On August 2, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported that the European Patent Office (EPO) and Japan Patent Office (JPO) have granted patents for the Company’s novel self-delivering RNAi (sd-rxRNA) therapeutic platform (Press release, RXi Pharmaceuticals, AUG 2, 2018, View Source [SID1234528839]). The EPO Patent #: 2949752 B1 and JPO Patent #: 620309 cover composition of matter, specifically structural and chemical attributes of sd-rxRNA. These patents will be set to expire in 2029.

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"We are pleased that both the European and Japan Patent offices have recognized our novel therapeutic platform with the granting of these patents," said Dr. James Cardia, Vice President of Business Operations at RXi Pharmaceuticals. He further added, "The Company recently reported the successful outcomes from both our dermatology and ophthalmology clinical trials where our lead candidate RXI-109, an sd-rxRNA therapeutic compound targeting connective tissue growth factor (CTGF), is being evaluated. We announced earlier this year that we would exclusively focus on developing the next generation of immuno-oncology therapeutics based on our self-delivering RNAi therapeutic platform. As such, we are actively seeking to partner or out-license both the Dermatology and Ophthalmology Franchises. The granting of these patents further provides for multiple commercial and development opportunities with RXI-109 and RXi’s sd-rxRNA technology platform, each broadly protected in the United States and International regions."

On August 2, 2018 Genprex, Inc. (NASDAQ:GNPX), a clinical stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology platform, reported that it has amended its agreement with The University of Texas MD Anderson Cancer Center to resume patient enrollment in its Phase I/II clinical trial evaluating the combination of the company’s investigational drug Oncoprex and erlotinib (Tarceva) for the treatment of Stage IV non-small cell lung cancer (NSCLC) (Press release, Genprex, AUG 2, 2018, View Source [SID1234529159]).

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Oncoprex is a TUSC2 gene encapsulated in a positively charged nanovesicle made from lipid molecules and injected intravenously, which can specifically target cancer cells and insert wild-type TUSC2 into cellular DNA, effectively increasing expression of the TUSC2 protein and promoting tumor cell death.

Previously announced interim data from nine patients from the Phase II portion of this Phase I/II clinical trial showed a disease control rate of 78%, with seven out of nine patients achieving stable disease or better, including one complete response. In a previous Phase I clinical trial at MD Anderson evaluating Oncoprex as a monotherapy, five of 23 patients with late-stage NSCLC achieved stable disease or better, with one durable metabolic response.

"We look forward to completing the Oncoprex/erlotinib trial and expanding the study of Oncoprex in combination with other targeted and immunotherapies in the future," said Rodney Varner, Chairman and Chief Executive Officer of Genprex. "We believe the data from the more than 50 late-stage NSCLC patients treated to date provide persuasive evidence of Oncoprex’s anti-tumor effects and favorable safety profile."

A subset of NSCLC patients (approximately 10% of NSCLC patients of North American and European descent and approximately 30% to 50% of NSCLC patients of Asian descent) carry an EGFR mutation that makes their tumors sensitive to tyrosine kinase inhibitors, or TKIs, such as erlotinib. However, even for these patients, tumor resistance to TKIs frequently develops within two years, resulting in eventual disease progression. While next generation TKIs show promise in targeting resistant EGFR positive tumors that carry a mutation known as T790M, only about one-half of EGFR positive patients (5% to 7.5% of all NSCLC patients of North American and European descent and 15% to 25% of NSCLC patients of Asian descent) carry the T790M mutation. This leaves a significant majority of NSCLC patients—those who are EGFR negative and those who are EGFR positive but have become resistant to erlotinib and do not have the T790M mutation—without a targeted therapy for their cancer.

Combination therapies targeting multiple anti-cancer pathways represent a promising approach to achieving greater response rates, and may also allow the expanded use of targeted therapies and immunotherapies in a larger population of cancer patients who are not currently candidates for these treatments.

About Lung Cancer

According to the World Health Organization, lung cancer is the leading cause of cancer deaths worldwide, and is the second most common type of cancer. Each year, there are over 1.8 million new lung cancer cases and 1.6 million deaths from lung cancer worldwide, and in the United States there are over 225,000 new cases and more than 150,000 deaths from lung cancer per year. NSCLC represents 80% of all lung cancers. According to a 2016 American Cancer Society report, the five-year survival rate for Stage IV (metastatic) NSCLC is about 1%, and overall survival for lung cancer has not improved appreciably in the last 25 years.

