On June 11, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that positive results from the Company’s ongoing Phase 1 COBALT-LYM trial evaluating the safety and efficacy of CTX130, its wholly-owned allogeneic CAR-T cell therapy targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies (Press release, CRISPR Therapeutics, JUN 11, 2022, View Source [SID1234615903]).

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"We are very pleased with the preliminary results from our COBALT-LYM trial, which showed efficacy and safety that suggest that CTX130, the first allogeneic CAR-T directed against the novel target CD70, can produce deep responses in patients with relapsed or refractory T cell lymphomas," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Additionally, we may be able to further optimize the profile by continuing our consolidation dosing strategy. These data reinforce our belief that engineered cell therapies are the future in our fight against cancer and we are well-positioned to be leaders in this field."

"While overall survival in a subset of patients with T cell lymphoma has improved with front-line combination chemotherapy, relapsed or refractory patients continue to have very limited treatment options," said Swaminathan P. Iyer, M.D., Professor, Lead of the T Cell Lymphoma Program, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. "The data from the CTX130 trial demonstrate the potential of cell therapies as a new treatment modality for these patients. I am particularly encouraged by the response rates and safety data, which suggest that treatment with CTX130 could elicit clinically meaningful responses, including complete responses, in patients with difficult-to-treat T cell lymphomas."

COBALT-LYM Trial Overview
The Phase 1 COBALT-LYM trial is an open-label, multicenter clinical trial evaluating the safety and efficacy of CTX130 in adult patients with relapsed or refractory T or B cell malignancies. Dose escalation of CTX130 was performed in adult patients with relapsed or refractory T cell lymphoma, with at least 10% expression of CD70. Patients who received prior treatment with any CD70 targeting agents were not eligible.

Patients received three days of lymphodepleting chemotherapy, consisting of fludarabine at 30 mg/m2/day and cyclophosphamide at 500 mg/m2/day, followed by a single CTX130 infusion. Patients completed screening in as few as five days, and the median time from enrollment to the start of lymphodepleting chemotherapy was only three days. This timeline was possible because there is no need for leukapheresis or bridging chemotherapy, and CTX130 is available at the site before a patient is enrolled. Additionally, patients who showed clinical benefit from the first CTX130 infusion could be re-dosed following disease progression.

As of the April 26, 2022, data cutoff, 19 patients with T cell malignancies had been enrolled, of which 18 patients had received CTX130 with at least 28 days of follow-up and are included in the analysis. Prior to enrollment, all patients were heavily pre-treated, with a median of four systemic therapies. Additionally, all patients were refractory to their last line of therapy. Eight patients had peripheral T-cell lymphoma (PTCL) and 10 patients had cutaneous T-cell lymphoma (CTCL).

The primary endpoints include safety as measured by the incidence of dose limiting toxicities (DLTs) and overall response rate (ORR). Key secondary endpoints include progression free survival (PFS) and overall survival (OS).

Safety
CTX130 was well tolerated across all dose levels. The adverse events of interest for all evaluable patients are shown in the table below.

There were no cases of Graft versus Host Disease (GvHD); no dose limiting toxicities (DLTs); and no instances of tumor lysis syndrome (TLS).

All cases of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were Grade 1 or 2 per the American Society for Transplantation and Cellular Therapy (ASTCT) criteria and either required no specific intervention or resolved following standard CRS management. Neither the frequency nor severity of CRS has increased in patients who were re-dosed with CTX130.

There was a sudden death in one patient with William’s syndrome in the context of a lung infection, deemed unrelated to CTX130. There were no treatment related deaths in the trial. Overall, CTX130 has an emerging safety profile that is well tolerated.

Clinical Activity
Deep responses were seen with CTX130 in a significant fraction of patients at DL3 and above. Data are shown below for the 18 patients who received CTX130 and had at least 28 days of follow-up. Disease assessment was performed by investigator review according to the 2014 Lugano Response Criteria for PTCL or the International Society for Cutaneous Lymphoma Response Criteria (Olsen criteria) for CTCL, as appropriate.

Patients were heavily pre-treated with a median of four systemic therapies prior to enrollment in the study. None of the 18 patients had achieved a complete response (CR) in their previous line of therapy.

Median CD70 expression among the patients was 90%. Responses were observed across all levels of CD70 expression.

