On June 24, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that the first patient has been dosed in the Phase 1b portion of KOMET-001, a Phase 1/2 clinical trial of the Company’s oral, potent and selective menin inhibitor, KO-539, in patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Kura Oncology, JUN 24, 2021, View Source [SID1234584316]).

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KO-539 demonstrated a wide therapeutic window in the Phase 1a dose-escalation portion of KOMET-001, with promising single-agent activity from 50 mg to 800 mg in an all-comer population of patients with relapsed or refractory AML, including patients with NPM1 mutations and KMT2A rearrangements. The Phase 1b portion of the study is designed to determine the lowest dose of KO-539 that provides maximum biologic and clinical effect in two expansion cohorts – a low dose (200 mg) and a high dose (600 mg) – each enriched with NPM1-mutant and KMT2A-rearranged relapsed/refractory AML patients. Both doses have demonstrated preliminary evidence of biologic and clinical activity and were determined to be safe and well tolerated in the Phase 1a portion of the study.

Kura expects to enroll at least 12 patients in each of the Phase 1b expansion cohorts and assess those patients for safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy in order to determine the recommended Phase 2 dose. In addition, the Phase 1b gives the Company flexibility to enroll up to 18 additional patients per cohort, as appropriate. Kura believes that data from patients enrolled in the cohort selected as the recommended Phase 2 dose can be included as part of the registration-directed portion of the KOMET-001 trial.

"We believe KO-539 has the potential to be both a first-in-class and a best-in-class menin inhibitor," said Stephen Dale, M.D., Chief Medical Officer of Kura Oncology. "We intend to conduct a comprehensive clinical development plan for KO-539, both as a monotherapy and in front-line combination studies. A critical component of this plan is the determination of an optimal dose, particularly given the compelling efficacy, safety and wide therapeutic window of KO-539. These Phase 1b expansion cohorts enable us to gather a more robust dataset in our targeted populations and help refine selection of a recommended dose for Phase 2 and beyond, while maintaining an aggressive development timeline for the program. We look forward to presenting data at a future medical meeting."

In December 2020, Kura reported preliminary clinical data from the KOMET-001 clinical trial of KO-539 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. These data were highlighted by single-agent activity in patients with relapsed or refractory AML, including patients with NPM1 mutations and KMT2A rearrangements. KO-539 also demonstrated a favorable safety and tolerability profile, with no drug discontinuations due to treatment-related adverse events and no evidence of QTc prolongation.

About Acute Myeloid Leukemia

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor, especially in the relapsed/refractory setting. More than 50% of patients with AML who achieve a CR after induction therapy relapse within one to three years, and less than 10% of those with relapsed/refractory AML are alive at three years. Prognosis is poor in patients with KMT2A rearrangements and in those with co-mutations that may include NPM1.

About KOMET-001

KOMET-001 (Kura Oncology Menin Inhibitor Trial) is a Phase 1/2, first-in-human, open-label trial to determine the safety, tolerability and anti-tumor activity of KO-539 in patients with refractory or relapsed AML. The Phase 1b portion is currently open to enroll patients with NPM1 mutations or KMT2A-rearrangments. A Phase 2 registration-enabling expansion portion is planned following determination of the optimal Phase 2 dose. Additional information about KOMET-001 can be found at kuraoncology.com/clinical-trials-komet.

About KO-539

KO-539 is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In preclinical models, KO-539 inhibits the KMT2A/MLL protein complex and exhibits downstream effects on HOXA9/MEIS1 expression and potent anti-leukemic activity in genetically defined preclinical models of AML. KO-539 has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML.

On June 24, 2021 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported the expansion of its royalty agreement with entities managed by HealthCare Royalty Management, LLC (HCR) for up to $100 million in new financing to support the ongoing development and commercialization of XPOVIO (selinexor), the Company’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound, and for the clinical development of Karyopharm’s other programs, including eltanexor (Press release, Karyopharm, JUN 24, 2021, View Source [SID1234584315]). XPOVIO is currently marketed in the U.S. for multiple hematologic malignancy indications and has received conditional marketing authorization by the European Commission for patients with heavily pretreated multiple myeloma. Eltanexor is currently being investigated for the treatment of patients with refractory myelodysplastic syndrome.

