On September 10, 2022 Elevar Therapeutics, Inc., a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, reported top-line results from the Phase 3 study of its drug candidate rivoceranib combined with camrelizumab versus sorafenib as a first-line therapy for unresectable hepatocellular carcinoma (uHCC) (Press release, Elevar Therapeutics, SEP 10, 2022, View Source [SID1234619352]). Camrelizumab plus rivoceranib significantly prolonged overall survival (OS) and progression-free survival (PFS), and improved overall response rate (ORR) versus sorafenib, a standard first-line treatment for uHCC. The findings were presented today during the annual Congress of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in Paris.

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"Camrelizumab (C) plus rivoceranib (R) vs. sorafenib (S) as first-line therapy for unresectable hepatocellular carcinoma (uHCC): a randomized, phase 3 trial," was shared as a proffered paper presentation at ESMO (Free ESMO Whitepaper).

This global, randomized, open-label trial (NCT03764293) included 543 patients (ITT population), 17.3% of whom were non-Asian. The study began in June 2019, reaching its primary endpoint in April 2022. Top-line data included:

Median OS for camrelizumab+rivoceranib was 22.1 mos. [95% CI 19.1-27.2] vs. 15.2 mos. [13.0-18.5]; hazard ratio (HR) 0.62 [95% CI 0.49-0.80]; 1-sided p<0.0001,
Median PFS for camrelizumab+rivoceranib was 5.6 mos. [95% CI 5.5-6.3] vs. 3.7 mos. [2.8-3.7]; HR 0.52 [95% CI 0.41-0.65]); 1-sided p<0.0001, and
Confirmed ORR for camrelizumab+rivoceranib was 25.4% (95% CI 20.3-31.0), compared to 5.9% (3.4-9.4) for sorafenib.
"The efficacy results were generally consistent across all subgroups and with those reported in the overall ITT population, suggesting the combination of rivoceranib and camrelizumab confers a benefit in a global unresectable HCC population," said Saeho Chong, chief executive officer of Elevar.

In China, more than two-thirds of HCC cases are associated with hepatitis B virus (HBV) infection, and approximately 10% of cases are associated with hepatitis C virus (HCV). In the U.S., approximately 10% of HCC cases are estimated to be associated with HBV and 60% are estimated to be due to HCV (deMartel, 2015).

"In our uHCC trial, there was an overall consistent trend of prolonged PFS for patients in the camrelizumab plus rivoceranib arm compared to those in the sorafenib arm, irrespective of geographical region (Asian vs. non-Asian) or etiology (HBV vs. HCV vs. non-viral)," said Jan M. Van Tornout, M.D., MSc., chief medical officer of Elevar. "Specifically, the combination of camrelizumab and rivoceranib demonstrated efficacy among those with HCV-based etiology, which comprises the majority of U.S. HCC cases1."

The following data from the trial compares OS HR and PFS HR in Asian vs. non-Asian populations and in patients with different virus-based etiology:

OS HR in Asian 0.66 (95% CI 0.51, 0.86) vs. non-Asian 0.55 (0.29, 1.02),
OS HR in HBV 0.66 (0.5, 0.87) vs. HCV 0.45 (0.18, 1.16) vs. non-viral 0.71 (0.37, 1.36),
PFS HR in Asian 0.55 (95% CI 0.45, 0.70) vs. non-Asian 0.56 (0.30, 1.07), and
PFS HR in HBV 0.53 (0.41, 0.68) vs. HCV 0.56 (0.22, 1.45) vs. non-viral 0.65 (0.36, 1.20)
"HCC is the most common type of liver cancer, which despite advances in surgery, transplantation and approval of systemic therapies, still conveys a poor prognosis and survival rate," said Ahmed Omar Kaseb, M.D., professor, Department of Gastrointestinal Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston. "The combination of camrelizumab and rivoceranib shows potential to address what is clearly an unmet medical need for HCC patients globally."

Elevar also announced at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2022 that in its Phase 2 clinical trial (Study RM-202) of rivoceranib monotherapy in patients with progressive recurrent or metastatic adenoid cystic carcinoma (R/M ACC), rivoceranib demonstrated clinical effectiveness, as indicated by substantially reduced tumor progression during the six months after rivoceranib treatment compared to the tumor progression during the six months prior to rivoceranib treatment.

