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Genmab 2018 Annual Report

On February 20, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported its Annual Report for 2018 (Press release, Genmab, FEB 20, 2019, View Source [SID1234533481]). Below is a summary of business progress and financial performance for the year, and financial outlook for 2019 from the report. The full report is attached as a PDF file and can be found on the investor section of the company’s website, www.genmab.com. An online summary of the report is available at View Source

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2018 ACHIEVEMENTS

Business Progress

Maximize daratumumab progress

FDA and EMA decision on Phase III ALCYONE multiple myeloma (MM) submission – Achieved
Start new Phase III MM study – Achieved
Report early clinical data in solid tumors – Not achieved
Phase III MAIA MM efficacy analysis in frontline – Achieved
Phase III CASSIOPEIA MM efficacy analysis in frontline – Achieved

Optimize ofatumumab value

Complete recruitment Phase III subcutaneous ofatumumab relapsing MS studies – Achieved
Maximize tisotumab vedotin progress

Start two Phase II studies in cervical cancer (recurrent / metastatic & combination study in frontline) – One Phase II study with tisotumab vedotin in cervical cancer was started in 2018. A Phase I/II study in cervical cancer was posted on www.clinicaltrials.gov in 2018, but had not started before year end.
Start Phase II study in additional solid tumor indications – Achieved
Strengthen differentiated product pipeline and technology partnership portfolio

Start HuMax-AXL-ADC expansion phase in ongoing Phase I/II study – Achieved
Progress HexaBody-DR5/DR5 Phase I/II study – Achieved
Progress DuoBody-CD3xCD20 Phase I/II study – Achieved
Accelerate proprietary Immuno-Oncology DuoBody programs towards clinic – Achieved
Enter new technology or product collaborations – Genmab entered one new technology collaboration, with Immatics, during 2018.

Disciplined financial management and building a commercial footprint

Execute controlled company growth with selective investments in product & technology pipeline – Achieved
Continue investing in building commercialization and launch capabilities
Financial Performance

Revenue was DKK 3,025 million in 2018 compared to DKK 2,365 million in 2017. The increase of DKK 660 million, or 28%, was mainly driven by higher DARZALEX royalties under our daratumumab collaboration with Janssen, the payment from Novartis of USD 50 million (DKK 304 million) and reimbursement income from our collaborations with Seattle Genetics and BioNTech, partly offset by a decrease in DARZALEX milestones.
Operating expenses increased by DKK 624 million, or 61%, from DKK 1,021 million in 2017 to DKK 1,645 million in 2018 driven by the advancement of tisotumab vedotin, additional investments in our product pipeline, and the increase in employees to support the expansion of our pipeline.
Operating income was DKK 1,380 million in 2018 compared to DKK 1,344 million in 2017. The improvement of DKK 36 million, or 3%, was driven by higher revenue, which was mostly offset by increased operating expenses.
2018 year end cash position of DKK 6,106 million, an increase of DKK 683 million, or 13%, from DKK 5,423 million as of December 31, 2017.

We expect our 2019 revenue to be approximately DKK 4,600 million, compared to DKK 3,025 million in 2018, an increase of DKK 1,575 million or 52%. Our projected revenue for 2019 primarily consists of DARZALEX royalties of DKK 2,685 million, based on estimated net sales of USD 3.0 billion. We project DARZALEX milestones of approximately DKK 1,500 million related to commercial net-sales based milestones for achieving net-sales in a calendar year of both USD 2.5 billion and USD 3.0 billion respectively. The remainder of the revenue consists of cost reimbursement income, Arzerra royalties, and DuoBody milestones.

Operating Expenses
We anticipate that our 2019 operating expenses will be approximately DKK 2,600 million, an increase of DKK 955 million or 58% compared to 2018. The increase is driven by the advancement of our clinical programs, particularly tisotumab vedotin and enapotamab vedotin.

Operating Result
We expect the operating income to be approximately DKK 2,000 million in 2019 compared to DKK 1,380 million in 2018, an increase of DKK 620 million or 45%.

More information on the Risks and Assumptions for the 2019 Financial Guidance can be found in the 2018 Annual Report available on our website www.genmab.com.

Conference Call
Genmab will hold a conference call in English to discuss the results for the full year results for 2018 today, February 20, 2019 at 6.00 pm CET, 5.00 pm GMT or noon EST. To join the call dial +1 866 966 1396 (US participants) or +44 2071 928000 (international participants) and provide conference code 8793105.

Mustang Bio and Nationwide Children’s Hospital Enter into Exclusive Worldwide License Agreement for Oncolytic Virus (C134) to Treat Glioblastoma Multiforme

On February 20, 2019 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR T) technology and gene therapies for rare diseases, and Nationwide Children’s Hospital ("Nationwide"), one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease, reported that they have partnered and entered into an exclusive worldwide license agreement to develop an oncolytic virus (C134) for the treatment of glioblastoma multiforme (Press release, Mustang Bio, FEB 20, 2019, View Source [SID1234533480]).

