On November 16, 2017 Genmab A/S (Nasdaq Copenhagen: GEN) reported that it has achieved a USD 50 million sales volume milestone in its DARZALEX (daratumumab) collaboration with Janssen Biotech, Inc (Press release, Genmab, NOV 17, 2017, View Source [SID1234522123]). The milestone was triggered by confirmation by Janssen that sales of DARZALEX reached USD 1 billion in a calendar year. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize DARZALEX.

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"We continue to be pleased with the rapid uptake seen with DARZALEX since its initial launch and approval and are excited to have reached the USD 1 billion sales milestone," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The milestone was included in Genmab’s 2017 financial guidance published on November 14, 2017.

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On November 16, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that Dr. David C. Dale, MD will present the first clinical data from an on-going study of X4P-001-RD in patients with WHIM Syndrome, a sub-type of a primary immunodeficiency disease, at the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, X4 Pharmaceuticals, NOV 16, 2017, View Source [SID1234522146]). Preliminary data from these abstracts are available on the ASH (Free ASH Whitepaper) conference website. The details of the poster presentation is as follows:

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X4P-001: A Novel Molecularly-Targeted Oral Therapy for WHIM Syndrome

Saturday, December 9, 2017, Abstract # 995, Presenter: David C. Dale, MD

"We are very pleased to share preliminary data showing that X4P-001-RD has a meaningful impact on the levels of circulating white blood cells in this severely immunodeficient patient population," said Paula Ragan, PhD, President and CEO of X4. "These data demonstrate the potential of X4P-001-RD to benefit patients with WHIM who otherwise have no approved treatment options and support our goal of advancing toward a pivotal study."

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease ("PID") caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the US that are classified with PID of unknown origin — of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Because patients are highly susceptible to infections, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

About X4P-001-RD for Primary Genetic Immunodeficiency Disease

X4P-001-RD, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, is being developed for use as a life-long treatment for patients with WHIM syndrome and other primary genetic immunodeficiencies. X4P-001-RD is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome. Within the bone marrow, a normally functioning CXCR4 receptor controls the release of neutrophils and leukocytes into the blood stream, thereby ensuring normal immune surveillance functions throughout the body. In patients with WHIM syndrome, mutations to the CXCR4 receptor cause aberrant signaling leading to retention of neutrophils and leukocytes in the bone marrow and inadequate immune surveillance and function.4,5 X4P-001-RD is designed to normalize the signaling for the mutant CXCR4 receptor to promote the release of neutrophils and leukocytes, thereby restoring healthy immunity.

On November 16, 2016 TNK Therapeutics, Inc. ("TNK"), a subsidiary of Sorrento Therapeutics, Inc. (NASDAQ: SRNE; "Sorrento"), reported that it has entered into a binding term sheet to acquire Virttu Biologics Limited ("Virttu") (Press release, Sorrento Therapeutics, NOV 16, 2017, View Source [SID1234518745]).

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Virttu, based in Glasgow, Scotland, is a privately-held biopharmaceutical company focused on the development of oncolytic virus therapy for treatment of cancer. Virttu’s lead product candidate Seprehvir (HSV1716) is a Herpes Simplex Virus (HSV)-based oncolytic virus that selectively kills cancer cells and eliciting an anti-tumor immune response in the patient. The oncolytic virus technologies of Virttu have potential for broad therapeutic application across various cancer indications as well as being synergistic with the immunotherapy portfolios of TNK and Sorrento.

Seprehvir has completed Phase I studies in Europe for treatment of solid tumors, including glioma as well as head and neck squamous cell carcinoma (HNSCC), in adult cancer patients. In addition, it is currently being evaluated in a Phase I/IIa study in the UK for treatment of adult patients with mesothelioma using regional (intrapleural) delivery of Seprehvir and in a Phase I study in the US as therapy for non-CNS malignancies in pediatric patients using intravenous (i.v.) infusion of Seprehvir.

The acquisition is contingent upon completion of each parties’ due diligence and other customary closing conditions. In consideration for the acquisition, Virttu equity holders will receive $5 million in stock of Sorrento at closing (expected in the first quarter of 2017) and an additional $20 million in stock of TNK upon its next financing within 12 months after the closing.

"With the acquisition of Virttu, we will add a clinical-stage oncolytic virus therapy to our armamentarium of immunotherapies. The Virttu team has done a tremendous job of advancing Seprehvir into clinical trials for treatment of adult cancer and pediatric malignancies. In addition to the conventional intratumoral injection route that the marketed HSV-based oncolytic virus therapy utilizes, the Seprehvir can be administered via i.v. infusion for treatment of both local and metastatic cancers due to absence of inherent neurotoxicity in contrast to other HSV-based oncolytic viruses." said Dr. Henry Ji, President and CEO of Sorrento. Dr. Ji added, "We look forward to continuing the clinical development of Seprehvir in the US and Europe as monotherapy but also initiate combination trials globally with TNK’s cellular therapies as well as Sorrento’s immuno-oncology mAb products."

