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.

On August 2, 2018 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the pricing of its previously announced Hong Kong initial public offering and global offering (the "Offering") of 65,600,000 ordinary shares, par value $0.0001 per share (the "Shares"), at a public offering price of HK$108.00 per Share (Press release, BeiGene, AUG 2, 2018, View Source;p=RssLanding&cat=news&id=2361603 [SID1234528374]). Based on an assumed exchange rate of HK$7.8478 to US$1.00, the public offering price equates to US$13.76 per Share, or US$178.90 per American Depositary Share ("ADS"). BeiGene’s ADSs are currently listed on the Nasdaq Global Select Market under the symbol "BGNE" and each ADS represents 13 ordinary shares.

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The gross proceeds to BeiGene from the Offering, before deducting underwriting discounts and commissions and other offering expenses, are expected to be approximately HK$7.08 billion, or approximately US$903 million. In addition, BeiGene has granted the joint global coordinators a 30-day option to purchase up to an additional 9,840,000 Shares at the public offering price, less underwriting discounts and commissions.

BeiGene intends to use proceeds from the Offering for clinical trials, preparation for registration filings, and for the launch and commercialization of its core product candidates (zanubrutinib, tislelizumab, and pamiparib), as well as to fund continued expansion of its product portfolio in cancer and potentially other therapeutic areas, and for working capital, expanding internal capabilities, and general corporate purposes.

The Shares are expected to begin trading on the Main Board of The Stock Exchange of Hong Kong Limited on August 8, 2018 under the stock code "06160." The Offering is expected to close on the same day, subject to customary closing conditions.

Morgan Stanley & Co. International plc, Goldman Sachs (Asia) L.L.C., Credit Suisse (Hong Kong) Limited and CLSA Limited are acting as joint global coordinators, joint bookrunners and joint lead managers for the Global Offering. China International Capital Corporation Hong Kong Securities Limited, Deutsche Bank AG, Hong Kong Branch, UBS AG Hong Kong Branch are acting as joint bookrunners and joint lead managers. China Renaissance Securities (Hong Kong) Limited is acting as joint lead manager.

Sales of Shares outside of Hong Kong, initially offered in the United States and sold outside the United States that may be resold from time to time in the United States, are being offered pursuant to an automatically effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement relating to and describing the terms of the Offering was filed with the SEC and is available on the SEC’s website at www.sec.gov. The final prospectus supplement relating to the Offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may be obtained for free from the offices of Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014; Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, or email:[email protected]; and Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, One Madison Avenue, New York, New York 10010, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction.

On August 2, 2018 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2018, and provided a business update (Press release, Adaptimmune, AUG 2, 2018, View Source [SID1234528617]).

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"2018 is a year of delivery for Adaptimmune," said James Noble, Adaptimmune’s CEO. "We have completed multiple studies with the NY-ESO program, and released data showing responses in a second solid tumor. We have transitioned it to GSK as planned and will shortly be receiving payment of $27 million. We are now entirely focused on our wholly owned INDs, and remain on track to deliver initial response data from our MAGE-A10 and MAGE-A4 programs in the second half of 2018."

Clinical momentum in wholly owned programs

Adaptimmune will now focus its clinical, regulatory, and manufacturing organization on its wholly owned therapies – MAGE-A4, MAGE-A10, and AFP

·Dosing at one billion or more SPEAR T-cells across all studies with MAGE-A10 and MAGE-A4, and on track for response readouts from multiple solid tumors throughout the remainder of 2018.

·Initial safety data from AFP in hepatocellular carcinoma also on track for late 2018.

NY-ESO program transitioned to GSK

·As announced on July 24, 2018 (https://bit.ly/2LKhSvm), the NY-ESO SPEAR T-cell program has transitioned to GSK.

·Adaptimmune will receive $27.5 million (£21.2 million) from GSK as a result of the transition, as well as subsequent development and sales milestones and royalties based on successful development by GSK of this program.