A Phase 1 clinical trial evaluating C134, an attenuated herpes simplex virus type 1 (HSV-1), in recurrent glioblastoma multiforme is being conducted at the University of Alabama at Birmingham ("UAB"). The trial is led by Dr. James Markert, M.D., Chairman of the Department of Neurosurgery at UAB, who developed C134 in collaboration with Dr. Kevin Cassady, M.D., Associate Professor of Pediatrics at Nationwide Children’s Hospital. C134 is a second-generation HSV-1 oncolytic virus that has improved replication in tumors in murine models, but with the same toxicity profile as its first-generation predecessors. In these preclinical studies, it not only demonstrates direct anti-tumor activity, but also elicits an immune response that can reverse tumor-associated immunosuppression.

Subsequent clinical trials will investigate a combination treatment of MB-101 (IL13Rα2-specific CAR) and C134. These trials are supported by preclinical studies that have appeared to demonstrate the synergistic potential of an oncolytic virus, which can induce an anti-tumor immune response when combined with CAR-T therapy to target solid tumors.

"Oncolytic viruses often trigger an immune response directed at tumors that are otherwise refractory to single agent immunotherapies. Our oncolytic virus C134 has demonstrated promising preclinical activity and we look forward to working with Mustang to advance its development in the clinic," Dr. Kevin Cassady said.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are very pleased to partner with Nationwide Children’s Hospital to develop oncolytic virus C134. We also plan to evaluate oncolytic virus C134 in combination with MB-101 to explore the potential synergies of this novel combination to treat patients with glioblastoma. At Mustang, we are committed to evaluating our CAR T therapies alone and in combination regimens with the goal of advancing treatment paradigms for cancers without alternate therapies."

DelMar Pharmaceuticals Achieves Halfway Enrollment Point for Phase 2 Clinical Trial of VAL-083 in Newly Diagnosed MGMT-unmethylated GBM

On February 20, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that its Phase 2 study testing VAL-083 in patients with newly diagnosed glioblastoma multiforme (GBM) has achieved its halfway enrollment point (Press release, DelMar Pharmaceuticals, FEB 20, 2019, View Source [SID1234533467]). This trial, targeted to enroll up to thirty patients, is a single-arm, open-label study testing VAL-083 in combination with standard radiotherapy in GBM patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene. An estimated 60% of GBM patients possess an unmethylated MGMT gene, which confers a more limited response to current standard of care treatment as well as a lower survival probability. This clinical trial was initiated in February 2017 and is being conducted at the Sun Yat-sen University Cancer Center (SYSUCC) in Guangzhou, China in collaboration with Guangxi Wuzhou Pharmaceutical Company.

The Company is pleased to report that for the 15 patients enrolled to date, 11 have completed their prospectively planned Magnetic Resonance Imaging (MRI) scans and have had their initial assessment for tumor progression. Tumor progression is based on the trial investigator’s clinical and radiologic assessment, according to the Response Assessment in NeuroOncology (RANO) criteria. Of these 11 patients, five were assessed by the Principal Investigator as having a "Complete Response", three of whom were based on significant tumor shrinkage, and two of whom were based on their tumors continuing to remain "below measurable level" from post-surgery baseline MRI to post-cycle 3 MRI. Additionally, six patients were assessed as having "Stable Disease." Of the remaining four patients, one died prior to their post-cycle 3 MRI and three have not been on study long enough to reach their planned post-cycle 3 MRI. As of the February 14, 2019 data cutoff, 12 of the 15 enrolled patients are still alive. Similar to prior experience, myelosuppression has been the most common adverse event observed. Two dose-limiting toxicities have been reported (thrombocytopenia) – one at the 40 mg/m2/day dose and one at the 30 mg/m2/day dose.

"GBM is a cancer with a very high unmet medical need, especially for patients with an MGMT-unmethylated biomarker who are provided with limited existing treatment options," stated Saiid Zarrabian, President and Chief Executive Officer of DelMar Pharmaceuticals. "We are encouraged that we have completed the dose escalation stage of this study and that 30 mg/m2/day of VAL-083 in combination with radiation therapy was generally safe and well-tolerated in this trial. And while these results are preliminary, we are also enthusiastic that five of the first twelve patients available for efficacy measurements have initially been assessed as having a Complete Response. With the clinical trial rapidly enrolling into its expansion phase, we look forward to providing a final data update once the study is completed."

The Company will be providing further details and an update on this trial at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) being held March 29 to April 3, 2019.