On August 2, 2018 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development and commercialization of innovative therapeutics for the treatment of cancer, reported its financial results for the second quarter ended June 30, 2018 (Press release, Curis, AUG 2, 2018, View Source [SID1234528309]).

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"Our second quarter has been marked by continued progress in advancing our three clinical drug candidates further in their respective development," said Ali Fattaey, Ph.D., Chief Executive Officer of Curis. "We continue to assess and hold productive discussions with the FDA to identify a path to register fimepinostat, which could provide much-needed benefit for patients with R/R DLBCL, and in particular those whose disease have MYC alterations. CA-170, our orally available small-molecule checkpoint inhibitor, continues to progress in Phase 1 and Phase 2 clinical studies with a planned update in the second half of 2018. Patient enrollment continues for our recently initiated Phase 1 study of precision oncology candidate CA-4948, currently the only IRAK4 kinase inhibitor in clinical development for cancer. In addition, we recently welcomed Dr. Robert Martell into his new role as Head of Research and Development. Dr. Martell is a practicing oncologist whose extensive experience in drug development will help strengthen Curis’s oncology portfolio. We believe Curis remains on track to advancing multiple attractive candidates with potential to substantially impact current oncology care."

Second Quarter 2018 Financial Results

Curis reported a net loss of $8.7 million, or $0.26 per share on both a basic and diluted basis for the second quarter of 2018, as compared to a net loss of $14.1 million, or $0.49 per share on both a basic and diluted basis for the same period in 2017. Curis reported a net loss of $19.4 million, or $0.59 per share, on both a basic and diluted basis for the six months ended June 30, 2018, as compared to a net loss of $29.8 million, or $1.04 per share on both a basic and diluted basis for the same period in 2017.

Revenues for the second quarter of 2018 were $2.4 million, as compared to $2.1 million for the same period in 2017. Revenues for the six months ended June 30, 2018 were $4.8 million, as compared to $4.2 million for the same period in 2017. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $10.2 million for the second quarter of 2018, as compared to $15.2 million for the same period in 2017. Operating expenses for the six months ended June 30, 2018 were $22.6 million, as compared to $32.4 million for the same period in 2017, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, resulting from payments to third-party university patent licensors associated with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the second quarter of 2018 and 2017. Cost of royalty revenues for the six months ended June 30, 2018 were $0.3 million, as compared to $0.2 million for the same period in 2017.

Research and Development Expenses. Research and development expenses were $6.5 million for the second quarter of 2018, as compared to $11.3 million for the same period in 2017. The decrease was primarily driven by decreased costs related to clinical activities for fimepinostat and CA-170, partially offset by increased costs related to CA-4948. Research and development expenses were $14.7 million for the six months ended June 30, 2018 as compared to $24.8 million for the same period in 2017.

General and Administrative Expenses. General and administrative expenses were $3.6 million for the second quarter of 2018 as compared to $3.8 million for the same period in 2017. The decrease in general and administrative expenses was primarily driven by lower personnel and stock-based compensation expense partially offset by higher legal services for the period. General and administrative expenses were $7.6 million for the six months ended June 30, 2018, as compared to $7.4 million for the same period in 2017.

Other Expenses. Net other expense for the second quarter 2018 totaled $0.8 million as compared to $1.0 million for the same period in 2017. Net other expense primarily consisted of interest expense related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations. Other expense, net was $1.6 million and $1.7 million for the six months ended June 30, 2018 and 2017, respectively.

As of June 30, 2018, Curis’s cash, cash equivalents, marketable securities and investments totaled $40.4 million and there were approximately 33.2 million shares of common stock outstanding.

