On April 29, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the first quarter ended March 31, 2019 (Press release, Mirati, APR 29, 2019, View Source [SID1234535450]).

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First Quarter Highlights

January 7th, we announced a clinical collaboration with Bristol-Myers Squibb Company to evaluate the combination of sitravatinib and nivolumab (OPDIVO), in our planned Phase 3 clinical trial in second line NSCLC patients who have progressed following prior treatment. Mirati will sponsor and fund the clinical trial and Bristol-Myers Squibb will provide nivolumab at no cost.
January 15th, we announced that we had dosed the first patient in the Phase 1/2 clinical trial of MRTX849, a novel KRAS G12C inhibitor.
January 22nd, we announced the successful completion of a public offering of common stock that provided net cash proceeds of $107.9 million.
February 19th, we announced the appointment of Faheem Hasnain as Chairman of the Board of Directors.
"In the first quarter we made significant advances across our pipeline, including the dosing of the first patient with our KRAS G12C inhibitor, MRTX849. We also strengthened our operational and strategic capabilities, highlighted by a successful public offering and the appointment of Faheem Hasnain as Chairman of the Board," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer. "Our clinical programs continue to advance, and we are on track for a second quarter initiation of the Phase 3 randomized trial of sitravatinib plus nivolumab, versus docetaxel, in second line non-small cell lung cancer (NSCLC). The Phase 1/2 clinical trial of our KRAS G12C inhibitor, MRTX849, is ongoing and enrollment is proceeding well. We will provide an initial clinical update on MRTX849 in the second half of 2019."

Financial Results for the First Quarter 2019

Cash, cash equivalents, and short-term investments were $301.0 million at March 31, 2019, compared to $222.8 million at December 31, 2018. In January 2019, we completed a public offering of common stock that provided net cash proceeds of $107.9 million.

License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd. ("BeiGene"), dated January 7, 2018. License and collaboration revenues for the first quarter of 2019 were $1.2 million and relate to revenues earned in connection with a manufacturing supply services agreement with BeiGene. License and collaboration revenues for the first quarter of 2018 were $9.5 million and relate to the license the Company granted to BeiGene under the Collaboration and License Agreement.

Research and development expenses for the first quarter of 2019 were $34.2 million, compared to $19.7 million for the same period in 2018. The increase in research and development expenses is due to an increase in expense associated with sitravatinib and MRTX849, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The increase in sitravatinib expense is due to increased costs to support the expansion of existing and new clinical trials, and the increase in MRTX849 expense relates to the Phase 1 clinical trial, which was initiated in the first quarter of 2019. The Company recognized research and development-related share-based compensation expense of $5.2 million during the first quarter of 2019, compared to $1.5 million for the same period in 2018.

General and administrative expenses for the first quarter of 2019 were $9.8 million, compared to $5.2 million for the same period in 2018. The increase is primarily due to an increase in share-based compensation expense due to an increase in the fair value of stock options granted during the three months ended March 31, 2019 compared to the same period in 2018. The Company recognized general and administrative-related share-based compensation expense of $5.9 million during the first quarter of 2019, compared to $2.2 million for the same period in 2018.

Net loss for the first quarter of 2019 was $40.9 million, or $1.17 per share basic and diluted, compared to net loss of $14.7 million, or $0.51 per share basic and diluted for the same period in 2018.

About Sitravatinib

Sitravatinib is a spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET. As an immuno-oncology agent, sitravatinib is being evaluated in combination with nivolumab (OPDIVO), an anti-PD-1 checkpoint inhibitor, in patients whose cancers have progressed despite treatment with a checkpoint inhibitor. Sitravatinib’s potent inhibition of TAM and split family RTKs may overcome resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment, enhancing antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation.

Sitravatinib is also being evaluated as a single agent in a Phase 1b expansion clinical trial emphasizing enrollment of patients whose tumors harbor specific mutations in the CBL protein. When CBL is inactivated by mutation, multiple RTKs, including TAM, VEGFR2 and KIT, are dysregulated and may act as oncogenic tumor drivers in NSCLC and melanoma. Sitravatinib potently inhibits these RTKs and is being investigated as a treatment option for cancer patients with CBL mutations.

About MRTX849

MRTX849 is an investigational, orally-available small molecule that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of NSCLC adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly-identified, KRAS G12C-positive advanced solid tumors.

