Sierra Oncology to Present at the Cowen 39th Annual Health Care Conference in Boston

On March 5, 2019 Sierra Oncology, Inc. (Nasdaq: SRRA), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that Dr. Christian Hassig, Chief Scientific Officer, will present an overview of the company at the Cowen 39th Annual Health Care Conference, scheduled for 10:00 a.m. ET on Wednesday, March 13 (Press release, Sierra Oncology, MAR 5, 2019, View Source [SID1234533995]). A live audio webcast and archive of the presentation will be accessible through the Sierra Oncology website at www.sierraoncology.com.

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VECT-HORUS and RadioMedix announce the signing of a LOI to co-develop a Vect-Horus’ radio-theranostic agent for Glioblastoma

On March 5, 2019 Vect-Horus and RadioMedix reported the signing of a Letter of Intent (LOI) to establish an agreement for the co-development of a Vect-Horus theranostic agent for the diagnostic and radiotherapy of Glioblastoma Multiforme (GBM) of the brain (Press release, Vect-Horus, MAR 5, 2019, View Source [SID1234534541]).

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This partnership will marry the expertise of Vect-Horus in targeting tumors with its technology VECTrans and the know-how of RadioMedix in developing and conducting pre-clinical evaluation and clinical trials with radiopharmaceuticals.

GBM is the most common and most aggressive malignant type of primary brain tumors and is a serious and life-threatening condition. The theranostic agent targets the Low-Density Lipoprotein Receptor (LDLR), which is highly expressed on many cancer cells, including glioblastoma. Positron Emission Tomography (PET) imaging and biodistribution studies in human glioblastoma xenograft and orthotopic models have shown a significant accumulation of the agent within the tumor.

"We are pleased that our technology, VECTrans in combination with expertise of RadioMedix will serve to advance an innovative and promising approach for glioblastoma treatment." said Alexandre Tokay, CEO of Vect-Horus "This co-development project is fully aligned with our strategy to expand the use of our technology in cancer indications with high unmet medical needs."

"We look forward to very compelling outcomes from our co-development project, as we seek to create new technologies for the benefit of cancer patients, by giving doctors and hospitals more imaging and treatment options." Said Dr Jamal Temsamani, Director of Drug Development of Vect-Horus.

"Development of a highly sensitive and effective theranostic approach for GBM has always been among our top priorities. Our initial evaluation of VECTrans technology appears to be extremely promising." said Dr Ebrahim Delpassand, CEO of RadioMedix. "Our plan is to utilize VECTrans technology to initially develop an accurate molecular diagnostic PET/CT probe to precisely map the tumor involvement in the brain. This will significantly improve precision of the surgical resection of the tumor. The treatment will then be followed by targeted alpha-emitter or beta-emitter radionuclide therapy using the same technology to eradicate any residual malignant cells at the molecular level" added Dr. Delpassand.

"RadioMedix has strong interests and all expertise needed to develop the VECTrans–based radio-theranostic agents for GBM", said Izabela Tworowska, PhD, CSO of RadioMedix. "Targeting LDLR can improve the blood -brain barrier (BBB) permeability of the drug and selectively deliver the isotope payload to GBM cancer cells."

Kura Oncology Reports Fourth Quarter and Full Year 2018 Financial Results

On March 5, 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 fourth quarter and full year 2018 financial results and provided a corporate update (Press release, Kura Oncology, MAR 5, 2019, View Source [SID1234533962]).

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"I am very pleased with the progress we have made over the past year, highlighted by the initiation of our registration-directed trial of tipifarnib in HRAS mutant head and neck squamous cell carcinoma (HNSCC)," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "In addition, we have made considerable strides toward broadening the clinical utility of tipifarnib, showing proof-of-concept in angioimmunoblastic T-cell lymphoma (AITL), validating CXCL12 as a therapeutic target in peripheral T-cell lymphoma (PTCL) and identifying a potential association between CXCL12 expression and clinical benefit in pancreatic cancer. Together, these efforts are helping us to expand the opportunity for tipifarnib beyond HRAS mutant solid tumors and into additional indications of high unmet need."

"As we begin 2019, we believe Kura is well positioned, with additional data from three ongoing Phase 2 trials of tipifarnib expected throughout the year and an emerging pipeline that includes KO-947, an ERK inhibitor with Phase 1 data expected later this year, and KO-539, a menin-MLL inhibitor that is anticipated to enter the clinic next quarter. We look forward to providing updates on each of our programs during the year as we continue to execute on our initial registration-directed trial."

Recent Highlights

New findings for tipifarnib in pancreatic cancer – In January 2019, Kura presented new findings at the 2019 Gastrointestinal Cancers Symposium identifying a potential association between CXCL12 expression and clinical benefit in patients with pancreatic cancer treated with tipifarnib. Elevated CXCL12 expression is known to be a poor prognostic factor in patients with certain solid tumors, including pancreatic cancer. The Company is currently working with key opinion leaders and investigators on the design of a proof-of-concept trial in this indication.

Validation of CXCL12 as a therapeutic target of tipifarnib in PTCL – In December 2018, Kura reported an association between CXCL12 expression and clinical benefit in the Phase 2 trial of tipifarnib in relapsed or refractory PTCL at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Specifically, patients with a high ratio of expression of CXCL12 to its receptor CXCR4 experienced a 50% objective response rate (five of 10 with either complete response or partial response) and a 90% clinical benefit rate (nine of 10, including stable disease) with tipifarnib. Patients in the Phase 2 trial were heavily pretreated, with a median of three prior lines of therapy (range 1-7).