Recent Operational Highlights

Precision oncology, fimepinostat (formerly CUDC-907):

Fimepinostat received Fast Track designation from the FDA for development in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy.
Immuno-oncology, CA-170 (PDL1 / VISTA antagonist; Aurigene collaboration):

Patient enrollment remained on track for the Phase 1 dose escalation trial evaluating CA-170 in patients with advanced solid tumors or lymphomas.
Curis collaborator Aurigene continued to enroll patients in a Phase 2 clinical study of CA-170 at select trial sites in India.
Investigators have recently identified that mesothelioma tumor samples express high levels of VISTA immune checkpoint protein. We are extending our current Phase 1 trial to enroll a cohort of patients with mesothelioma.
Precision oncology, CA-4948 (IRAK4 Kinase Inhibitor; Aurigene collaboration):

Curis continued to enroll patients with relapsed or refractory non-Hodgkin lymphoma in a Phase 1 clinical trial evaluating CA-4948, a novel oral, small molecule IRAK4 kinase Inhibitor that has shown potent in vivo anti-tumor activity in preclinical animal models.
We have recently generated non-clinical data that demonstrate CA-4948 is active in in vivo models of AML. We are extending our current Phase 1 clinical trial to enroll a cohort of patients with AML.
Recent Corporate Highlights

Robert Martell, M.D., Ph.D., a practicing oncologist and experienced drug developer, was appointed Head of Research and Development and will directly manage the day-to-day operations of clinical development and research.
Curis announced a 1-for-5 reverse stock split reducing the number of Curis’s outstanding common stock from approximately 165.6 million to approximately 33.1 million on March 29, 2018, the date of the reverse split.
Upcoming Activities

Curis anticipates providing an outline of the clinical development path for fimepinostat in 2H 2018.
Curis and collaborator Aurigene expect to provide additional updates in 2H 2018 on the clinical progress of CA-170, which is currently being evaluated in one Phase 1 and one Phase 2 study.
Curis expects to provide an update in 2H 2018 on the progress of CA-4948, which is currently being evaluated in a Phase 1 trial in patients with advanced lymphomas.
Conference Call Information

Curis management will host a conference call today, August 2, 2018, at 8:30 a.m. EDT, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 (United States) or 1-412-317-5252 (International), shortly before 8:30 a.m. EDT. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section. A replay of the call will be available on the Curis website shortly after the commencement of the meeting.

On August 2, 2018 OPKO Health, Inc.(NASDAQ: OPK) reported its operating and financial results for the three and six months ended June 30, 2018 after the close of the U.S. financial markets on Tuesday, August 7, 2018 (Press release, Opko Health, AUG 2, 2018, View Source [SID1234528325]).

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OPKO’s senior management will provide a business update and discuss its financial results in a live conference call and audio webcast beginning at 4:30 p.m. Eastern time on Tuesday, August 7, 2018.

Conference Call & Webcast Information

WHEN: Tuesday, August 7, 2018 at 4:30 p.m. Eastern time
DOMESTIC DIAL-IN: (866) 634-2258
INTERNATIONAL DIAL-IN: (330) 863-3454
PASSCODE: 9243717
WEBCAST: View Source

For those unable to participate in the live conference call or webcast, a replay will be available beginning August 7, 2018 two hours after the close of the conference call. To access the replay, dial (855) 859-2056 or (404) 537-3406. The replay passcode is: 9243717. The replay can be accessed for a period of time on OPKO’s website at View Source.

On August 2, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, reported its financial results and corporate update for the quarter ended June 30, 2018 (Press release, Ultragenyx Pharmaceutical, AUG 2, 2018, View Source [SID1234528353]).

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"Enthusiasm from the XLH community has resulted in promising commercial uptake of Crysvita in the United States, and momentum continues to grow among both pediatric and adult patients," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In the second half of the year we will continue to focus on the launches of both Crysvita and Mepsevii, and advance the rest of our pipeline including our clinical-stage gene therapy programs with key data readouts expected for both programs."

Financial Results

For the second quarter of 2018, Ultragenyx reported a net loss of $52.7 million, or $1.06 per share, basic and diluted, compared with a net loss for the second quarter of 2017 of $72.9 million, or $1.72 per share, basic and diluted. The loss for the second quarter of 2018 includes a $40.3 million gain from Ultragenyx’s portion of the sale of the priority review voucher (PRV) received with the Crysvita (burosumab) approval. For the six months ended June 30, 2018, net loss was $22.5 million, or $0.46 per share, basic and diluted, compared with a net loss for the same period in 2017 of $141.2 million, or $3.35 per share, basic and diluted. In addition to the Crysvita PRV, the loss from the first six months also includes the sale of the Mepsevii (vestronidase alfa) PRV in January 2018 for net proceeds of $130.0 million. The net loss for the first six months of 2018 reflected cash used in operations of $165.6 million compared to $110.0 million for the same period in 2017.

