First Patient Infused in Joint Humanigen/Kite, A Gilead Company Clinical Study

On June 30, 2020 Humanigen, Inc., (HGEN) ("Humanigen"), a clinical stage biopharmaceutical company focused on preventing and treating cytokine release syndrome (CRS) with lenzilumab, the company’s proprietary Humaneered anti-human granulocyte macrophage-colony stimulating factor (GM-CSF) monoclonal antibody, reported that the first patient has been infused in the ZUMA-19 study, which is being conducted in collaboration with Kite, A Gilead Company (Press release, Humanigen, JUN 30, 2020, View Source [SID1234561585]). Details of the ZUMA-19 study can be found at www.clinicaltrials.gov/ct2/show/NCT04314843.

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"We are excited to see this clinical collaboration move forward as we seek to understand the potential benefit of lenzilumab being administered with CAR T therapy in patients with relapsed or refractory large B-cell lymphoma," said Cameron Durrant, MD, chief executive officer of Humanigen.

Exelixis Announces Initiation of CONTACT-02 Phase 3 Pivotal Trial of Cabozantinib in Combination With Atezolizumab in Previously Treated Metastatic Castration-Resistant Prostate Cancer

On June 30, 2020 Exelixis, Inc. (NASDAQ: EXEL) reported the initiation of CONTACT-02, a global phase 3 pivotal trial of cabozantinib (CABOMETYX) in combination with atezolizumab (TECENTRIQ) in patients with metastatic castration-resistant prostate cancer (CRPC) who have been previously treated with one novel hormonal therapy (Press release, Exelixis, JUN 30, 2020, View Source [SID1234561584]). CONTACT-02 is part of a clinical trial collaboration between Exelixis and Roche that includes two additional phase 3 pivotal trials – CONTACT-01 in patients with metastatic non-small cell lung cancer (NSCLC) who have been previously treated with an immune checkpoint inhibitor and platinum-containing chemotherapy, which was initiated in June, and the planned CONTACT-03 trial in patients with metastatic renal cell carcinoma (RCC) who previously received an immune checkpoint inhibitor.

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"As many patients with advanced, castration-resistant prostate cancer who have progressed on a novel hormonal therapy wish to avoid or delay chemotherapy, more treatment options are needed," said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. "We are encouraged by the positive early stage results for the combination of cabozantinib and atezolizumab seen for prostate cancer patients in cohort 6 of the COSMIC-021 trial, and we are pleased to begin this pivotal trial that will further evaluate how the combination may improve outcomes for these patients as part of our ongoing partnership with Roche."

CONTACT-02 is a global, multicenter, randomized, phase 3, open-label study that plans to enroll approximately 580 patients at 250 sites. Patients will be randomized 1:1 to the experimental arm of cabozantinib in combination with atezolizumab and the control arm of a second novel hormonal therapy (either abiraterone and prednisone or enzalutamide). The co-primary endpoints of the trial are progression-free survival and overall survival. Additional endpoints include objective response rate, prostate-specific antigen response rate and duration of response. The trial is sponsored by Exelixis and co-funded by Roche, Ipsen and Takeda Pharmaceutical Company Limited.

The design of CONTACT-02 was informed by results from the ongoing COSMIC-021 trial — a phase 1b study of cabozantinib and atezolizumab in multiple advanced solid tumors including NSCLC, CRPC and RCC. Initial results from cohort 6 were presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium. The interim analysis was updated with additional biomarker results and presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program.

More information about CONTACT-02 is available at ClinicalTrials.gov (NCT04446117).

About CRPC
According to the American Cancer Society, in 2020, approximately 192,000 new cases of prostate cancer will be diagnosed and 33,000 people will die from the disease.1 Prostate cancer that has spread beyond the prostate and does not respond to androgen-suppression therapies — a common treatment for prostate cancer — is known as metastatic CRPC.2 Researchers estimate that in 2020, 43,000 men will be diagnosed with metastatic CRPC, which has a median survival of less than two years.3,4,5

About CABOMETYX (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide.