On April 29, 2019 Teneobio, Inc. and its affiliate TeneoOne, Inc. reported that their investigational new drug application (IND) for TNB-383B, a bispecific T-cell engaging antibody for the treatment of multiple myeloma, was cleared for the initiation of Phase I clinical studies by the US Food and Drug Administration (FDA) on April 24th, 2019 (Press release, TeneoBio, APR 29, 2019, View Source [SID1234535622]). The ongoing development of TNB-383B is being pursued in collaboration with AbbVie, Inc.

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TNB-383B is a fully human bispecific antibody with two binding moieties for B-Cell Maturation Antigen (BCMA) on one arm and a unique anti-CD3 on the other. In preclinical studies, TNB-383B induced T-cell dependent killing of myeloma cells (which express BCMA) but with reduced cytokine secretion, a feature that could limit immune mediated toxicities while retaining cytotoxic activity.

Roland Buelow, CEO of Teneobio, Inc. added "We are looking forward to starting clinical studies with TNB-383B. We believe that Teneobio’s differentiated anti-BCMAxCD3 (TNB-383B), which incorporates a unique T-cell activation anti-CD3, will provide a better therapeutic window for the treatment of multiple myeloma than current BCMA-targeting bispecific antibodies in the clinic. Our T-cell redirecting anti-CD3 platform is also the foundation for additional therapeutics that we are rapidly advancing in our pipeline. These include TNB-486 (anti-CD19xCD3) and TNB-585 (anti-PSMAxCD3) for the treatments of lymphoma and prostate cancer, respectively. We look forward to filing INDs on these additional programs in H2 of 2020."

On April 29, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended March 31, 2019 and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, APR 29, 2019, View Source [SID1234535451]).

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"We remain focused on building awareness of tardive dyskinesia and are encouraged by the continued adoption of INGREZZA among healthcare providers and the patient community. Importantly, a record number of new patients started treatment with INGREZZA during the first quarter," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Within our product pipeline, we remain on track for the NDA submission of opicapone for Parkinson’s disease, plan to discuss the registration program for congenital adrenal hyperplasia with the FDA, and look forward to making progress on the Parkinson’s disease and Friedreich’s ataxia gene therapy programs in collaboration with Voyager Therapeutics. We have laid the foundation to make meaningful strides throughout 2019 and work towards our goal of having three FDA-approved treatments in four indications by 2020."

Financial Results

Total revenue was $138.4 million for the first quarter of 2019, compared to $71.1 million for the same period in 2018.

INGREZZA received U.S. Food and Drug Administration (FDA) approval in 2017, becoming the first medicine approved in the United States for the treatment of adults with tardive dyskinesia. ORILISSA (elagolix) was approved by the FDA for the treatment of endometriosis with associated moderate to severe pain during the third quarter of 2018, with AbbVie sales beginning in August 2018.

The Company reported a net loss of $102.1 million, or $1.12 net loss per share, for the first quarter of 2019, compared to $41.8 million, or $0.47 net loss per share, for the same period in 2018. The increase in net loss for the first quarter of 2019 is primarily due to $113.1 million of in-process research and development (IPR&D) in connection with the strategic collaboration with Voyager. This was partially offset by increased INGREZZA net product sales.

Research and development (R&D) expenses decreased to $37.7 million for the first quarter of 2019, from $48.9 million for the same period in 2018. The decrease in R&D expenses is primarily due to a prior year $8 million non-recurring share-based compensation charge and a prior year $10 million event-based payment to BIAL – Portela & CA, S.A. (BIAL) for opicapone.

In connection with the closing of the Voyager transaction in March 2019, the Company made an upfront payment of $115 million and purchased $50 million of Voyager’s common stock. The Company accounted for this transaction, including related transaction costs, as an asset acquisition and expensed $113.1 million as IPR&D and recorded the $50 million equity investment at $54.7 million as an asset on the Company’s balance sheet based upon the fair value at the timing of closing. The equity investment will be marked to market each quarter with gains and losses recorded to other income/expense.

Sales, general and administrative (SG&A) expenses increased to $87.5 million for the first quarter of 2019, from $58.6 million for the same period in 2018. The increase in SG&A expenses is primarily due to the sales force expansion in the third quarter of 2018, the national launch of a patient-focused disease state awareness campaign, Talk About TD, and an increase in the Branded Pharmaceutical Drug fee expense.

The Company’s balance sheet at March 31, 2019, reflected total assets of $957.7 million, including cash and available for sale investments of $700.8 million, compared to total assets of $993.2 million at December 31, 2018.