"Based on the positive results of the combination study, Elevar plans to work closely with the U.S. Food & Drug Administration for submission of a New Drug Application (NDA) for rivoceranib in combination with camrelizumab as a treatment option for HCC," said Chong. "In addition, Elevar anticipates submission of an NDA to the U.S. Food & Drug Administration for rivoceranib as a treatment option for ACC by the end of 2022."

1. Yoshizawa H. Hepatocellular carcinoma associated with hepatitis C virus infection in Japan: Projection to other countries in the foreseeable future. Oncology. 2002;62(Suppl 1):8–17. [PubMed: 11868791]; El-Serag HB, Rudolph KL. Hepatocellular carcinoma: Epidemiology and molecular carcinogenesis. Gastroenterology. 2007;132:2557–76. [PubMed: 17570226]

About Hepatocellular Carcinoma (HCC)

HCC is the most common type of primary liver cancer. It most frequently occurs in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection. HCC typically has a poor prognosis and a lack of treatment options and is therefore a condition with an urgent medical need.

About Camrelizumab

Camrelizumab (SHR-1210) is a humanized monoclonal antibody targeting the programmed death-1 (PD-1) receptor. Blockade of the PD-1/PD-L1 signaling pathway is a therapeutic strategy showing success in a wide variety of solid and hematological cancers. Camrelizumab is developed by Hengrui Pharma and has been studied in more than 5,000 patients. Currently, 50 clinical trials are underway in a broad range of tumors (including liver cancer, lung cancer, gastric cancer, and breast cancer et al.) and treatment settings.

Camrelizumab, under the brand name AiRuiKa, is currently approved for eight indications in China, including monotherapy for the treatment of HCC (second-line), relapsed/refractory classic Hodgkin’s lymphoma (third-line), esophageal squamous cell carcinoma (second-line) and nasopharyngeal carcinoma (third-line or further) and in combination with chemotherapy for the treatment of non-small cell lung cancer (non-squamous and squamous), esophageal squamous cell carcinoma and nasopharyngeal carcinoma in the first-line setting. The U.S. Food and Drug Administration granted Orphan Drug Designation to camrelizumab for advanced HCC in April 2021.

About Rivoceranib

Rivoceranib is the first small-molecule tyrosine kinase inhibitor (TKI) approved in gastric cancer in China (November 2014). Rivoceranib is a highly potent inhibitor of vascular endothelial growth factor receptor 2 (VEGFR-2), a primary pathway for tumor angiogenesis. VEGFR-2 inhibition is a clinically validated approach to limit tumor growth and disease progression. Rivoceranib is co-developed by Hengrui Pharma in China and by Elevar Therapeutics, Inc. globally (excluding China). It has been studied in more than 6,000 patients worldwide and was well tolerated in clinical trials with a comparable safety profile to other TKIs and VEGF inhibitors. Rivoceranib is currently being studied as a monotherapy and in combination with chemotherapy and immunotherapy in various solid tumor indications. Clinical studies are ongoing in multiple solid tumor types including gastric cancer (as a monotherapy and in combination with paclitaxel), hepatocellular carcinoma (HCC) (in combination with camrelizumab), adenoid cystic carcinoma (as monotherapy) and colorectal cancer (in combination with Lonsurf). Orphan drug designations have been granted in gastric cancer (U.S., EU and South Korea), in adenoid cystic carcinoma (U.S.) and in HCC (U.S.). Elevar holds the global rights (excluding China) and has partnered for the development and marketing of rivoceranib with HLB-LS in South Korea. Rivoceranib, under the name apatinib, is currently approved in China for advanced gastric cancer and in second-line advanced HCC by the Chinese-territory license-holder, Hengrui Pharma, under the brand name Aitan.

On September 10, 2022 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biogress 2022 (Press release, SpringWorks Thpharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that data from the Phase 3 DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, as a late-breaking oral presentation during a Presidential Symposium at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Conerapeutics, SEP 10, 2022, View Source [SID1234619351]).

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"Desmoid tumors can have a debilitating impact on patients’ lives and there is an urgent need for a new standard-of-care treatment," said Saqib Islam, Chief Executive Officer of SpringWorks. "DeFi is the largest and most robust Phase 3 trial conducted to date in patients with desmoid tumors and we believe these positive data bring us a step closer toward potentially introducing the first approved therapy for this underserved community. We look forward to completing our full NDA filing package by the end of the year, which will be submitted for review under FDA’s RTOR program."

