On May 7, 2019 Sensei Biotherapeutics, Inc., a clinical-stage biopharmaceutical company developing precision immuno-oncology therapies, reported a clinical trial collaboration with AstraZeneca to evaluate the safety, tolerability and preliminary activity of AstraZeneca’s IMFINZI (durvalumab), a human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in combination with SNS-301 (Press release, Sensei Biotherapeutics, MAY 7, 2019, View Source [SID1234535852]). SNS-301, Sensei’s first-in-class immunotherapy candidate, is a therapeutic cancer vaccine utilizing a bacteriophage viral vector targeting aspartate β-hydroxylase (ASPH), a novel embryonic antigen.

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Under the terms of the agreement, AstraZeneca and Sensei will collaborate to evaluate the combination of SNS-301 and IMFINZI in patients with locally advanced head and neck cancer in the neoadjuvant setting in conjunction with TPF chemotherapy prior to surgical resection and in ASPH+ patients with various locally advanced unresectable or metastatic/recurrent solid tumors. AstraZeneca will supply IMFINZI for these clinical studies and Sensei will sponsor and fund the Phase 2 studies. AstraZeneca and Sensei will each retain full worldwide rights to their respective molecules.

"We are extremely pleased to collaborate with AstraZeneca, a leader in the cancer immunotherapy field. SNS-301 targets a novel mechanism to activate and direct ASPH-specific T-cells to tumors. We believe that when combined with IMFINZI, SNS-301 has the potential to deepen and broaden the overall immune-mediated response," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "This clinical collaboration strengthens our position as pioneers of precision oncology therapeutics by focusing on improved clinical outcomes for ASPH-positive patients through novel clinical combinations."

About SNS-301

SNS-301 is a first-in-class immunotherapy candidate utilizing a bacteriophage viral vector that targets human aspartate β-hydroxylase (ASPH), a cell surface enzyme that is normally expressed during fetal development. Following fetal development, the protein is no longer expressed. Expression of ASPH is uniquely upregulated in more than 20 different types of cancer and is related to cancer cell growth, cell motility and invasiveness. ASPH expression levels are inversely correlated with disease prognosis. Though enhanced antigen presentation and other engineered immunotherapeutic features, SNS-301 is designed to overcome self-tolerance and induce robust and durable humoral and cellular immune responses that are specific to ASPH. SNS-301 is delivered through intradermal injection and avoids time consuming and uncomfortable infusions, greatly facilitating ease of use.

About IMFINZI (Durvalumab)

IMFINZI (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumor’s immune-evading tactics and releasing the inhibition of immune responses. As part of a broad development program, IMFINZI is being investigated as monotherapy and in combination with immuno-oncology (IO) agents, small molecules, and chemotherapies across a range of tumors and stages of disease.

On May 7, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported first quarter 2019 financial results and highlighted upcoming milestones (Press release, Kura Oncology, MAY 7, 2019, View Source [SID1234535880]).

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"We have made considerable progress over the past quarter, as we continue to execute against our first registration-directed trial for tipifarnib in patients with HRAS mutant squamous head and neck cancers," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "Meanwhile, our elucidation of the CXCL12 pathway as a therapeutic target of tipifarnib, coupled with identification of associated farnesylated proteins, offers the potential to significantly expand the population of patients who may benefit from treatment with tipifarnib. Ultimately, we believe that CXCL12 pathway biomarkers could enable registrational strategies for tipifarnib in multiple hematologic and solid tumor indications."

"Now, we look forward to a series of important milestones," continued Dr. Wilson, "beginning with an update from our ongoing Phase 2 trial of tipifarnib in angioimmunoblastic T-cell lymphoma (AITL) and CXCL12-positive peripheral T-cell lymphoma (PTCL), which was accepted for oral presentation at both the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress and the International Conference on Malignant Lymphoma (ICML) next month. This will be followed by additional data from our ongoing Phase 2 trial of tipifarnib in HRAS mutant solid tumors, including head and neck squamous cell carcinoma (HNSCC) and other squamous cell carcinomas (SCCs), as well as an update from our ongoing Phase 2 trial in chronic myelomonocytic leukemia (CMML) later in the year."

Recent Highlights

Registration-directed trial of tipifarnib in HRAS mutant HNSCC – Kura’s global, multi-center, open-label, non-comparative registration-directed trial of tipifarnib continues on track. The clinical trial has two cohorts: A non-interventional screening and outcomes cohort (SEQ-HN) and a treatment cohort (AIM-HN). AIM-HN is designed to enroll at least 59 evaluable patients with HRAS mutant HNSCC who have received prior platinum-based therapy. AIM-HN initiated in November 2018 and is expected to take approximately two years to fully enroll.