CABOMETYX in combination with atezolizumab is not indicated for previously treated metastatic CRPC.

About Exelixis’ Collaboration with Ipsen
On February 29, 2016, Exelixis and Ipsen jointly announced a collaboration agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. On December 21, 2016, this agreement was amended to include commercialization rights for Ipsen in Canada. Ipsen has opted in to participate in the funding of CONTACT-01 and CONTACT-02. Under the parties’ collaboration agreement, Ipsen will have access to the respective study results to support potential future regulatory submissions in their territory.

About Exelixis’ Collaboration with Takeda
On January 30, 2017, Exelixis and Takeda jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications in Japan. Takeda has opted in to participate in the funding of CONTACT-01 and CONTACT-02. Under the parties’ collaboration agreement, Takeda will have access to the respective study results to support potential future regulatory submissions in their territory.

Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.

Important Safety Information

Warnings and Precautions
Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients in RCC and HCC studies. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.

Perforations and Fistulas: Gastrointestinal (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of perforations and fistulas, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a Grade 4 fistula or a GI perforation.

Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic event requiring medical intervention.

Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension occurred in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.

Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.

Proteinuria: Proteinuria occurred in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.

Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 3 weeks prior to scheduled dental surgery or invasive dental procedures, if possible. Withhold CABOMETYX for development of ONJ until complete resolution.

Impaired Wound Healing: Wound complications occurred with CABOMETYX. Withhold CABOMETYX for at least 3 weeks prior to elective surgery. Do not administer CABOMETYX for at least 2 weeks after major surgery and until adequate wound healing is observed. The safety of resumption of CABOMETYX after resolution of wound healing complications has not been established.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic findings on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.

Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, decreased appetite, PPE, nausea, hypertension, and vomiting.

Drug Interactions

Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.

Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.

Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment.

Oncternal Therapeutics Announces Orphan Drug Designations of Cirmtuzumab ROR1 Antibody for Treatment of Mantle Cell Lymphoma and for Treatment of Chronic Lymphocytic Leukemia

On June 30, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that the United States Food and Drug Administration (FDA) has granted the company orphan drug designations of cirmtuzumab for treatment of mantle cell lymphoma (MCL) and for treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL) (Press release, Oncternal Therapeutics, JUN 30, 2020, View Source [SID1234561583]). Cirmtuzumab is an investigational anti-ROR1 monoclonal antibody being evaluated in clinical trials in patients with MCL, CLL and HER2-negative breast cancer.

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Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs or biologics intended to treat rare diseases or conditions, which are defined as diseases or conditions that affect fewer than 200,000 people in the United States or that affect more than 200,000 people but where there is no reasonable expectation that the costs of developing and marketing the drug will be recovered through future sales of the drug in the United States. Orphan drug designation for cirmtuzumab qualifies Oncternal for certain benefits including tax credits for qualified clinical trials, exemption from certain FDA application fees, and the potential for market exclusivity upon regulatory approval, if received, for an orphan-designated indication.

"We are pleased to receive orphan drug designations for cirmtuzumab, our potentially first-in-class investigational ROR1 antibody," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are excited about cirmtuzumab’s potential for the treatment of patients with ROR1-expressing cancers, including MCL, CLL, HER2-negative breast cancer and other solid tumors, and look forward to further advancing its development to benefit patients with significant unmet medical needs."

MCL is an aggressive form of non-Hodgkin’s lymphoma. MCL prevalence is estimated to be approximately 13,000 to 21,000 patients in the United States. MCL is an aggressive cancer that carries a poor prognosis, with a median survival of about two to five years and a 10-year survival rate of approximately 5%-10%.

CLL is the most common form of leukemia in adults, accounting for 25-30% of all leukemias in the United States. CLL prevalence is estimated to be approximately 158,000 to 178,000 patients in the U.S. Despite various recently approved therapies, CLL generally remains incurable.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL or MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with HER2-negative metastatic breast cancer is being conducted at the UC San Diego School of Medicine.

ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. The FDA has granted Orphan Drug Designations to cirmtuzumab for the treatment of mantle cell lymphoma and chronic lymphocytic leukemia/small lymphocytic lymphoma. Cirmtuzumab is in clinical development and has not been approved by the FDA for any indication.

Oncternal Therapeutics Announces Increased Focus of the Cirmtuzumab ROR1 Antibody Program on Mantle Cell Lymphoma

On June 30, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported an updated clinical strategy for its investigational ROR1 monoclonal antibody, cirmtuzumab, that prioritizes development in mantle cell lymphoma (MCL), based on encouraging interim clinical results from the ongoing Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma (CIRLL) Phase 1/2 clinical trial that were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Annual Meeting (ASCO 2020) in May 2020 (Press release, Oncternal Therapeutics, JUN 30, 2020, View Source [SID1234561582]).

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The Company reported a 58% complete response (CR) rate, a 83% overall best objective response rate (ORR), and a progression free survival rate of 17.5 months with a median follow-up of 8.3 months, for patients with relapsed/refractory MCL in the ongoing Phase 1/2 CIRLL clinical trial of cirmtuzumab in combination with ibrutinib, a Bruton’s tyrosine kinase (BTK) inhibitor, at ASCO (Free ASCO Whitepaper) 2020. These response rates in heavily pre-treated patients were higher than the historical published CR of 23% and ORR of 67% for single-agent ibrutinib for patients with MCL who had received more than one prior therapy (Rule 2019, Haematologica). Four of these patients with MCL had been previously treated with and responded to ibrutinib, prior to participating in the CIRLL study. All four of these patients responded to the combination of cirmtuzumab and ibrutinib, two achieving CRs and two achieving partial responses. The Company believes that the interim results presented at ASCO (Free ASCO Whitepaper) 2020 are clinically relevant given the unmet medical need for patients with MCL.

As a result, the Company is amending the CIRLL study to increase the number of patients with relapsed/refractory MCL to be enrolled in the Phase 2 Expansion Cohort to at least 20 patients and to allow enrollment of patients with a broader range of prior BTK inhibitor treatments.

The Company has also requested a meeting with the U.S. Food and Drug Administration (FDA) to discuss the results of the recent interim analysis of the CIRLL study and to seek guidance on a potential accelerated approval pathway for cirmtuzumab plus ibrutinib in patients with relapsed/refractory MCL.

At ASCO (Free ASCO Whitepaper) 2020, the Company also reported a 100% progression-free survival rate, 88% ORR and 3% CR rate, with a median follow-up of 12.8 months, for patients with chronic lymphocytic leukemia (CLL) treated with cirmtuzumab in combination with ibrutinib in the CIRLL study. These interim data did not satisfy the hypothesis that ibrutinib plus cirmtuzumab would produce a CR rate 25% greater than the historical response rate for ibrutinib alone. Based on these interim results, Oncternal will continue treatment and follow-up of the patients with CLL who are already enrolled in the CIRLL study for up to two years or until disease progression, but will limit total enrollment of patients in the randomized Phase 2 CLL cohort to approximately 35 patients, in order to focus resources on the MCL portion of the study. The Company believes that, while significant unmet medical need exists in both CLL and MCL, the MCL indication may offer a more rapid path to potential regulatory approval.

Additionally, Oncternal plans to further explore clinical combination strategies for cirmtuzumab for patients with hematologic malignancies. Accordingly, the Company is supporting a new, investigator-sponsored Phase 2 clinical trial of cirmtuzumab in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL in collaboration with the University of California San Diego School of Medicine (UC San Diego). Preclinical studies performed in the laboratory of Dr. Thomas Kipps at UC San Diego reported synergy between cirmtuzumab and venetoclax, providing a rationale for this combination clinical trial (Rassenti 2017, PNAS).

