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In the U.S., retinoblastoma shows an incidence rate of 3.3 per 1,000,000 with only about 200 to 300 children diagnosed per year according to the American Cancer Society. Preserving life and preventing the loss of an eye, blindness and other serious effects of treatment that reduce the patient’s life span or the quality of life, remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.

About VCN-01

VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

On April 23, 2024 PharmaMar Group (MSE: PHM) reported total revenues of €38.0 million, representing a 12% increase compared to the €34.0 million reported in the first quarter of 2023 (Press release, PharmaMar, APR 23, 2024, View Source [SID1234642249]). Recurring revenues, resulting from net sales plus royalties received from our partners, have increased by 15% to €31.7 million, compared to €27.4 million in the same period of the previous year.

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Sales in oncology have increased by 25% to €19.0 million. This increase is primarily due to commercial sales of Zepzelca in Europe amounting to €4.2 million, as well as raw material sales to our partners, both for Yondelis and Zepzelca, totaling €3.3 million, and revenue from the "early access" program, which increased by 12% to €6.3 million. These latter revenues mainly come from France, although there are also ongoing "early access" programs in countries such as Spain and Austria.

Yondelis sales in the European market, after the entry of generics, total €5.2 million (compared to €8.1 million in 1Q23).

As of March 31, 2024, royalty revenues amounted to €12.7 million, representing a 14% increase compared to the same period of the previous fiscal year. These revenues include royalties received from our partner Jazz Pharmaceuticals for lurbinectedin sales in the U.S., which have increased by 13% to €11.6 million. Royalties for the first quarter of 2024 are an estimate, as information on sales made by Jazz was not available as of the publication date of this report. Any discrepancies will be corrected in the following quarter.
In addition to royalties received from Jazz Pharmaceuticals, royalties for Yondelis sales from our partners in the U.S. and Japan amounted to €1.1 million in the first quarter of 2024, compared to €0.9 million in the same period of the previous fiscal year.

Regarding non-recurring revenues from licensing agreements, as of the end of the first quarter of 2024, these amounted to €6.0 million, of which €5.7 million correspond to the deferred revenue portion of the 2019 agreement with Jazz Pharmaceuticals regarding Zepzelca.

Investment in R&D reached €27.2 million in the first quarter of 2024, representing a 29% increase compared to the previous fiscal year.

Of the total R&D investment in this first quarter of 2024, the amount allocated to the oncology segment increased by 39% to €24.6 million, compared to €17.8 million in the first quarter of 2023. This increase is directly related to the significant increase in activity related to ongoing lurbinectedin clinical trials, primarily the LAGOON (phase III clinical development in small cell lung cancer indication) and SaLuDo (phase IIb/III clinical development in leiomyosarcoma indication) trials. Additionally, the company continues to invest in the clinical development of other molecules in earlier stages. In this regard, a phase II clinical trial with ecubectedin is underway in solid tumors, and phase I clinical trials are also underway, with ecubectedin, PM534 and PM54 for the treatment of solid tumors.

With all this, the PharmaMar Group reports a net profit of €2.3 million at the end of the first quarter of 2024.

As of March 31, 2024, PharmaMar Group has a cash and equivalents position of €164.5 million and reduced total debt by 8% since December 2023, to €36.8 million. Thus, the net cash position stands at €127.7 million

On April 23, 2024 Novartis reported that the U.S. Food and Drug Administration (FDA) approved Lutathera (USAN: lutetium Lu 177 dotatate / INN: lutetium (177Lu) oxodotreotide) for the treatment of pediatric patients 12 years and older with somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs (Press release, Novartis, APR 23, 2024, View Source [SID1234642248]). This approval makes Lutathera the first therapy specifically reviewed and approved for use in pediatric patients with GEP-NETs.

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"Lutathera is now the very first therapy approved specifically for children with GEP-NETs, offering new hope to young patients living with this rare cancer," said Tina Deignan, Therapeutic Area Head, Oncology US. "Radioligand therapies have extraordinary potential to shape the future of cancer care. With this approval, we have taken another vital step toward fulfilling that vision, strengthening our commitment to researching and developing the RLT platform across multiple cancer types and treatment settings."

NETs are a type of cancer that originates in neuroendocrine cells throughout the body and are commonly considered slow-growing malignancies1. The diagnosis of NETs is often delayed due to the inactive nature of the disease, and approximately 10% to 20% of pediatric patients are diagnosed with metastatic disease2,3. Even though NETs are an orphan disease, their incidence has increased over the past several decades1,4-6.

"While GEP-NETs in children and adolescents are rare, the impact can be devastating. Today’s approval addresses a critical need for new treatment options for these vulnerable patients," said Dr. Theodore Laetsch, trial investigator and Director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia (CHOP), a NETTER-P clinical trial site. "The introduction of radioligand therapy significantly advanced how we treat GEP-NETs, and I’m encouraged that younger patients now have the potential to benefit from this innovation."