Farnesylated proteins associated with CXCL12 expression – In April 2019, Kura reported new findings at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting suggesting that gene expression of the exclusively farnesylated proteins RHOE and PRICKLE2 is strongly associated with CXCL12 expression in bone marrow stroma. These findings provide key evidence supporting the inhibition of the CXCL12 pathway as a mechanism of action mediating the activity of tipifarnib and other farnesyl transferase inhibitors.

Potential expansion to other CXCL12+ lymphoma indications – Kura reported additional findings at AACR (Free AACR Whitepaper) identifying CXCL12 expression as a potential biomarker of clinical benefit in patients with diffuse large B-cell lymphoma (DLBCL) as well as mycosis fungoides, the most common form of cutaneous T-cell lymphoma (CTCL). The results were obtained from an analysis of a subset of samples from a previously conducted Phase 2 trial of tipifarnib in patients with relapsed and refractory lymphomas and are consistent with similar findings from the Company in other indications such as PTCL, acute myeloid leukemia (AML) and pancreatic cancer.

Emerging pipeline of clinical-stage drug candidates – Kura is advancing two additional pipeline programs: KO-947, a small molecule inhibitor of extracellular signal-related kinase (ERK), and KO-539, a potent and selective small molecule inhibitor of the menin-mixed lineage leukemia (menin-MLL) interaction. The Company continues to evaluate dosing regimens for KO-947 and anticipates having data from its Phase 1 clinical trial later this year. Meanwhile, the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for KO-539 and the Company is preparing to initiate a Phase 1 clinical trial in patients with relapsed or refractory AML in mid-2019.

Financial Results

Research and development expenses for the first quarter of 2019 were $10.4 million, compared to $11.6 million for the first quarter of 2018.

General and administrative expenses for the first quarter of 2019 were $4.6 million, compared to $3.4 million for the first quarter of 2018.

Net loss for the first quarter of 2019 was $13.9 million, compared to $14.6 million for the first quarter of 2018.

Cash, cash equivalents and short-term investments totaled $165.5 million as of March 31, 2019, compared with $179.0 million as of December 31, 2018.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2021.

Upcoming Milestones

Additional data from the ongoing Phase 2 trial of tipifarnib in PTCL, including duration of response data from the AITL cohort and additional data from the CXCL12-positive PTCL cohort, at EHA (Free EHA Whitepaper) and ICML in June 2019

Additional data on biomarkers associated with elevated CXCL12 expression at EHA (Free EHA Whitepaper) and ICML in June 2019

Additional data from the ongoing Phase 2 trial of tipifarnib in HRAS mutant solid tumors, including HNSCC and other SCCs, in the second half of 2019

Additional data from the ongoing Phase 2 trial of tipifarnib in CMML in 2019

Data from the Phase 1 dose-escalation trial of KO-947 in 2019

Initiation of the Phase 1 clinical trial of KO-539 in mid-2019

Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, May 7, 2019, to discuss the financial results for the first quarter 2019 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and entering the conference code: 4436235. A live webcast of the call will be available from the Investors and Media section of the Company’s website at www.kuraoncology.com, and will be archived there for 30 days.

On May 7, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended March 31, 2019 (Press release, Opko Health, MAY 7, 2019, View Source [SID1234535948]).

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Business Highlights

RAYALDEE total prescriptions reported by IQVIA increased 121% in 1Q 2019 compared with 1Q 2018: Total prescriptions for the first three months of 2019 increased to over 10,400, compared with approximately 4,700 during the comparable period of 2018.

RAYALDEE Marketing Authorization Applications filed by Vifor Fresenius accepted for review in several European countries: These applications request approval to market RAYALDEE for the treatment of secondary hyperparathyroidism (SHPT) in adult non-dialysis patients with chronic kidney disease (CKD).

American Journal of Nephrology published article and editorial: A post hoc analysis of RAYALDEE pivotal trials was published in the March edition of the American Journal of Nephrology and provides the first prospective data demonstrating that the current clinical practice guideline target for serum total 25-hydroxyvitamin D (30 ng/ml) is too low to decrease elevated serum PTH levels in CKD patients. These data show that the correct target is at least 51 ng/mL, and possibly as high as 93 ng/mL, levels no competitive therapy can reliably attain. This analysis received a favorable review in a co-published editorial by prominent nephrology KOLs.