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Under the terms of the amended agreement, Karyopharm received $60 million and is eligible to receive two additional $20 million payments subject to certain prespecified future development and commercial milestones. HCR will receive tiered royalty payments based on worldwide net revenues of XPOVIO and any other future products. This expanded agreement follows a previous investment of $75 million that HCR made in Karyopharm in September 2019.

Karyopharm expects that the $60 million received from HCR under this expanded royalty agreement, combined with its existing cash, cash equivalents and investments, and the cash expected to be generated from product sales, will be sufficient to fund its operations into the middle of 2023.

"We are thrilled to expand our relationship with HCR and this additional non-dilutive financing provides Karyopharm with immediate and substantial capital to support the ongoing commercialization and clinical development of XPOVIO and our other products in future cancer indications, including in a variety of high unmet need solid tumor settings," said Richard Paulson, MBA, President and Chief Executive Officer of Karyopharm. "As we continue to make progress on our commercialization efforts, we are delighted to have the support and confidence of HCR to further expand our clinical development efforts on behalf of patients across the globe."

Clarke Futch, Chairman and Chief Executive Officer of HCR stated: "Based on the strong relationship we have built with the Karyopharm team over the past few years and our increasing confidence in the long-term potential for XPOVIO, we are delighted to broaden our investment in Karyopharm and further support the expansion of XPOVIO, including into solid tumors."

On June 24, 2021 Incyte (Nasdaq:INCY) reported the outcome of a meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which reviewed the Biologics License Application (BLA) for retifanlimab, an intravenous PD-1 inhibitor, as a potential treatment for adult patients with locally advanced or metastatic squamous cell carcinoma of the anal canal (SCAC) who have progressed on, or who are intolerant of, platinum-based chemotherapy (Press release, Incyte, JUN 24, 2021, View Source [SID1234584314]). The Committee voted 13-4 that a regulatory decision on retifanlimab for the treatment of advanced or metastatic SCAC should be deferred until further data are available from clinical trial POD1UM-303, a confirmatory trial in platinum-naïve advanced SCAC that is currently underway.

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The ODAC provides the FDA with independent opinions and recommendations from outside experts on marketed and investigational medicines for use in the treatment of cancer. The FDA considers the advice of the ODAC in its review but is not bound to follow its recommendations.

"While we are disappointed by the outcome of today’s ODAC vote, we will continue to work closely with the FDA as it completes its review of the BLA for retifanlimab," said Lance Leopold, M.D., Group Vice President, Immuno-Oncology Clinical Development, Incyte. "Patients with advanced SCAC who have progressed after first-line platinum-based chemotherapy currently have no FDA-approved treatments available to them and face an extremely poor prognosis. We continue to believe that retifanlimab can provide an additional, much-needed option for these patients based on the favorable benefit/risk shown in our trial."

The BLA for retifanlimab is based on data from the Phase 2 POD1UM-202 trial evaluating retifanlimab in 94 previously treated patients with locally advanced or metastatic SCAC who have progressed on, or are intolerant of, platinum-based chemotherapy. The FDA previously granted retifanlimab Orphan Drug Designation for the treatment of anal cancer, along with Priority Review, and set the Prescription Drug User Fee Act (PDUFA) target action date for retifanlimab as July 25, 2021.

SCAC is almost always associated with human papillomavirus (HPV) and HIV infections and accounts for nearly 3% of digestive system cancers.1 Patients with metastatic SCAC have a poor 5-year survival, and there are no FDA-approved treatments for patients who have progressed after first-line chemotherapy.2

About POD1UM-202

POD1UM-202 (NCT03597295) is an open-label, single-arm, multicenter, Phase 2 study evaluating retifanlimab in 94 patients with locally advanced or metastatic squamous cell carcinoma of the anal canal (SCAC) who have progressed on, or were ineligible for or intolerant of, platinum-based chemotherapy. Retifanlimab 500mg is administered as an intravenous infusion every 4 weeks for up to 2 years.

The primary endpoint was overall response rate (ORR) as determined by independent central review using RECIST v1.1. Secondary endpoints included duration of response (DOR), disease control rate (DCR), progression-free survival (PFS) and overall survival (OS); safety and pharmacokinetics.