Proof-of-concept in AITL – Kura also reported preliminary data from its Phase 2 clinical trial of tipifarnib in patients with relapsed or refractory PTCL at ASH (Free ASH Whitepaper). As of the November 21, 2018 data cutoff date, a total of 39 patients were enrolled in the trial, including 19 patients with AITL, an aggressive form of PTCL often characterized by high levels of CXCL12 expression. Of the 13 evaluable AITL patients, two achieved a complete response and four achieved a partial response, for an objective response rate of 46%, meeting the pre-specified primary efficacy endpoint for the AITL cohort.

New AITL patent strengthens intellectual property protection for tipifarnib – In November 2018, Kura announced the issuance of U.S. Patent No. 10,137,121, which includes multiple claims directed to the use of tipifarnib as a method of treating patients with AITL. The patent has an expiration date of November 2037, excluding any possible patent term extension. The AITL patent was issued just six months after a U.S. patent was issued for the use of tipifarnib as method of treating patients with certain CXCL12-expressing cancers, namely PTCL and acute myeloid leukemia (AML). Kura continues to pursue U.S. and foreign patent protection in these and other indications.

Continued progress in dose-escalation trial of KO-947 – KO-947 is a potent and selective small molecule inhibitor of extracellular signal related kinase (ERK), which Kura is advancing as a potential treatment for patients with tumors that have dysregulated activity due to mutations or other mechanisms in the mitogen-activated protein kinase (MAPK) pathway. The Company continues to evaluate a number of doses and schedules for KO-947 and anticipates having data from its Phase 1 clinical trial in 2019.

Investigational new drug (IND) cleared for KO-539 – KO-539 is a potent and selective small molecule inhibitor of the menin-mixed lineage leukemia (menin-MLL) protein-protein interaction. Kura has generated preclinical data that support the potential anti-tumor activity of KO-539 in genetically defined subsets of acute leukemia. The U.S. Food and Drug Administration (FDA) has cleared Kura’s IND application and the Company anticipates initiating a Phase 1 clinical trial of KO-539 in relapsed or refractory AML in the second quarter of 2019.
Financial Results

Research and development expenses for the fourth quarter of 2018 were $12.1 million, compared to $8.1 million for the fourth quarter of 2017. Research and development expenses for the full year 2018 were $46.8 million, compared to $26.4 million for the prior year.

General and administrative expenses for the fourth quarter of 2018 were $4.6 million, compared to $2.9 million for the fourth quarter of 2017. General and administrative expenses for the full year 2018 were $16.1 million, compared to $9.7 million for the prior year.

Net loss for the fourth quarter of 2018 was $16.1 million, compared to a net loss of $10.7 million for the fourth quarter of 2017. Net loss for the full year 2018 was $60.4 million, compared to a net loss of $35.4 million for the prior year. Net loss for the fourth quarter and full year of 2018 included non-cash, share-based compensation expense of $1.7 million and $8.7 million, respectively, compared to $1.4 million and $4.5 million for the same periods in 2017, respectively.

Cash, cash equivalents and short-term investments totaled $179.0 million as of December 31, 2018, compared with $93.1 million as of December 31, 2017.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its 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-high PTCL cohort, in mid-2019

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

Additional data from the ongoing Phase 2 trial of tipifarnib in chronic myelomonocytic leukemia (CMML) in 2019

Additional data on the molecular mechanisms of action of tipifarnib 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 the second quarter of 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, March 5, 2019, to discuss the financial results for the fourth quarter and full year 2018 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: 8996475. 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.

Iksuda Therapeutics and Femtogenix Sign License Agreement

On March 5, 2019 Iksuda Therapeutics (Iksuda), the next-generation Antibody Drug Conjugate (ADC) company, reported it has signed a licensing agreement with Femtogenix Limited (FGX), the next-generation ADC payload company (Press release, Iksuda Therapeutics, MAR 5, 2019, View Source [SID1234533978]). As part of this agreement, Iksuda will use FGX’s sequence-selective DNA-interactive payload molecules to progress its lead ADC towards the clinic, with the aim of targeting difficult-to-treat solid tumours.

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By harnessing FGX’s highly potent and broad-acting DNA mono-alkylating payloads in combination with its own PermaLink conjugation platform, Iksuda aims to significantly improve the therapeutic index of its ADCs and further advance the current standard of care for solid tumour types, which can be resistant to treatment. The agreement marks another key step in the build-out of Iksuda’s ADC technology-suite and drug pipeline, from which it aims to progress multiple candidates towards first clinical studies in 2020.

Dr Dave Simpson, Chief Executive Officer, Iksuda, said: "This agreement is an exciting progression of our ADC pipeline as it maximises potential for the greatest anti-cancer impact and enhanced therapeutic index, further underpinning our ambition to advance multiple ADCs to the clinic and treat the broadest patient population possible."

Dr Chris Keightley, Chief Executive Officer, Femtogenix, said: "We are pleased to be working with Iksuda to further validate the clinical potential of our payloads, which should provide improved efficacy and safety in comparison to those currently in the clinic. We have developed a wide range of easily conjugated payloads with novel mechanisms of action and potency levels, and these are available for licensing. In particular, we are developing payloads that can recognise and bind to transcription factor recognition site profiles within the genome. Such profiles are characteristic of specific tumour types, and this allows FGX to develop payloads with reduced toxicity and enhanced target specificity."

Puma Biotechnology to Present at Cowen’s Annual Health Care Conference

On March 5, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at 10:00 a.m. EDT on Tuesday, March 12 at the Cowen and Company 39th Annual Health Care Conference (Press release, Puma Biotechnology, MAR 5, 2019, https://investor.pumabiotechnology.com/press-release/puma-biotechnology-present-cowens-annual-health-care-conference [SID1234533996]). The conference will be held at the Boston Marriott Copley Place.

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A live webcast of the presentation will be available on the Company’s website at www.pumabiotechnology.com . The presentation will be archived on the website and available for 30 days