Clinically meaningful responses were observed with CTX130 with a higher percentage of patients responding at higher dose levels. At DL3 and above, ORR was 70% with 30% of patients achieving a CR. In addition, 90% of patients at DL3+ had clinical benefit defined as a stable disease or better response. These responses were largely consistent in both PTCL and CTCL with ORRs of 80% and 60%, respectively, at DL3+.

Broad activity and deep responses were seen in all disease compartments including lymph nodes, skin and blood in patients with CTCL following treatment with CTX130.
These preliminary data demonstrate that CTX130 has the potential to provide meaningful clinical benefit with a well-tolerated safety profile. Given the inherent difficulties and potential risks of manufacturing a CAR-T therapy from a patient’s own diseased T cells, allogeneic cellular therapy approaches for T cell lymphoma have greater potential to address the unmet need in this patient population.

CTX130 is currently being investigated in two ongoing Phase 1 clinical trials for the treatment of various subtypes of lymphoma (COBALT-LYM) or relapsed or refractory renal cell carcinoma (COBALT-RCC), respectively. Additional details on COBALT-LYM may be found at clinicaltrials.gov, using identifier: NCT04502446. In parallel, the Company continues to advance the rest of its immuno-oncology portfolio.

The Company plans to recap this data during the CRISPR Therapeutics Innovation Day, an event focused on early research and development, on June 21, 2022, at 2:00 pm ET.

Innovation Day Webcast
A live webcast of the event will be available on the "Events & Presentations" page in the Investors section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 30 days following the presentation. Please contact [email protected] for any questions regarding the event.

About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively. CTX130 for the treatment of T-cell lymphoma has received Orphan Drug Designation from the FDA.

About COBALT-LYM
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, COBALT-LYM, is designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory T- or B-cell malignancies.

About COBALT-RCC
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, COBALT-RCC, is designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory Renal Cell Carcinoma.

On June 11, 2022 EORTC reported that Acute myeloid leukemia (AML) predominantly occurs in older adults over 65 years, who exhibit a lower tolerance to conventional induction chemotherapy (IC) compared with younger patients (Press release, EORTC, JUN 11, 2022, View Source [SID1234615902]). Despite treatment with IC, older patients with AML have poor long-term survival without hematopoietic stem cell transplantation (HSCT). For the past decade, DNA-hypomethylating agents such as decitabine have been considered a safer alternative for patients who are unfit for IC. Prolonged (10-day) decitabine treatment has shown promising efficacy in previous studies with older patients with AML, and may be a more suitable treatment before HSCT than IC in fit patients.

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The open-label randomized phase III study of the EORTC Leukemia group, GIMEMA, CELG, and GMDSSG (NCT02172872) compared the efficacy and safety of 10-day decitabine with conventional 3+7 IC as a treatment before HSCT in older (≥60 years) patients with newly diagnosed AML. Patients with at least stable disease and an HLA-matched donor were encouraged to undergo HSCT after ≥1 treatment cycle.

The rate of HSCT in decitabine-treated patients was similar to IC-treated patients (40% vs 39%). Although IC achieved higher complete remission rates compared with decitabine (61% vs 48%), the overall survival was comparable, with a median overall survival of 15 months in the decitabine group and 18 months in the IC group. At 4 years, 26% of patients from the decitabine arm and 30% of patients
from the IC arm were alive. A notable difference between the treatment arms was the incidence of grade 3–5 adverse events before HSCT. Decitabine treatment showed lower incidences of febrile neutropenia, platelet reduction, oral mucositis, and diarrhea. In addition, the 30-day mortality rate was 3.6% for decitabine compared with 6.4% for IC.

In conclusion, decitabine treatment for older patients with AML exhibited a superior safety profile compared with IC while maintaining similar overall survival and rates of HSCT. The results of this study will be presented by Prof. Michael Lübbert on Saturday, June 11.

Presenter: Prof Dr med Michael Lübbert
Affiliation: Universitätsklinikum Freiburg, Freiburg, Germany
Presentation: S125 will be presented by Prof Michael Lübbert | Saturday, June 11, 2022 | 16:30 –17:45 CEST | Hall A7, Messe Wien Exhibition & Congress Center, Vienna, Austria.

About the EHA (Free EHA Whitepaper) Annual Congress

Every June, EHA (Free EHA Whitepaper) organizes its Annual Congress in a major European city. In 2020 and 2021, the format was virtual due to COVID-19. However, this year, EHA (Free EHA Whitepaper) has organized a Hybrid congress for the first time. The Congress is aimed at health professionals working in or interested in the field of hematology. The scientific program topics range from stem cell physiology and development to leukemia, lymphoma, diagnosis and treatment, red blood cells, white blood cells and platelet disorders, hemophilia and myeloma, thrombosis and bleeding disorders, as well as transfusion and stem cell transplantation.