For more information about the study, please visit View Source

About POD1UM

The POD1UM (PD1 Clinical Program in Multiple Malignancies) clinical trial program for retifanlimab includes Phase 1, 2 and 3 studies for patients with solid tumors both as monotherapy and in combination with Incyte’s early development portfolio. Additional indications in late-stage development include platinum-naïve squamous cell carcinoma of the anal canal (SCAC), microsatellite instability-high endometrial cancer, Merkel cell carcinoma and non-small cell lung cancer.

About Retifanlimab

Retifanlimab (formerly INCMGA0012), an investigational intravenous PD-1 inhibitor, is currently under evaluation in Phase 1, 2 and 3 studies for patients with solid tumors both as monotherapy and in combination with Incyte’s early development portfolio. Additional indications in late-stage development include platinum-naïve squamous cell carcinoma of the anal canal (SCAC), microsatellite instability-high endometrial cancer, Merkel cell carcinoma and non-small cell lung cancer.

Retifanlimab has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of anal cancer. A Marketing Authorization Application for retifanlimab is also under review by the European Medicines Agency.

In 2017, Incyte entered into an exclusive collaboration and license agreement with MacroGenics, Inc. for global rights to retifanlimab. In 2019, Incyte and Zai Lab announced a collaboration and license agreement for the development and commercialization of retifanlimab in Greater China.

On June 24, 2021 GT Biopharma, Inc. ("GT Biopharma" or the "Company") (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary natural killer (NK) cell engager (TriKE) protein biologic technology platform, reported that GT Biopharma CEO, Tony Cataldo will be presenting as a VIP speaker at the upcoming Sir Anthony Ritossa’s Global Family Office Investment Summit in Monaco from June 30 to July 2, 2021 (Press release, GT Biopharma, JUN 24, 2021, View Source [SID1234584313]).

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The Sir Anthony Ritossa’s Global Family Office Investment Summit in Monaco is one of a series of international summits held annually as a platform for exceptional networking between Family Offices and Thought Leaders from all over the world. Summit delegations consist of prestigious family offices, private investors, sheikhs, royal families and leading businesses from 30+ countries representing more than $4.5 trillion in wealth.

On June 24, 2021 EQRx, a company committed to developing and delivering important new medicines to patients at radically lower prices, and Exscientia, an AI-driven pharmatech company with a mission to radically improve how drugs are discovered, reported a strategic research and development collaboration agreement (Press release, EQRx, JUN 24, 2021, View Source [SID1234584312]). The collaboration will leverage the AI capabilities of Exscientia to accelerate the discovery of small molecule therapeutic drug candidates in multiple therapeutic areas, including oncology and immunology, further expanding the breadth of EQRx’s pipeline of novel therapies.

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This innovative collaboration is focused on creating significant improvements in the traditional drug discovery and development processes by improving the probability of success through each company’s unique capabilities to accelerate the delivery of innovative new medicines—from discovery to commercialization.

Under the terms of the agreement, the strengths of both companies will be leveraged throughout the drug development process. Exscientia will lead the discovery phase through to Investigational New Drug application (IND) filing, while EQRx will be responsible for clinical development, regulatory and commercialization efforts. EQRx and Exscientia will equally share in the discovery, development and commercialization costs.

"Exscientia is a leader in AI-driven drug discovery, and of particular note, has now brought multiple AI-engineered drug candidates to clinical trials. We believe that our aligned focus on efficiency and quality sets a truly unique course to bring the next generation of innovative medicines to patients faster and at a fraction of the cost," said Alexis Borisy, chairman and chief executive officer of EQRx. "This is a significant step in building EQRx’s robust, sustainable and industry-leading pipeline of important new medicines and substantially accelerates our early-stage research capabilities."

"We are impatient for patients. EQRx and Exscientia are partners who are equally focused on re-engineering the way we create and distribute drugs for better outcomes for more patients. This exciting new collaboration brings together technologies through a new model that has the potential to truly disrupt how we think about efficiently creating innovative new medicines for all patients," said Andrew Hopkins, chief executive officer of Exscientia. "Together with the team at EQRx we look forward to building a successful partnership, to enable more patients to access better drugs, faster."