"We are excited about the promising clinical data reported for cirmtuzumab in combination with ibrutinib for patients with relapsed/refractory MCL, for whom a significant unmet medical need exists for well-tolerated therapies that provide more complete and durable responses. We plan to prioritize the development of cirmtuzumab for patients with MCL and expect that the planned changes will accelerate the Company’s timetable for initiating a potential registrational study for cirmtuzumab, while having an overall favorable budget impact," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "Additionally, we are pleased to support a new, investigator-sponsored Phase 2 clinical trial of cirmtuzumab in combination with venetoclax for the treatment of patients with CLL, based on promising published preclinical data. Our collaborators at UC San Diego and the California Institute for Regenerative Medicine (CIRM) have indicated that they support our revised development strategy for cirmtuzumab."

About the CIRLL Clinical Trial

The CIRLL clinical trial (Cirmtuzumab and Ibrutinib for Relapsed Lymphoma and Leukemia, Study CIRM-0001) is a Phase 1/2 trial evaluating cirmtuzumab in combination with ibrutinib in separate groups of patients with MCL or CLL. Enrollment has been completed in the dose-finding cohorts in CLL and MCL, and the dose-expansion cohort in CLL. Based on the data from the dose-finding cohorts, the recommended dosing regimen was determined to be 600 mg of cirmtuzumab administered intravenously every two weeks for three doses, followed by dosing every four weeks, in combination with 560 mg of ibrutinib once daily for patients with MCL, or 420 mg of ibrutinib administered once daily for patients with CLL, which are the FDA-approved doses of ibrutinib in these indications.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL or MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine.

ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. Cirmtuzumab is in clinical development and has not been approved by the FDA for any indication.

BioInvent Submits a CTA for a Phase I/IIa trial of BI-1808, A First-in-class Anti-TNFR2 Antibody for the Treatment of Patients With Solid Tumors and CTCL

On June 30, 2020 BioInvent International AB ("BioInvent") (OMXS: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, reported it has submitted a clinical trial application (CTA) to begin a Phase I/IIa, first-in-human study of BI-1808, a monoclonal antibody to tumor necrosis factor receptor 2 (TNFR2), as a single agent and in combination with KEYTRUDA (pembrolizumab) for the treatment of solid tumors and cutaneous T-cell lymphoma (CTCL) (Press release, BioInvent, JUN 30, 2020, View Source [SID1234561581]).

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The study will explore the safety, tolerability, and potential signs of efficacy of BI-1808 as a single agent and in combination with KEYTRUDA in patients with ovarian cancer, non-small cell lung cancer and cutaneous T cell lymphoma. It will also investigate the expression of potential immunological markers that might be associated with clinical responses. It will be conducted at several sites across Europe and the U.S. and is expected to enroll approximately 120 patients.

The Phase I stage is divided into two sections. Part A is a dose escalation of BI-1808 to assess safety, tolerability, and pharmacokinetics & pharmacodynamics, and to determine the recommended dose as a single agent for Phase II trials. It will be followed by part B, which will explore the safety, tolerability and recommended dose of BI-1808 in combination with KEYTRUDA. Phase IIa will consist of expansion cohorts to assess signs of efficacy of BI-1808 as single agent and in combination with KEYTRUDA in lung cancer and ovarian cancer patients. A separate cohort will explore the activity as single agent in CTCL (Sézary syndrome and mycosis fungoides).

Martin Welschof, CEO of BioInvent, says, "Targeting TNFR2 for cancer therapy is a very promising approach and BI-1808 is a further demonstration of the ability of our proprietary n-CoDeR and F.I.R.S.TTM platforms to generate antibodies with novel, first-in-class mechanisms of action. This Phase I/IIa study of BI-1808 marks the first anti-TNFR2 antibody to enter clinical evaluation. With CTAs filed in Europe, we expect to submit a U.S. IND in the coming months, and to enroll the first patient in Q4 2020."