On June 11, 2022 AbbVie (NYSE: ABBV) reported primary results from the large B-cell lymphoma (LBCL) expansion cohort in the EPCORE NHL-1 phase 2 clinical trial evaluating epcoritamab (DuoBody-CD3xCD20), an investigational subcutaneous bispecific antibody (Press release, AbbVie, JUN 11, 2022, View Source [SID1234615901]). In this study, epcoritamab demonstrated efficacy with durable responses in patients who had previously received at least two prior lines of anti-lymphoma therapy including chimeric antigen receptor (CAR) T-cell therapy. These data were presented today in a late-breaking oral presentation as a part of the Presidential Symposium at the 27th Annual Meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2022) in Vienna, Austria (Abstract #LB2364).

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"Large B-cell lymphoma is a fast-growing, difficult to treat type of aggressive non-Hodgkin’s lymphoma. Some treatment approaches like chemotherapy and immunotherapy have been in place for decades and newer treatments like CAR T-cell therapies involve multiple steps before a patient can begin treatment so there is still a need for additional treatment options," said Professor Catherine Thieblemont, head of the Hemato-Oncology Department at Hôpital Saint-Louis, Paris, France. "The data presented today suggest that epcoritamab has the potential to provide patients living with LBCL an accessible, effective treatment with a safety profile that may fulfill an unmet need."

The study cohort, which included 157 relapsed/refractory LBCL patients, previously treated with a median of three lines of prior therapy, demonstrated an overall response rate (ORR) of 63 percent and a complete response (CR) rate of 39 percent. Baseline characteristics included 61 percent of patients who were refractory to primary treatment, 20 percent who had prior autologous stem cell transplantation (ASCT), and 39 percent who were treated with CAR T-cell therapy (75 percent of those refractory to CAR T). Patients enrolled in the study who were naïve to CAR T-cell therapy achieved a 69 percent ORR and a 42 percent CR and patients who received prior CAR T-cell therapy achieved a 54 percent ORR and a 34 percent CR. After a median follow up of 10.7 months, the median duration of response (mDOR) was estimated to be 12 months, while the mDOR among patients achieving a CR was not reached, with 89 percent still in CR at nine months. Topline results from this study were previously announced in April 2022.

The safety profile of epcoritamab was consistent with previous findings. The majority of treatment-emergent AEs (TEAEs) occurred during the first 12 weeks of treatment and resolved. The most common TEAEs of any grade (greater than or equal to 15 percent) included cytokine release syndrome (CRS) (49.7 percent), pyrexia (23.6 percent), fatigue (22.9 percent), neutropenia (21.7 percent), diarrhea (20.4 percent), injection site reaction (19.7 percent), nausea (19.7 percent) and anemia (17.8 percent). The most common Grade 3 or 4 TEAEs (greater than or equal to 5 percent) included neutropenia (14.6 percent), anemia (10.2 percent), neutrophil count decrease (6.4 percent), and thrombocytopenia (5.7 percent). The observed Grade 3 CRS was 2.5 percent. No Grade 4/5 CRS was observed.

"The epcoritamab data suggests a potentially compelling clinical profile for patients with relapsed/refractory LBCL, which currently have limited treatment options," said Mohamed Zaki, M.D., Ph.D., vice president and head, global oncology development, AbbVie. "Our partnership with Genmab allows us to continue exploring new standards of care for patients with blood cancer."

Epcoritamab is being co-developed by AbbVie and Genmab as part of the companies’ broad oncology collaboration. The companies remain committed to evaluating epcoritamab as a monotherapy, and in combination, across lines of therapy for a variety of hematologic malignancies, including an ongoing phase 3, open-label, randomized trial evaluating epcoritamab as a monotherapy in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (NCT: 04628494).

