On December 18, 2018 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported its financial outlook for 2019 (Press release, Ligand, DEC 18, 2018, View Source [SID1234532150]). Ligand expects revenue in 2019 to be at least $212 million, with up to an additional $40 million of potential milestone and license payments. Approximately two thirds of the $212 million of revenue is expected to be royalty revenue, and the remainder is expected to consist of contract payments and material sales. With projected revenue of $212 million, adjusted earnings per diluted share for 2019 is estimated to be approximately $5.50. So far during the fourth quarter of 2018, Ligand has repurchased over 420,000 shares under its authorized share repurchase plan, and will potentially continue to execute additional repurchases going forward.

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Ligand is also raising its previous guidance for 2018, mostly due to higher material sales, and now expects revenue to be approximately $244 million, including royalties of approximately $123 million, material sales of approximately $28 million and license fees and milestones of approximately $93 million. Revenue for 2018 includes a milestone payment of $47 million from WuXi Biologics. With projected revenue of approximately $244 million, adjusted earnings per diluted share for 2018 is estimated to be approximately $6.63. This compares with previous guidance for 2018 revenue of approximately $240 million, and adjusted earnings per diluted share for 2018 which was previously estimated to be $6.52.

On December 18, 2018 Oncology Venture A/S ("OV" or the Company) reported that the US Food and Drug Administration, FDA has responded positively on questions posed by the company in a Pre-IND/IDE package for the approval pathway for LiPlaCis and its companion diagnostic DRP – Drug Response Predictor – in metastatic breast cancer (Press release, Oncology Venture, DEC 18, 2018, View Source [SID1234532169]).

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The FDA agreed that the 505(b)(2) pathway is an acceptable registration route for LiPlaCis and that no further toxicology studies are needed. Based on current good data the number of patients to be treated is in line with previous guidance in the upcoming pivotal phase 3 trial of LiPlaCis and its DRPÒ for the treatment of patients with advanced breast cancer.
The FDA accepts objective response rate (ORR) as the primary endpoint but asked for further characterization of sub-groups in the breast cancer population aimed for treatment with LiPlaCis.
LiPlaCis is an intelligent, target controlled liposome formulation of one of the world’s most widely used chemotherapies, cisplatin. The specific LiPlaCis formulation allows delivery of LiPlaCis directly at tumor site. The specific LiPlaCis DRP selects the patients whom are expected to benefit from the treatment. LiPlaCis has shown very promising results in an ongoing phase 2 trial, a study that will continue as planned. Recruitment timelines of the pivotal phase 3 study will be updated following the FDA approval of the Investigational New Drug Application (IND) and the Investigational Device Exemption (IDE) expectedly in H1 2019.

"Oncology Venture in-licensed LiPlaCis as a phase 1 project in 2016. The 505(b)(2) strategy allows us to refer to data for a listed drug and will save us important time and resources. Our team has done a remarkable job by moving this project from an early stage to a late stage project in only two years. The discussions with the FDA gave no barrier for proceeding with our pivotal development plans for a fast route to commercialization and we can now increase our partnering activities with pharma," comments Peter Buhl Jensen, M.D., CEO of Oncology Venture.

Data from the ongoing Phase 2 LiPlaCis study in patients with metastatic breast cancer shows a
50% objective response rate (five out of ten patients) in the upper one third of DRP selected patients and a 24% objective response rate (6 out of 25 patients) in the upper two thirds of DRP selected patients. These data should be compared with historical response rates to the established cancer drugs in metastatic breast cancer with a 10-12% objective response rate of eribulin, vinorelbine and gemcitabine and 10% of conventional cisplatin.

If the ongoing phase 2 study will continue to show strong efficacy data, Oncology Venture aims for a Break through designation. This application is planned for filing shortly after the IND and its IDE application for LiPlaCis.

Oncology Venture’s regulatory strategy is to first obtain approval in the US. The aim is then to run randomized pivotal studies in Europe and Asia.

On December 18, 2018 CTI BioPharma Corp. (NASDAQ:CTIC) reported that it has received input from the U.S. Food and Drug Administration (FDA) at a recent Type C meeting on key elements of the design of a new randomized Phase 3 study of pacritinib in adult patients with myelofibrosis (primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis) and who have severe thrombocytopenia (as defined by patients with platelet counts of less than 50,000 per microliter), an indication that has been recognized by the medical community as an important unmet medical need (Press release, CTI BioPharma, DEC 18, 2018, View Source [SID1234532119]).

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The planned Phase 3 study is designed to evaluate the effects of pacritinib as compared to physician’s choice of treatment. The primary efficacy endpoint will be the proportion of patients achieving a greater than or equal to 35% spleen volume reduction (SVR) between baseline and Week 24. Secondary efficacy endpoints of the study include total symptom score reduction and overall survival.