"We are excited to join the Sorrento/TNK team and together accelerate the development of Seprehvir. Sorrento’s antibody-centric therapies and TNK’s CAR-T and CAR.NK programs are a perfect match with our oncolytic virus as our preclinical research has shown significant synergy between Seprehvir and CAR-T therapies as well as with immune checkpoint antibodies. Furthermore, our Seprehvec platform technology will allow for the development of next generation oncolytic virus programs, such as expression of Sorrento’s immune checkpoint antibodies at the tumor site or pro-inflammatory cytokines for enhanced tumor killing and immune activation, by incorporating these genes into the Seprehvir genome," stated Dr. Joe Conner, Chief Scientific Officer of Virttu. "The field of oncolytic virus therapies is rapidly gaining attraction as evident by recent marketing approvals and transactions, so we believe that with Virttu becoming part of TNK, we will be well positioned to become a leader in this exciting immunotherapy space."

On November 16, 2017 Innovation Pharmaceuticals Inc. (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported additional perspectives on the planned continued development of Brilacidin-OM for the prevention and treatment of Oral Mucositis (OM) in Head and Neck Cancer (HNC) patients (Press release, Innovation Pharmaceuticals, NOV 16, 2017, View Source [SID1234522105]).

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As previously announced, the Phase 2 randomized, placebo-controlled clinical trial of Brilacidin-OM (see NCT02324335), enrolling a total of 61 patients, recently was completed, with top-line analysis to be reported shortly.

After unblinding, should final study results compare favorably to those observed at interim, in which patients treated with Brilacidin-OM showed a markedly reduced rate of severe OM (WHO Grade ≥3 ) compared to those on placebo, the Company will initiate an aggressive development plan going forward, including: applying to the Food and Drug Administration (FDA) for Breakthrough Therapy Designation and possibly applying for a similar expedited path in Europe; enhancing oral rinse administration with patient-friendly formulation and packaging; and, dedicating internal resources to Phase 3 trial design planning and execution.

Formal collaboration with pharmaceutical companies that have expressed an interest in partnering Brilacidin-OM may well assist further with expediting the drug candidate’s development timetable. Some of these partnering conversations have matured to the point of potentially structuring mutually beneficial licensing agreements, pending the final Phase 2 study results.

"We very much look forward to taking what could be the final step in Brilacidin’s development for the prevention of OM—top-line results reporting followed by possible advancement into a pivotal Phase 3 trial," said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "The OM market comprises a huge unmet medical need. To think we might one day help end the pain of so many patients suffering from OM, better enabling them to get the critical cancer care they need, would be an incredible accomplishment for Innovation Pharmaceuticals’ staff and a proud moment for our shareholders. Furthermore, securing partnerships with Pharma would represent a significant milestone for the Company, further anchoring the rapidly expanding Brilacidin Franchise."

"What’s important to understand about Brilacidin-OM, at this stage of its development, is that it truly represents a potential breakthrough treatment in OM," commented Arthur P. Bertolino, MD PhD, MBA, President and Chief Medical Officer at Innovation Pharmaceuticals. "A majority of OM treatments currently being evaluated in clinical trials still target reduction in duration of OM symptoms, rather than going after OM prevention—what we’ve established as our primary efficacy outcome in the completed Phase 2 trial. We’ve set for ourselves a high bar, to be sure, but feel confident that if we deliver strong top-line results, Brilacidin-OM will stand apart from any future competition—a potential first-to-market and best-in-class OM treatment."

About Oral Mucositis

Oral Mucositis (OM) is a frequent, painful and debilitating complication of chemoradiation. Head and Neck Cancer (HNC) patients—comprising an estimated 65,000 newly diagnosed cases in the U.S. alone in 2017, and an estimated 700,000 worldwide (source: GLOBOCAN)—are at the greatest risk of developing OM (a 90 to 100 percent rate of occurrence). By 2030, the global incidence of HNC cases is expected to exceed 1 million per year. Moreover, between 25 and 60 percent of cancer patients, regardless of cancer type, also will experience OM. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak and eat (requiring the insertion of a feeding tube) and are more susceptible to infections, with severe cases leading to hospitalization at increased treatment costs of up to $25,000. There currently are no approved medications for the prevention of OM in the HNC population, with only limited palliative care options available. Worldwide, the potential market for OM is expected to exceed $1 billion in the next few years.