About Large B-cell Lymphoma (LBCL)
LBCL is a fast-growing type of non-Hodgkin’s lymphoma (NHL) – a cancer that develops in the lymphatic system – that affects B-cell lymphocytes, a type of white blood cell. There are an estimated 150,000 new LBCL cases each year globally. LBCL includes DLBCL, which is the most common type of NHL worldwide and accounts for approximately 31 percent of all NHL cases.1,2,3,4

About the EPCORE NHL-1 Trial
EPCORE NHL-1 an open-label, multi-center safety and preliminary efficacy trial of epcoritamab including a phase 1 first-in-human, dose escalation part; a phase 2 expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-NHL, including LBCL and DLBCL, the most common subtype of LBCL. The dose escalation findings, which determined the recommended phase 2 dose, were published in The Lancet in 2021. In the phase 2 expansion part, additional patients are treated with epcoritamab to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed/refractory B-NHLs who had limited therapeutic options.

The primary endpoint of the phase 2 expansion part was ORR as assessed by an IRC. Secondary efficacy endpoints included duration of response, complete response rate, progression-free survival, and time to response as determined by the Lugano criteria. Overall survival, time to next therapy, and rate of minimal residual disease negativity were evaluated as secondary efficacy endpoints.

About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response towards target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T cell mediated killing of CD20+ cells.5 CD20 is expressed on B-cells and a clinically validated therapeutic target in many B-cell malignancies, including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma and chronic lymphocytic leukemia.6,7 Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ broad oncology collaboration.

About AbbVie in Oncology
At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world’s most widespread and debilitating cancers. As we work to have a remarkable impact on people’s lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit View Source

On June 10, 2022 Zai Lab Limited, a patient-focused, innovative, commercial-stage, global biopharmaceutical company, reported that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted two Breakthrough Therapy Designations for investigational repotrectinib for the treatment of patients with ROS1-positive metastatic NSCLC who have received one prior line of ROS1 TKI and one prior line of platinum-based chemotherapy (EXP-2) and for those with ROS1-positive metastatic NSCLC who have received one prior line of ROS1 TKI and no chemotherapy or immunotherapy (EXP-4) (Press release, Zai Laboratory, JUN 10, 2022, View Source [SID1234633496]). The Breakthrough Therapy Designations for repotrectinib were supported by the data from both global and Chinese TKI-pretreated ROS1-positive NSCLC patients enrolled in the Phase 1/2 TRIDENT-1 study.

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"Since repotrectinib received Breakthrough Therapy Designation by the CDE earlier this year for ROS1-positive TKI-naïve patients, today’s recognition further supports repotrectinib as a potential best-in-class treatment for ROS1-positive NSCLC in both TKI-naïve and pretreated patients in China," said Alan Sandler, M.D., President and Head of Global Development, Oncology at Zai Lab. "There remain significant unmet needs for ROS1-positive NSCLC patients, and we look forward to our continued partnership with regulatory authorities in China to bring this important medicine to patients in need as soon as possible."

The Breakthrough Therapy Designation review policy is designed to facilitate the development and expeditious review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or diseases that severely impact the quality of life for which there is no existing treatment, or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs granted Breakthrough Therapy Designations receive priority communications and guidance from the CDE to promote and expedite the drug development process.

Lung cancer is both the most commonly diagnosed cancer type and the leading cause of cancer death in China. The incidence of lung cancer in China in 2020 was 815,563 cases, with 714,699 deaths1. NSCLC accounts for approximately 85% of lung cancer, and about 70% of NSCLC is locally advanced or metastatic at initial diagnosis. In China, ROS1 rearrangements occur in 2-3% of patients with advanced NSCLC.

1Globocan 2020.

About Repotrectinib

Repotrectinib is a next-generation kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of NSCLC and advanced solid tumors. Tumors with mutations to their ROS1 and NTRK genes have a higher likelihood of developing resistance to existing targeted therapies. In many cases, these mutations prevent existing medicine from targeting and binding to the tumor as effectively as tumors that do not carry the mutations. Repotrectinib is designed to be smaller and less bulky than existing targeted therapies and may circumvent some of the resistance mechanisms found in tumors with ROS1 and NTRK mutations. Zai Lab and Turning Point Therapeutics, Inc. are studying repotrectinib in TRIDENT-1, a registrational Phase 1/2 study in adults, and CARE, a Phase 1/2 study in pediatric patients. The compound has shown antitumor activity and durable responses among kinase inhibitor treatment-naïve and pre-treated patients. Zai Lab is enrolling patients in the registrational Phase 2 portion of TRIDENT-1 in Greater China, while Turning Point Therapeutics is enrolling patients in other regions of the world.