Pipeline Highlights

ORILISSA (elagolix) Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine, announced FDA approval and in October 2018 Health Canada approval for ORILISSA for the management of endometriosis with associated moderate to severe pain. The FDA granted priority review to ORILISSA. The FDA grants priority review designation to medicines that, if approved, would provide a significant improvement in the safety or effectiveness of treatment of a serious condition. AbbVie began commercialization of ORILISSA in the United States in August 2018.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension study during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. These replicate Phase III studies were randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with low-dose hormone (add-back) therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies enrolled approximately 400 patients each for an initial six-month placebo-controlled dosing period. At the end of the six months of placebo-controlled evaluation, patients were eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study was an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints were evaluated including the change in fibroid volume and hemoglobin. Bone mineral density was assessed via dual-energy x-ray absorptiometry (DEXA) scan at baseline at the conclusion of dosing and at six months post-dosing. Results from these studies will form the basis for an anticipated New Drug Application (NDA) submission to the FDA for the approval of elagolix in the treatment of uterine fibroids in the middle of 2019.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with BIAL for the development and commercialization of opicapone in the United States and Canada. Opicapone is a once-daily, oral, peripherally-acting, highly-selective catechol-O-methyltransferase inhibitor, being developed as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors for adult patients with Parkinson’s disease and motor fluctuations. The Company met with the FDA in January 2018 and based upon the BIPARK-I and BIPARK-II pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The Company remains on track for an NDA submission in the second quarter of 2019.

CAH Program (NBI-74788) Update

In the second quarter of 2017, the Company successfully completed the Phase I investigational new drug (IND)-opening study of NBI-74788 in healthy volunteer participants. The study was a randomized, open-label, two-period crossover study to evaluate the pharmacokinetics, the effect of food on pharmacokinetics, and the safety of NBI-74788 in a total of 16 healthy adults.

The Company began a Phase II proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adult males and females with classic 21-hydroxylase deficiency CAH in November of 2017. This study will evaluate the safety and tolerability of NBI-74788 in patients with CAH together with the relationship between exposure and specific steroid hormone levels in these patients. In March 2019, positive interim results from this ongoing study demonstrated a clinically meaningful reduction in key biomarkers associated with the management of CAH. NBI-74788 was shown to be well tolerated with no serious adverse events reported to date. The Company plans to meet with the FDA during the third quarter of 2019 to discuss the registration program for NBI-74788 in adult and pediatric patients with CAH.

Voyager Collaboration and VY-AADC Program

Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine’s expertise in neuroscience, drug development and commercialization with Voyager’s innovative gene therapy programs targeting severe neurological diseases. The collaboration became effective in March 2019 following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Based on the results from the VY-AADC Phase I programs in Parkinson’s disease, RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial was initiated to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF-time during the day as measured by a validated self-reported patient diary.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 877-876-9173 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine’s website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective VMAT2 inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release from presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications
INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

On April 29, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) and I-Mab Biopharma (I-Mab), a China-based clinical stage biopharmaceutical company exclusively focused on the development of innovative biologics in immuno-oncology and autoimmune diseases, reported that the first patient has been dosed in a phase 3 randomized and multi-center clinical study in Taiwan to evaluate MorphoSys’s investigational human CD38 antibody MOR202/TJ202 in combination with lenalidomide in patients with relapsed or refractory multiple myeloma (Press release, MorphoSys, APR 29, 2019, View Source [SID1234556333]). I-Mab has exclusive rights for development and commercialization of MOR202/TJ202 in China, Taiwan, Hong Kong and Macao.

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"The initiation of our first phase 3 trial represents another important milestone in advancing MOR202/TJ202 towards registration with the hope of providing more therapeutic options for multiple myeloma patients globally. With planned enrollment of 291 patients, this will be a broad trial of this second most common blood cancer worldwide," said Dr. Joan Shen, M.D., Head of R&D at I-Mab. "In parallel with our pivotal phase 2 trial of MOR202/TJ202 in combination with dexamethasone, the phase 3 study will further assess the efficacy of MOR202/TJ202 as a potential second line treatment in multiple myeloma."