Manufacturing

Adaptimmune is increasing the capacity of its dedicated manufacturing facility

·Routinely manufacturing SPEAR T-cells at the Navy Yard at target cell doses

Developing the capability at the Navy Yard to scale up to 30 manufacturing slots per month from the current number of 8 to 10 per month

·Maintaining 8 to 10 dedicated patient manufacturing slots per month at HCAT

Other corporate news

Adaptimmune is focused on its next stage of development and in a strong position to deliver success with SPEAR T-cell therapies

·Rafael Amado, Adaptimmune’s Chief Medical Officer, has assumed a new role as President of Research & Development effective from August 1, 2018. This brings together the clinical and research teams under a single leadership, which will allow better alignment and integration of all parts of R&D, from target identification and selection, to regulatory filings, enabling the delivery of Adaptimmune’s key priorities.

· Adaptimmune is ready for the next stage of clinical development and actively planning for registration trials, whether indication or target specific

· Funded through to early 2020 with cash and cash equivalents of $42.3 million and total liquidity(1) of $129.0 million, which does not include $27.5 million in payments from GSK as a result of the transition as this was invoiced after the second quarter

·Announced in April 2018 (https://bit.ly/2v7v3D3) that John Furey, Chief Operating Officer at Spark Therapeutics, was appointed as an independent Non-Executive Director to Adaptimmune’s Board of Directors, which became effective July 5, 2018

Financial Results for the three-month period ended June 30, 2018

Cash / liquidity position: As of June 30, 2018, Adaptimmune had cash and cash equivalents of $42.3 million and Total Liquidity(1) of $129.0 million, which does not include $27.5 million in payments from GSK as a result of the transition as this was invoiced after the second quarter

·Revenue: With effect from January 1, 2018, the Company has adopted a new accounting standard(2). Under this new accounting standard, revenue represents the upfront payment and milestones under the GSK Collaboration and License Agreement, which are recognized based on the percentage completion of the NY-ESO and PRAME development programs. Revenue for the three and six months ended June 30, 2018 was $9.0 million and $17.2 million, respectively. Revenue for the three and six months ended June 30, 2018 under the previous guidance would have been $3.3 million and $12.3 million, respectively, compared to $3.5 and $6.4 million for the same periods of 2017. The increase in revenue, compared to the six-month period in 2017, is primarily due to a reduction in the period over which the Company is recognizing revenue following GSK’s exercise of its option over the NY-ESO program in September 2017 and additional development milestones achieved.

·Research and development ("R&D") expenses: R&D expenses for the three and six months ended June 30, 2018 were $26.7 million and $52.0 million, respectively, compared to $19.6 million and $38.2 million for the same periods of 2017. The increase was primarily due to increased costs associated with clinical trials, manufacturing for clinical trials, and increased personnel costs.

· General and administrative ("G&A") expenses: G&A expenses for the three and six months ended June 30, 2018 were $11.3 million and $22.5 million, respectively, compared to $7.7 million and $14.2 million for the same periods of 2017. The increase was primarily due to increased personnel costs consistent with the Company’s planned growth, an increase in costs associated with developing its IT infrastructure and an increase in other corporate costs.

·Other (expense) income, net: Other expense for the three and six months ended June 30, 2018 was $15.4 million and $8.3 million, respectively, compared to an income of $3.2 million and $3.7 million for the same periods of 2017. Other income primarily comprises unrealized foreign exchange gains, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.

· Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six months ended June 30, 2018 was $43.8 million and $64.6 million respectively ($(0.08) and $(0.11) per ordinary share) compared to $20.2 million and $42.0 million ($(0.04) and $(0.09) per ordinary share) in the same periods of 2017.

Financial guidance

The Company believes that its existing cash, cash equivalents, marketable securities and income from GSK upon transition of the NY-ESO program will fund the Company’s current operations through to early 2020.

Conference call information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) today, August 2, 2018. The live webcast of the conference call will be available via the events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available

(1) Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

(2) ASC 606, Revenue from Contracts with Customers.

after the call at the same address. To participate in the live conference call, please dial (833) 652-5917 (U.S.) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (8149978).

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.