The approval was based on the NETTER-P trial, which evaluated Lutathera in patients aged 12 to <18 years old with SSTR+ GEP-NETs7. The study reported a safety profile consistent with the adult population studied in NETTER-1, the pivotal trial for approval of Lutathera in adults. In addition, the estimated radiation absorbed dose in pediatric patients was within established organ thresholds for external beam radiation and comparable to that in adults for the approved dose.

About Lutathera
Lutathera (lutetium Lu 177 dotatate) is approved in the US for the treatment of adults and children 12 years and older with SSTR-positive GEP-NETs, including those in the foregut, midgut and hindgut, an indication which includes the populations studied in the randomized, controlled Phase III trials NETTER-1 and NETTER-2. Lutathera is also approved in Europe for unresectable or metastatic, progressive, well-differentiated (G1 and G2), SSTR-positive GEP-NETs in adults, and in Japan for SSTR-positive NETs

On April 23, 2024 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, OTCQB: DTCFF, FSE: DTC), a Canadian biopharmaceutical company developing novel immune-oncology therapeutics and drug delivery technologies, reported that its second-generation ARMTM anti-cancer vaccine using AccuTOX, called ARM-002TM, is therapeutically effective against pre-established melanoma when combined with the anti-PD-1 immune-checkpoint inhibitor (Press release, Defence Therapeutics, APR 23, 2024, View Source;utm_medium=rss&utm_campaign=defences-accutox-anti-cancer-arm-002-vaccine-exhibits-potent-antigen-presentation [SID1234642247]).

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Defence’s application of reprogrammed mesenchymal stromal cells ("MSCs") represents a leading vaccination platform due to its ease in manufacturing and the therapeutic potency that this allogeneic off-the-shelf vaccine can provide. The use of AccuTOX to reprogram these MSCs relies mainly on the induction of protein aggregation. This process is known to induce the activation of the unfolded protein response, a cellular defense mechanism normally triggered to destroy any captured protein aggregates due to the toxicity and disturbance it causes to cell integrity.

"Defence’s AccuTOX was initially known for its ability to kill cancer cells. In addition, our team found that at specific doses, AccuTOX forms protein aggregates when mixed with tumor lysate, a process that pushes MSCs to degrade these intracellular complexes resulting in potent antigen presentation." says Mr. Plouffe, Chief Executive Officer of Defence Therapeutics.

The original ARMTM vaccine was potent against melanoma. The ARM-002TM vaccine is even more potent, as it actually requires 10x less protein. This was confirmed both in vitro (using antigen cross-presentation assay) and in vivo where ARM-002TM pulsed with 0.05 mg/ml of tumor lysate resulted in similar outcomes compared to ARM-002TM generated using a dose of 0.5 mg/ml. In addition, the potency of the ARM-002TM vaccine was comparable in both male and female mice, with no noticeable side effects detected in vaccinated animals. Defence is currently testing the ARM-002TM vaccine on "hard-to-treat" cancers such as pancreatic, colon and ovarian cancers. These results will set the target indication for the Phase I trials, and it also shows how versatile and adaptable the ARM-002TM anti-cancer vaccine is.

On April 23, 2024 Curium, a world leader in nuclear medicine, reported that it has successfully enrolled and scanned patients in its SOLAR clinical trials (Press release, Curium, APR 23, 2024, View Source [SID1234642246]):

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The first trial is SOLAR-STAGE: a Phase 3, multicenter, open-label study to evaluate the diagnostic performance of copper Cu 64 PSMA I&T PET/CT in staging of men with newly diagnosed unfavorable intermediate-risk, high-risk or very-high-risk prostate cancer electing to undergo radical prostatectomy with pelvic lymph node dissection.
The second study, SOLAR-RECUR: a Phase 3, multicenter, open-label study to evaluate the diagnostic performance of copper Cu 64 PSMA I&T PET/CT in men with suspected biochemical recurrence of prostate cancer after radical prostatectomy or radiation therapy.
These clinical trials are currently enrolling patients at sites across the U.S. and will be opening clinical trial sites in Europe later this year.

Sakir Mutevelic, MD, Curium’s Chief Medical Officer said: "The successful start of patient enrollment is a significant step forward for Curium’s Phase 3 SOLAR-STAGE and SOLAR-RECUR clinical trials. As we continue Curium’s journey to redefine the experience of cancer through our trusted legacy in nuclear medicine, we look forward to building on our well-established and successful 64-Cu diagnostic imaging platform."

Amy Bartalotta, Vice President of Clinical Operations added: "To ensure that the SOLAR clinical trials recruit a diverse set of patients – including minority groups that are often more affected by and less likely to be screened for prostate cancer – we have 50 sites in the U.S. and over 30 sites in Europe participating in these trials. We are pleased to announce the first patients enrolled and scanned were at XCancer under the direction of Dr. Luke Nordquist. We look forward to additional trial sites scanning patients across the U.S. and Europe in the coming months."

For more information about the SOLAR-STAGE (NCT06235151) and SOLAR-RECUR (NCT06235099) studies visit www.solarclinicaltrials.com or contact Curium’s Clinical Trial team directly at [email protected] with questions or to locate a clinical trial site near you.