OPK88003 topline results reported from Phase 2 diabetes and obesity trial: In the mITT patient population, which included all patients who had a single dose of drug and one post-baseline efficacy value, HbA1c decreased by -1.3% (p-value <0.0001) and body weight decreased by -4.4 Kg (p-value <0.0001) from baseline. In the Per Protocol patient population, which included patients who were treated for at least 26 weeks and were compliant with the protocol for the duration of the trial, HbA1c decreased by -1.47% (p-value <0.0001) and body weight decreased by -5.5 Kg (p-value <0.0001) from baseline. The safety profile was similar to that expected for the incretin class of drugs, with GI side effects such as nausea, vomiting and diarrhea mostly mild and occurring during the dose-escalation phase.

4Kscore utilization remained strong during 1Q 2019 with approximately 19,400 tests performed: With all required documentation nearly complete, the company plans to make a submission shortly to the FDA for approval or clearance. In addition, OPKO has submitted a request for Medicare coverage reconsideration to its Medicare Administrative Contractor.

BioReference and its GeneDx subsidiary expand access to commercially insured lives: BioReference and GeneDx have been selected for inclusion in the UnitedHealthcare Preferred Lab Network beginning July 1, 2019. This was a comprehensive process that evaluated service standards, turnaround time, quality and accreditation. More than 300 laboratories were invited to apply; only 100 submitted applications due to the complexity of the requirements, and BioReference and GeneDx, along with five other laboratories, earned a place in the Preferred Lab Network. In addition, effective April 1, 2019, BioReference and GeneDx are now in-network providers with Humana, which provides access to 11 million additional lives.
Financial Highlights

Consistent with financial guidance issued in February 2019, the net loss for the first quarter of 2019 was $80.8 million, compared with a net loss of $43.1 million for the comparable period of 2018.

Consolidated revenues for the first quarter of 2019 were $222.5 million, compared with $254.9 million for the comparable period of 2018. During the 2019 quarter, revenue from services was $178.9 million, revenue from products was $25.3 million, including RAYALDEE net revenue of $5.8 million, and revenue from licensing and intellectual property was $18.3 million. The decrease in revenue from services principally reflect reimbursement headwinds from higher denial rates in the company’s esoteric testing lines of business.

Operating expenses for the first quarter of 2019 of $297.8 million included continued investment in the company’s pharmaceutical pipeline, with R&D expense of $36.5 million, principally for pediatric trials with the hGH-CTP long-acting human growth hormone product.

In February 2019 OPKO completed the sale of $200 million aggregate principal amount of 4.5% Convertible Senior Notes due 2025, as well as retired and repaid $28.8 million of its 2033 Convertible Notes.

Cash, cash equivalents and marketable securities were $207.3 million as of March 31, 2019.
CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, May 7, 2019. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN: 330-863-3454
PASSCODE: 1547027
WEBCAST: View Source

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the completion of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 1547027. The replay can be accessed for a period of time on OPKO’s website at View Source.

On May 7, 2019 Oncoceutics reported that the US Food and Drug Administration invited the company to present information regarding its lead compound ONC201 at the public advisory committee meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee on June 20, 2019 (Press release, Oncoceutics, MAY 7, 2019, View Source [SID1234558355]).

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The meeting will provide Oncoceutics with the opportunity to present its clinical data for patients treated with ONC201, including studies for both adult and pediatric patients with gliomas containing the H3 K27M mutation, a disease that has been recognized to be an area of high unmet medical need. Additionally, the meeting will enable Oncoceutics to receive feedback from the subcommittee on issues concerning diseases to be studied, patient populations to be included, and possible study designs in the development of ONC201 for pediatric use.

"We are honored and grateful for the opportunity to present to the Pediatric Oncology Subcommittee of the ODAC," said Wolfgang Oster, MD, PhD, Chief Executive Officer and Chairman of Oncoceutics. "ONC201 has shown biological activity that we hope will translate into the clinic and eventually make a difference in patients’ lives. We look forward to furthering the clinical development of ONC201 with the support and guidance of the FDA."

The Public Advisory Committee Meeting will take place in the Great Room of the FDA’s White Oak campus from 8am to 4:30pm. Oncoceutics will present at 12:30pm. Background materials will be available at least 2 business days before the meeting at View Source, and interested persons from the public may present data, information, or views, orally or in writing, on issues pending before the subcommittee.

On May 7, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported it will release its unaudited financial results for the quarter ended March 31, 2019 on Tuesday, May 14, 2019, before the US markets open (Press release, BioLineRx, MAY 7, 2019, View Source;p=RssLanding&cat=news&id=2397480 [SID1234535796]).

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The Company will host a conference call on Tuesday, May 14, 2019 at 10:00 a.m. EDT featuring remarks by Philip Serlin, Chief Executive Officer. The conference call will be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

To dial into the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0644 internationally. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until May 16, 2019; please dial +1-888-782-4291 from the U.S. or +972-3-925-5925 internationally.