Net Revenues

For the second quarter of 2018, Ultragenyx reported $12.8 million in total revenue. Ultragenyx recognized $8.9 million in revenue from the research agreement with Bayer. For Crysvita, Ultragenyx recognized $1.6 million in profit sharing and royalty revenue from the collaboration and license agreement with Kyowa Hakko Kirin. This includes $1.1 million in collaboration revenue in the U.S profit share territory, where Crysvita became commercially available on April 27, 2018, as well as $0.5 million in royalty revenue in the European territory, where Crysvita received conditional marketing authorization on February 23, 2018. There were nominal net product sales for Crysvita in other regions. Mepsevii product revenue for the second quarter of 2018 was $2.0 million, and UX007 named patient revenue was $0.2 million.

Operating Expenses

Total operating expenses for the second quarter of 2018 were $107.7 million compared with $78.4 million for the same period in 2017, including non-cash stock-based compensation of $19.6 million and $16.8 million in the second quarter of 2018 and 2017, respectively. Total operating expenses for the six months ended June 30, 2018 were $214.9 million compared with $148.4 million for the same period in 2017, including non-cash stock-based compensation of $38.4 million and $31.3 million in the first six months of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows and advances its pipeline.

Cash, cash equivalents, and investments

Cash, cash equivalents, and investments were $547.1 million as of June 30, 2018.

Recent Highlights

Crysvita in X-Linked Hypophosphatemia (XLH)

Positive data from the Phase 3 pediatric study demonstrated that Crysvita was superior to oral phosphate and active vitamin D (conventional therapy) in improving rickets in children with XLH after 40 weeks of treatment.
Mepsevii in mucopolysaccharidosis VII (MPS VII)

In Europe, Mepsevii received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), recommending the marketing authorization under exceptional circumstances of Mepsevii for the treatment of non-neurological manifestations of MPS VII. A decision from the European Commission, which has the authority to approve medicines for the European Union, is expected in the third quarter of 2018.
UX007 in long-chain fatty acid oxidation disorders (LC-FAOD)

Discussions are ongoing with FDA and EMA to determine the acceptability of filing UX007 for the treatment of LC-FAOD based on the totality of currently available data. The data from the Phase 2 study for UX007 show a significant reduction in major clinical events; however, the FDA continues to believe that the data are confounded and are not sufficient to support a New Drug Application (NDA). We continue to pursue potential filings with FDA and EMA based on the current data and expect to conclude discussions in the second half of 2018. These discussions also should provide further clarity regarding whether an additional study would be required for approval.
DTX401 gene therapy in glycogen storage disease type Ia (GSDIa)

The first patient has been dosed in the Phase 1/2 study of DTX401, our adeno-associated virus 8 (AAV8) gene therapy program for the treatment of patients with GSDIa. Data from the three-patient first dose cohort are expected in the second half of 2018.
The U.S. FDA granted fast-track designation to DTX401 for the treatment of GSDIa. This designation is designed to facilitate the development and expedite the review of drugs that are intended to treat serious conditions and fill an unmet medical need, and it allows for more frequent interaction with the FDA review team. It also enables eligibility for priority review if relevant criteria are met and the potential for a rolling review of the Biologic License Application (BLA) as data become available.
Upcoming Key Milestones

Crysvita (burosumab) in tumor-induced osteomalacia (TIO)

Data from all patients in the Phase 2 study in TIO are expected in the second half of 2018. This is an open label Phase 2 study evaluating the safety and efficacy of burosumab in adult patients with TIO.
UX007 in LC-FAOD and glucose transporter type-1 deficiency syndrome (Glut1 DS)

In LC-FAOD, additional clarity from FDA and EMA on the regulatory pathway is expected in the second half of 2018.
The fully-enrolled Phase 3 movement disorder study in patients with Glut1 DS is on track and data are expected in second half of 2018.
DTX301 gene therapy in ornithine transcarbamylase (OTC) Deficiency

Results from the fully-enrolled cohort 2 of the Phase 1/2 study are expected in second half of 2018.
DTX401 Gene Therapy in GSDIa

Data from the first, lowest dose cohort are expected in the second half of 2018.
Conference Call & Webcast Information

Ultragenyx will host a conference call today, Thursday, August 2, 2018 at 5pm ET to discuss second quarter 2018 financial results and to provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial 855-797-6910 (USA) or 262-912-6260 (international) and enter the passcode 2895644. The replay of the call will be available for one year.