The DeFi trial met its primary endpoint of improving progression-free survival (PFS), as assessed by blinded independent central review, demonstrating a statistically significant improvement for nirogacestat over placebo, with a 71% reduction in the risk of disease progression (hazard ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p< 0.001). The median Kaplan-Meier estimate of PFS was not reached in the nirogacestat arm and was 15.1 months in the placebo arm. A PFS benefit was observed across all prespecified subgroups, including gender, tumor location, prior treatment or surgery, and mutational status. Confirmed objective response rate (complete response + partial response) based on RECIST v1.1 was 41% with nirogacestat versus 8% with placebo (p<0.001). The complete response rate was 7% in the nirogacestat arm and 0% in the placebo arm. Nirogacestat demonstrated statistically significant and clinically meaningful improvements in patient-reported outcomes (PRO), which were key secondary endpoints of the study. Specifically, nirogacestat significantly reduced pain (p<0.001) and other DT-specific symptoms (p<0.001) and also significantly improved physical/role functioning (p<0.001) and overall health-related quality of life (p=0.007). Most PRO benefits were observed as early as Cycle 2, which was the first timepoint for post-treatment evaluation, and were sustained over the duration of the study.

At the time of primary analysis (April 7, 2022), the median duration of treatment was 20.6 months for participants on nirogacestat and 11.4 months for those on placebo, with the majority of nirogacestat patients continuing on treatment. Nirogacestat exhibited a manageable safety profile in the DeFi trial, with 95% of all treatment-emergent adverse events (TEAEs) reported as Grade 1 or 2. The most frequently reported TEAEs in participants receiving nirogacestat as compared to the placebo arm were diarrhea (84% vs 35%), nausea (54% vs 39%), and fatigue (51% vs 36%). Forty-two percent of patients in the nirogacestat arm vs 0% in the placebo arm required dose reductions due to TEAEs and 20% of patients in the nirogacestat arm vs 1% in the placebo arm discontinued treatment due to TEAEs. Ovarian dysfunction, which was defined by investigator-reported events of amenorrhea, premature menopause, menopause, and ovarian failure, was observed in 75% (27/36) of women of childbearing potential receiving nirogacestat. These events resolved in 74% (20/27) of the affected participants, including 64% (9/14) of such participants who remained on nirogacestat treatment and 100% (11/11) of those participants who discontinued treatment for any reason.

"The DeFi study enrolled patients with progressing desmoid tumors at baseline and included a high proportion of patients with multifocal disease and uncontrolled pain, representing a very difficult-to-treat patient population," said Bernd Kasper, M.D., Ph.D., University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the DeFi trial. "The consistently positive data generated across progression-free survival, objective response rate, and patient-reported outcomes, coupled with a safety profile that is suitable for long-term dosing, support the potential for nirogacestat to become an important and much needed treatment for patients with desmoid tumors."

ESMO Oral Presentation Details

Title: DeFi: A Phase 3, Randomized Controlled Trial of Nirogacestat Versus Placebo for Progressing Desmoid Tumors (DT)
Presentation Number: LBA2
Presenter: Bernd Kasper, M.D., Ph.D., University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany
Session/Type: Presidential Symposium 1, Proffered Paper Session
Date: Saturday, September 10, 2022
Time: 4:55-5:10 p.m. CEST (10:55-11:10 a.m. ET)

Investor Event Details

Presenters: SpringWorks’ management team will be joined by Bernd Kasper, M.D., Ph.D., University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the DeFi trial.
Date: Sunday, September 11, 2022
Time: 4:00 p.m. CEST (10:00 a.m. ET)
Instructions: To join the live webcast and view corresponding slides, please visit the Events & Presentations page within the Investors & Media section of the Company’s website at View Source To join via audio teleconference, please register here. Once registration is complete, participants will be provided with a dial-in number and conference code to access the call. A replay will be available on the Company’s website for a limited time following the event.

About the DeFi Trial

DeFi (NCT03785964) is a global, randomized (1:1), double-blind, placebo-controlled Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The double-blind phase of the study randomized 142 patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by >20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to the first dose of study treatment. The primary endpoint is progression-free survival, as assessed by blinded independent central review. Secondary and exploratory endpoints include safety and tolerability measures, objective response rate (ORR), duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes (PROs). DeFi includes an open label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors are rare, aggressive, locally invasive, and potentially morbid tumors of the soft tissues.1,2 While they do not metastasize, desmoid tumors are associated with a high rate of recurrence.2,3,4 Sometimes referred to as aggressive fibromatosis, or desmoid fibromatosis, these soft tissue tumors can be serious, debilitating, and, in rare cases when vital structures are impacted, they can be life-threatening.2,5

Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years, with a two-to-three times higher prevalence in females.4,6,7,8 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.7,8,9

Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.1,4,10 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

About Nirogacestat

Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

In addition, gamma secretase has been shown to directly cleave membrane-bound B cell maturation antigen (BCMA), resulting in the release of the BCMA extracellular domain (ECD) from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat’s ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has eight collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies, three bispecific antibodies and a monoclonal antibody. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA-directed therapies using a variety of preclinical multiple myeloma models.

Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. SpringWorks plans to submit a New Drug Application (NDA) to the FDA in the second half of 2022, which will be submitted for review under the FDA’s Real-Time Oncology Review (RTOR) program.

On September 10, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported positive initial data from the single agent dose escalation portion of the Phase 1 study of DCC-3116, the Company’s first-in-class, potent, and selective small molecule switch-control kinase inhibitor of ULK1/2, in patients with advanced or metastatic tumors with a mutant RAS or RAF gene (Press release, Deciphera Pharmaceuticals, SEP 10, 2022, View Source [SID1234619350]). Results from the study were presented in an oral presentation as a Proffered Paper titled "Initial monotherapy results of a phase 1 first‑in‑human study of ULK1/2 inhibitor DCC‑3116 alone and in combination with MAPK pathway inhibition" at the ESMO (Free ESMO Whitepaper) Congress 2022.

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"We are excited to report first-in-human DCC-3116 clinical data demonstrating a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested," said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. "As the first ULK1/2 inhibitor to enter clinical development, these positive initial results represent a significant milestone as we prepare to initiate combination dose escalation later this year. With a novel mechanism of action and strong preclinical data demonstrating compelling anti-tumor activity in combination with a broad array of RTK, RAS, and other MAP kinase pathway inhibitors, we believe DCC-3116 has the potential to open a new frontier in the treatment of cancer."

Anthony Tolcher, M.D., FRCPC, Co-Founder and Director of Clinical Research, NEXT Oncology said, "The initial DCC-3116 monotherapy results reported today are very encouraging and strongly support the advancement of DCC-3116 into the combination setting. The preliminary data show DCC-3116 to be a very well-tolerated agent that has demonstrated strong target inhibition of ULK 1/2 from even the lowest tested dose. I look forward to the selection of the combination starting dose and advancing the program into the first combination studies with MEK and KRASG12C inhibitors."

Summary of Data and Findings

As of June 9, 2022, 18 patients with locally advanced or metastatic cancer with a RAF or RAS mutation were enrolled across four cohorts dosed with DCC-3116 twice daily (BID): 50 mg BID (n=3); 100 mg BID (n=4); 200 mg BID (n=7); and 300 mg BID (n=4). The median number of prior anti-cancer regimens was three (range 1-10). The most common cancer types were colorectal (56%) and pancreatic (28%) and patients had KRAS (83%) and BRAF (17%) mutations.

The results of the primary objectives of safety and tolerability as well as the additional objectives of pharmacokinetics, pharmacodynamics, and anti-tumor activity are summarized below:

Safety and Tolerability:

Treatment with DCC-3116 was well tolerated and most treatment-emergent adverse events (TEAEs) were Grade 1/2; the most common (≥15%) TEAEs regardless of relatedness reported (all grades) were: fatigue (39%), dehydration (22%), alanine transaminase (ALT) increases (17%), anemia (17%), aspartate transaminase (AST) increases (17%), decreased appetite (17%), hyponatremia (17%), nausea (17%), and vomiting (17%).
No dose-limiting toxicities or treatment-related serious adverse events were observed with DCC-3116; two asymptomatic, reversible Grade 3 alanine transaminase increases that led to dose interruption and reduction were reported as treatment-related.
Pharmacokinetics, Pharmacodynamics and Anti-Tumor Activity:

DCC-3116 exposure appeared to increase dose-proportionally across the four dose levels tested from 50 mg BID to 300 mg BID; at all doses levels, the area under the curve (AUC) of DCC-3116 was at or above the AUC of the lowest tested dose that was effective in preclinical studies.
DCC-3116 demonstrated target inhibition with significant decreases in phosphorylation of ATG14, a direct ULK1/2 substrate, in peripheral blood mononuclear cells; at all dose levels, reductions in phosphorylated ATG14 were observed that were associated with anti-tumor activity in preclinical studies combining DCC-3116 and a MEK inhibitor as measured by reductions in phosphorylated ATG13 in tumors.
Fourteen patients were evaluable for response per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 as of the cutoff date; best overall response was stable disease and the disease control rate at week 16 was 29%.
Dose cohorts 100 to 300 mg BID are being expanded to further characterize the safety, pharmacokinetics, and pharmacodynamics of DCC-3116. In the fourth quarter of 2022, we expect to select the starting dose of DCC-3116 for, and initiate, dose escalation cohorts in combination with MEK and KRASG12C inhibitors.