Repotrectinib has been granted three Breakthrough Therapy Designations from the U.S. Food and Drug Administration in: ROS1-positive metastatic NSCLC patients who have not been treated with a ROS1 TKI; patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK TKIs, with or without prior chemotherapy, and have no satisfactory alternative treatments; and ROS1-positive metastatic NSCLC patients who have previously been treated with a ROS1 TKI and who have not received prior platinum-based chemotherapy. Additionally, repotrectinib was previously granted four Fast-Track designations in: ROS1-positive advanced NSCLC patients who are ROS1 TKI naïve; ROS1-positive advanced NSCLC patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior ROS1 TKI; ROS1-positive advanced NSCLC patients pretreated with one prior ROS1 TKI without prior platinum-based chemotherapy; and NTRK-positive patients with advanced solid tumors who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments. Repotrectinib was also granted an Orphan Drug designation in 2017.

Zai Lab has an exclusive license agreement with Turning Point Therapeutics to develop and commercialize repotrectinib in Greater China.

On June 10, 2022 Ratio Therapeutics Inc. reported its launch with a mission to develop best-in-class targeted radiopharmaceuticals for the treatment of cancers (Press release, Ratio Therapeutics, JUN 10, 2022, View Source [SID1234633188]). Founded by entrepreneurial scientists Jack Hoppin, Ph.D., and John Babich, Ph.D., Ratio emerges from stealth mode with more than $20 million in seed funding, fully funded development alliances with Bayer AG and Lantheus Holdings Inc., a robust portfolio of assets developed with two proprietary technologies, and a growing team of world-class experts in radiopharmaceuticals discovery and development. The company’s near-term plans call for the submission of its first investigational new drug (IND) applications, which are expected this quarter, and the initiation of clinical trials later this year. Based in Boston, Ratio is set to move to a new 19,000-square-foot headquarters and research facility in the Seaport District in January.

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Ratio’s radiopharmaceuticals strategy is focused on optimizing tumor localization while minimizing uptake by normal tissues. To achieve this, the company uses its proprietary technology platform called Trillium that is based on Dr. Babich’s prior research at Weill-Cornell Medical College and first developed and validated by the founders in a previous venture. Trillium is a trifunctional small molecule scaffold that can be fine-tuned to alter its plasma clearance, target affinity and therapeutic payload. Each component of the scaffolding can be independently optimized to boost tumor uptake over normal tissue uptake, thereby maximizing therapeutic index. Ratio has successfully applied this framework to several tumor targets and multiple therapeutic payloads.

In addition, Ratio is developing a technology platform to take advantage of the tumor killing power of the alpha emitter, Actinium-225. This proprietary technology is called the Macropa chelate platform. Ratio’s scientists have already successfully incorporated Macropa into the Trillium platform as well as several peptides and antibodies. Macropa’s unique chemistry enables ease of manufacture and robust in vitro and in vivo stability of the resulting radiotherapeutic compound.

"The ability to fine-tune our targeted radiotherapeutics using Trillium and Macropa enables us to address head-on the trifecta of typical challenges we see with most radiopharmaceuticals: delivery, safety and efficacy," said Dr. Babich, Ratio’s President and Chief Scientific Officer. "Over the past year, we have generated significant preclinical data that demonstrate our ability to create excellent performing drug candidates that now are advancing into the clinic. Our goal is to become the partner of choice for pharmaceutical companies committed to this area of cancer therapy by enabling the optimization of a broad array of targeting compounds. We will shepherd these therapies through early clinical studies on our own or in collaboration."

Dr. Hoppin, Ratio’s Chairman and Chief Executive Officer, added, "Targeted radiotherapy is an exciting and emerging field where chemistry meets physics meets medicine. We have assembled and will continue to build a world-class interdisciplinary team of researchers and developers with a singular focus on delivering these treatments to cancer patients. It isn’t often that a start-up company has in place the early financial backing and industry support to advance entirely new drug discoveries to clinical development at this pace. It is with great pride that we announce our formal launch and exit from stealth mode."

In collaboration with Bayer, Ratio has leveraged its Trillium platform for the identification of lead prostate-specific membrane antigen (PSMA)-targeted therapeutic compounds for prostate cancer. At the same time, Ratio is working with Lantheus to develop a lead fibroblast activation protein (FAP)-targeted PET diagnostic compound for a broad array of epithelial-derived cancers, such as breast, pancreatic, lung and stomach cancer. Both collaborations are fully funded and reflect the types of partnerships that Ratio is currently pursuing with other companies.