Before commencing the Phase 3 study, CTI plans to meet with the FDA to discuss the final optimal dose analysis from the PAC203 Phase 2 study. To expedite the transition to Phase 3, CTI intends to amend the PAC203 protocol to include a Phase 3 component. The PAC203 Phase 3 component is designed to enroll approximately 200 patients with enrollment expected to commence in the third quarter of 2019. The anticipated cost of the Phase 3 study is approximately $25 million. Taking into account the impact of recently-announced cost saving efforts and the anticipated cost of the new Phase 3 trial, the current CTI financial analysis projects a cash runway that extends into 2020.

On December 17, 2018 RhoVac AB ("RhoVac") reported that the first prostate cancer patient included in the clinical phase I / II study with RhoVac’s drug candidate RV001 has completed it’s 12-month follow-up and thus the entire clinical phase I / II- study (Press release, RhoVac, DEC 17, 2018, https://www.rhovac.com/rhovac-announces-that-the-first-patient-completed-the-follow-up-phase-of-the-clinical-phase-i-ii-study/ [SID1234532083]).

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Prior to the clinical phase I / II study, conducted between 2017 and 2018, 22 patients with diagnosed prostate cancer and prostatectomized. Blood samples were taken before, during and after the study to analyse the product mediated immune response to treatments. The results, reported earlier this year, showed a significant immune response in 86% of the patients. Patients have also participated in a 3-, 6-, 9- and 12-month follow-up phase of the study, to assess the duration of the immune response.

RhoVac announces that the first patient has completed his 12-month follow-up and thus also completed his participation in RhoVac’s phase I / II clinical study. The samples for immunological analysis have been sent to the University of Tübingen. The interim report on the duration of the immunological responses, from all participating patients’ 3- and 6-month follow-up will be published in January 2019.

The last patient is expected to complete his 12-month follow-up in March 2019. The final report on the duration of the immunological responses is expected in the second quarter of 2019.

The company’s drug candidate RV001 activates T cells of the immune system to identify and eliminate cancer cells expressing the protein RhoC. As RhoC is generally overexpressed in metastatic cancer cells, RV001 can potentially be used in the treatment of several different cancer indications. It is the company’s ambition that the product being developed shall be used as a preventive treatment against metastases, thus preventing, or limiting the spread and recurrence of cancer

On December 17, 2018 Kineta Immuno-Oncology, LLC (KIO), a subsidiary of Kineta, Inc., reported that it has entered into a strategic research collaboration with Pfizer Inc. (NYSE: PFE) to develop RIG-I agonist immunotherapies for the treatment of cancer (Press release, Kineta, DEC 17, 2018, View Source [SID1234532100]). The research collaboration and license agreement grants to Pfizer the exclusive rights to KIO’s RIG-I screening platform and related compounds and technologies. The companies will collaborate to develop and test small molecule agonists that target RIG-I, an innate immunostimulatory pathway that can elicit immunogenic cell death (ICD) in tumors, providing both direct tumor cell killing and enhanced anti-tumor immune responses.

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In preclinical models, Kineta’s RIG-I agonists have demonstrated complete tumor regression and an increase in tumor-specific T cells when given as a monotherapy. Additionally, the compounds have demonstrated synergistic effects when used in combination with other immunotherapies like checkpoint inhibitors. Kineta’s proprietary approach is part of an internally developed discovery program focused on innate immune drug targets in cancer. The lead program is focused on the RIG-I pathway, which is differentiated from other innate immune-targeted immunotherapies as its compounds are small molecule drugs with the potential for oral and systemic administration.

"This alliance will enable Kineta and Pfizer to leverage each company’s expertise to accelerate the development of our RIG-I immuno-oncology program," said Kineta CEO, Shawn Iadonato. "Pfizer is an excellent partner for Kineta’s technology, with a strong commitment to oncology and outstanding development capabilities. We are very enthusiastic about this new cancer immunotherapy collaboration, which represents the second major partnering transaction for Kineta this year."

Dr. Robert Abraham, Senior Vice President and Group Head of Pfizer’s Oncology Research & Development Group said: "Therapies that trigger activation of the innate immune response in tumors have significant potential to expand the number of patients who will benefit from cancer immunotherapy, especially if they can be administered systemically. Kineta has taken a promising, differentiated approach to the discovery of RIG-I agonists, and we are looking forward to collaborating with Kineta and jointly developing these RIG-I-targeted agents into medicines with potentially unique immune-stimulating properties."

Under the terms of the agreement, KIO will receive a $15 million upfront payment and will be eligible to receive up to $505 million in potential research, development and sales milestone payments. Additionally, KIO is eligible to receive tiered royalties on net sales. Pfizer will fund RIG-I target-related research conducted by Kineta for an initial period of three years, after which Pfizer will be responsible for further development and commercialization of product candidates.

Torreya acted as exclusive financial advisor to Kineta on this transaction.