Conference Call and Webcast
Deciphera will host a conference call and webcast to discuss data presentations from the Company’s DCC-3116 and vimseltinib clinical programs at the ESMO (Free ESMO Whitepaper) Congress 2022 on Sunday, September 11, 2022, at 7:30 AM ET/ 1:30 PM CEST. The event may be accessed by registering at View Source A webcast of the event will be available in the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source The archived webcast will be available on the Company’s website within 24 hours after the event and will be available for 30 days following the event.

About DCC-3116
DCC-3116 is an investigational, orally administered, potent, and highly selective switch-control inhibitor designed to inhibit cancer autophagy, a key tumor survival mechanism in cancer cells, by inhibiting the ULK1/2 kinases, which have been shown to be the enzymes responsible for initiating autophagy. DCC-3116 is currently being studied in a Phase 1/2, multicenter, open-label, first-in-human study as a single agent and in combination with RAS/MAPK pathway inhibitors in patients with advanced or metastatic solid tumors with a RAS/MAPK pathway mutation (NCT04892017).

On September 9, 2022 Heidelberg Pharma AG (FSE: HPHA) reported that its subsidiary Heidelberg Pharma Research GmbH and Takeda signed a license agreement granting Takeda an exclusive license to commercially develop an Antibody Targeted Amanitin Conjugate directed to a previously selected target molecule (Press release, Heidelberg Pharma, SEP 9, 2022, View Source [SID1234635111]).

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Takeda obtained access to Heidelberg Pharma’s proprietary Amanitin toxin-linker platform technology under an exclusive multi-target research agreement effective as of June 2017. Takeda provided different antibodies to Heidelberg Pharma to generate new ATACs. ATACs are ADCs (Antibody Drug Conjugates) based on Heidelberg Pharma’s ATAC technology.

On September 9, 2022 Radiopharm Theranostics (ASX:RAD, "Radiopharm" or the "Company"), a developer of a world-class platform of radiopharmaceutical products for both diagnostic and therapeutic uses, reported that the US Food & Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for its DUNP19 technology for the treatment of osteosarcoma (Press release, Radiopharm Theranostics, SEP 9, 2022, View Source [SID1234619598]).

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The RPD program is aimed at advancing development of drugs with the potential to treat serious, rare pediatric diseases.

RPD allows companies to receive a priority review voucher (PRV) from the FDA at the time a marketing authorization is granted. A PRV can be used by the Company to expedite approval, or can be transferred/sold to other companies for use in the same manner. The price of two recent examples of PRVs sales have ranged from US$105,0001 to US$110,0002.

Radiopharm signed an exclusive licensing agreement with University of California Los Angeles (UCLA) Technology Development Group (UCLA-TDG) for the promising LRRC15 antibody "DUNP19" in April 2022.

LRCC15 expression is produced by cancer cells and the surrounding tumour microenvironment, but not by healthy normal tissues, and LRRC15 production is very high in aggressive and treatmentresistant tumours.

While currently available antibodies for cancer treatment omit tumour micro-environment (TME) cells, such as stromal and immune cells, which comprise >50% of tumour masses, the DUNP19 antibody has a unique ability to effectively find, internalize and destroy both cancer-, and TME cells. DUNP19 is a first-in-class therapy thanks to its unique dual action tumour targeting and to its fast internalization.

Osteosarcoma is a type of bone cancer that primarily affects children, adolescents and young adults, with surgery and chemotherapy the only currently available treatments. As aggressive osteosarcoma has one of the highest expressions of LRRC15, it’s an ideal candidate for proof-of-concept testing.

Riccardo Canevari, CEO and Managing Director of RAD, said: "This is again excellent recognition of the work to date by Dr David Ulmert and his team and the potential for DUNP19 to make a significant difference to young patients in need. The RPD and associated PRV can be incredibly valuable and we look forward to progressing the DUNP19 program and eventually taking advantage of this."