Professor Zhong-ping Chen, Founder Chairman of the Department of Neurosurgery/Neuro-oncology at Sun Yat-sen University Cancer Center, and who is also the study’s Principal Investigator, stated that "treating glioblastoma patients with an unmethylated MGMT promoter is particularly challenging. While the clinical trial is still early, and we are only at the halfway point in enrollment, we are highly encouraged at the enhanced levels of tumor shrinkage and the complete responses we are observing after treatment with VAL-083 in combination with radiation. This preliminary data appears to support the premise that VAL-083 has the potential to provide a valuable treatment option for these patients."

The clinical trial in newly diagnosed GBM is designed to enroll up to 30 patients to determine if first-line treatment with VAL-083 plus radiotherapy can provide improvements over the historical efficacy of standard of care temozolomide (TMZ) plus radiotherapy. Efficacy will be measured based on tumor response to treatment, progression-free survival, progression-free survival at six months, and overall survival compared to historical results in the target population.

The clinical trial consists of two parts:

Part 1 is a dose-escalation and induction format to confirm the recommended dose of VAL-083 when administered concurrently with radiation therapy based on safety and tolerability. The patients received VAL-083 at 20 mg/m2/day, 30 mg/m2/day or 40 mg/m2/day along with standard radiation treatment. The dose escalation phase of the study was concluded in October 2018.
Part 2 comprises an expansion phase whereby VAL-083 will be studied in up to 20 additional patients. Based on the best balance of efficacy and tolerability, the dose of VAL-083 chosen for the expansion phase of the study was 30 mg/m2/day. This phase of the study is ongoing and is continuing to enroll patients.
This phase 2 clinical trial (ClinicalTrials.gov Identifier: NCT03050736) is expected to provide the scientific basis for larger studies to support submission of marketing applications. Ideally, data from these larger studies will result in approval of VAL-083 as first line therapy for all newly-diagnosed patients with an unmethylated MGMT gene promoter.

About VAL-083

VAL-083 (Dianhydrogalactitol) is a novel bi-functional DNA targeting agent that rapidly induces interstrand cross-links at N7-guanine, leading to DNA double-strand breaks and ultimately cell death. VAL-083’s unique cytotoxic mechanism circumvents MGMT mediated chemoresistance and differentiates it from other therapies used in the treatment of GBM, including TMZ. This makes VAL-083 an ideal candidate to explore treating patients who are unlikely to respond to TMZ due to MGMT expression in their GBM as per the 2017 National Comprehensive Cancer Network guidelines.

VAL-083 has been granted orphan drug designations by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 has been granted fast-track status for the treatment of recurrent GBM by the US FDA.

ZETAGEN THERAPEUTICS, INC. RECEIVES PATENTS FOR METHODS IN STIMULATING BONE GROWTH USING SMALL-MOLECULE

On February 19, 2019 Zetagen Therapeutics, Inc., a private, US-based biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and orthopedic interventions, reported that the U.S. Patent and Trademark Office ("USPTO") has issued U.S. Patent No. 10208306 and the South African Patent Office issued PCT. 2017/00029 to the Company (Press release, Zetagen Therapeutics, FEB 19, 2019, View Source [SID1234643687]). These patents, entitled Composition and Methods to Promote Bone Formation, include claims covering the use of methods for stimulating bone growth using its novel molecular pathway, ZetaMet (Zeta-BC-003). ZetaMet (Zeta-BC-003) is a synthetic, small molecule, inductive biologic, delivered via a drug-eluting implant on an osteopromotive carrier.

"These new patent issuances are an important step forward towards our ultimate goal of obtaining the global, commercial rights for our treatments," said Joe C. Loy, CEO of Zetagen Therapeutics, Inc. "We believe in the potential our therapies hold for patients around the world who live with debilitating metastatic bone cancers."

The new patent is part of an expanding and comprehensive portfolio of patents, patent applications and other intellectual property covering the composition, synthesis, manufacturing, formulations and uses for the treatment of a variety of metastatic bone lesions and osteologic interventions. Zetagen exclusively licensed its platform technology from the State University of New York in 2016.

Apexigen to present at upcoming Investor Conferences

On February 19, 2019 Apexigen, Inc., a clinical-stage biopharmaceutical company, reported that Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer, will present at the following upcoming investor conferences (Press release, Apexigen, FEB 19, 2019, View Source [SID1234590998]):

Citi’s 7th Annual Immuno-oncology Leadership Summit
New York, NY
Tuesday, February 19, 2019 at 3:30 pm ET

SVB Leerink’s 8th Annual Global Healthcare Conference
New York, NY
Wednesday, February 27, 2019 at 2:10 pm ET

Cowen’s 39th Annual Healthcare Conference
Boston, MA
Tuesday, March 12, 2019 at 4:00 pm ET

William Blair’s IO Conference
New York, NY
Thursday, March 21, 2019 at 2:05 pm ET