Under I-Mab’s fast-to-market development strategy, the phase 3 study, if successful, could lead to a biologics license application (BLA) in Greater China. The randomized, open-label, parallel-controlled, multicenter study will be conducted in mainland China and Taiwan to evaluate the efficacy and safety of the combination of MOR202/TJ202 plus lenalidomide (LEN) and dexamethasone (DEX) versus the combination of LEN and DEX in patients with relapsed or refractory multiple myeloma who received at least one prior line of treatment. The primary endpoint is to evaluate the progression-free survival (PFS) comparing the efficacy of MOR202/TJ202 plus LEN/DEX versus LEN/DEX.

The dosing of the first patient triggers a milestone payment of USD 3 million to MorphoSys.

"We are delighted that our partner I-Mab has started a phase 3 trial of MOR202/TJ202 in combination with lenalidomide in Asia in addition to the ongoing phase 2 trial of MOR202 in combination with dexamethasone. We see a high medical need for the treatment of patients with multiple myeloma in the Chinese region and look forward to supporting I-Mab in developing this investigational compound for these patients," said Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

With MorphoSys’s support through a licensing agreement in November 2017, I-Mab is currently leading the clinical development of MOR202/TJ202 in Greater China, including mainland China, Hong Kong, Macao and Taiwan. In addition to Taiwan, I-Mab has filed an investigational new drug (IND) application to China’s National Medical Products Administration in August 2018. Previously on March 20, 2019, MorphoSys and I-Mab announced the first patient dosing of MOR202/TJ202 in a phase 2 multi-center clinical study in Taiwan in patients with relapsed or refractory multiple myeloma.

About MOR202/TJ202
MOR202/TJ202 is an investigational human monoclonal antibody derived from MorphoSys’s HuCAL antibody technology. The antibody is directed against CD38 on the surface of multiple myeloma cells, which has been characterized as one of the most strongly and uniformly expressed antigens on the surface of malignant plasma cells. According to its suggested mode of action, the antibody recruits cells of the body’s immune system to kill the tumor through antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). The antibody does not involve complement dependent cytotoxicity, or CDC, an additional immune mechanism involved in tumor cell killing. Scientific research suggest that an anti-CD38 antibody may have therapeutic potential also in other cancers as well as autoimmune diseases. Based on an exclusive regional licensing agreement signed in late 2017, I-Mab owns the exclusive rights for development and commercialization of MOR202/TJ202 in China, Taiwan, Hong Kong and Macao.

On April 29, 2020 Oncoceutics, Inc. reported that the United States Patent and Trademark Office (USPTO) has issued patent #10,266,533 entitled "7-BENZYL-4-(2-METHYLBENZYL)-2,4,6,7,8,9-HEXAHYDROIMIDAZO [1,2-A]PYRIDO[3,4-E]PYRIMIDIN-5(1H)-ONE, ANALOGS THEREOF, AND SALTS THEREOF AND METHODS FOR THEIR USE IN THERAPY" with an expiration date of January 29, 2036 (Press release, Oncoceutics, APR 29, 2019, View Source [SID1234558357]). This patent covers the composition of matter for ONC213, its di-salt formulation, and its use in the treatment of cancer. In addition to the claims related to ONC213, the patent also contains claims for millions of structurally-related imipridones.

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ONC213 is the fourth molecule in the company’s pipeline of "imipridone" family of anti-cancer small molecules that target G protein-coupled receptors, following ONC201, ONC206 and ONC212. As with other members of the imipridone class, ONC213 has very attractive chemical and biological properties including oral bioavailability, chemical stability and a large therapeutic index.

"We are delighted by the decision of the US Patent Office to grant Composition of Matter to the novel molecule ONC213," said Martin Stogniew, Ph.D., Chief Development Officer of Oncoceutics. "This patent paves the way for future generations of imipridones to enter the clinic and eventually benefit the lives of patients."

ONC213 has demonstrated anti-cancer activity and safety in various preclinical oncology models across hematological malignancies and solid tumors tested in the lab of Principal investigator Dr. Yubin Ge, Associate Professor of Oncology at the Karmanos Cancer Institute and Wayne State University School of Medicine. Results presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated that ONC213 targets leukemic stem cells in patient-derived xenograft mouse models, is well tolerated and combines synergistically with Bcl-2 inhibitor Venetoclax. Dr. Ge recently received a grant from the Kids Without Cancer and the Children’s Hospital of Michigan Foundation to further determine the mechanism of action of ONC213 and enable biomarker selection for clinical studies.

"Our team at the Karmanos Cancer Institute is excited by the potential of ONC213 as a new type of cancer therapy," said Dr. Yubin Ge. "We look forward to continuing development of this novel agent as it makes its way towards the clinic."