On May 10, 2018 Synergy Pharmaceuticals Inc. (NASDAQ:SGYP), a biopharmaceutical company focused on the development and commercialization of novel gastrointestinal (GI) therapies, reported its financial results and business update for the three months ended March 31, 2018 (Press release, Synergy Pharmaceuticals, MAY 10, 2018, View Source [SID1234526501]).

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"The first quarter of 2018 was all about executing on our three key business priorities of optimizing the value of TRULANCE, ensuring a strong financial foundation, and continuing to explore all strategic business development opportunities," said Troy Hamilton, Chief Executive Officer of Synergy Pharmaceuticals Inc. "With TRULANCE, we saw continued growth in prescriptions, market share and its prescriber base and with the IBS-C launch in late February, we have the opportunity to continue to drive further sales growth. In addition, we continued to efficiently manage our operating expenses by prioritizing key investments in areas of high return, such as expanding market access. Finally, we amended our debt agreement to allow for more flexible access to capital as we are pursuing strategic options that align with our core mission to deliver exceptional value to our patients, customers and shareholders. Overall, our progress against our key business priorities during the first quarter reflect our commitment to maximizing shareholder value while also maintaining our focus on providing safe and effective treatment options for patients living with chronic GI conditions."

First Quarter 2018 and Recent Highlights

Optimizing the Value of TRULANCE

44,177 TRULANCE 30-count packs were dispensed in the first quarter of 2018, resulting in a total of 132,628 TRULANCE 30-count packs dispensed since the product’s launch on March 20, 2017, per IQVIA.
TRULANCE was the only prescription brand for CIC and IBS-C to show positive total and new prescription volume growth in the first quarter over the prior quarter, per IQVIA.
In the nine weeks since the launch of the IBS-C indication in late February, TRULANCE prescription volume grew 24% versus the prior nine weeks or nearly five times the branded CIC and IBS-C prescription market growth rate, per IQVIA.
The total number of unique healthcare practitioners prescribing TRULANCE since launch reached nearly 12,000 in the first quarter of 2018, increasing more than 20% over the prior quarter, per IQVIA.
TRULANCE currently has over 70% payer coverage across all segments including commercial, Medicare Part D and Managed Medicaid.
Ensuring a Strong Financial Foundation

Financial Results

Reported TRULANCE net sales were $8.6 million in the first quarter of 2018 compared to net sales of $9.4 million during the fourth quarter of 2017. TRULANCE first quarter net sales of $8.6 million increased by 18% compared to fourth quarter of 2017 adjusted net sales (non-GAAP) of $7.3 million.
Reported total operating expenses were $43.5 million in the first quarter of 2018 compared to total operating expenses of $43.8 million during the fourth quarter of 2017.
Total adjusted operating expenses (non-GAAP) were $40.6 million in the first quarter of 2018 compared to $41.1 million in total adjusted operating expenses (non-GAAP) in the fourth quarter of 2017.
In February 2018, Synergy amended its Term Loan agreement with CRG to provide more financial flexibility while the company continues to evaluate various strategic options. Synergy has the ability to access up to an additional $100 million in 2018 in three tranches.
Synergy received $5.0 million in non-refundable upfront payments related to the TRULANCE Canadian licensing agreement with Cipher Pharmaceuticals completed in February 2018. This payment was recorded as deferred revenue for the quarter and will be recognized as revenue upon meeting future contractual obligations. Under the terms of the licensing agreement, Synergy is eligible for an additional milestone payment upon regulatory approval in Canada, as well as royalties from Trulance product sales in Canada.
Synergy reported a net loss of $36.1 million, or $0.15 per share, for the first quarter of 2018.
Cash and cash equivalents were approximately $98.7 million at the end of the first quarter.
2018 Financial Guidance

As a result of ongoing efforts to improve cost efficiency measures, Synergy is lowering projected total adjusted operating expense (non-GAAP) guidance for 2018 to be in the range of $165 million – $175 million versus previously guided $175 million – $185 million.
Exploring All Strategic and Business Development Opportunities

Collaborations & Partnerships

Synergy initiated a partnership with the National Cancer Institute (NCI) to collaborate on a NCI-funded and managed clinical biomarker study to evaluate dolcanatide’s potential to prevent colorectal cancer. The study will assess the colorectal bioactivity of dolcanatide in healthy volunteers and will inform the feasibility and design of a larger study. This is the first clinical biomarker study evaluating the potential benefit of using a uroguanylin analog in colorectal cancer prevention. The advancement of Synergy’s proprietary uroguanylin analog, dolcanatide, into this clinical trial builds on Synergy and NCI scientists’ pioneering work showing the important role of uroguanylin in the complex biology of colorectal cancer.
Synergy’s Canadian partner, Cipher Pharmaceuticals, is currently in discussions with Health Canada and plans to file a New Drug Submission for TRULANCE in IBS-C in the second half of 2018. The regulatory review period is approximately one-year from the submission date.
Ongoing Strategic Review

Synergy continues to engage in an ongoing review of strategic business opportunities focused on maximizing shareholder value. This review process includes, but is not limited to, potential US and ex-US partnerships, licensing, and merger and acquisition transactions. Synergy expects to provide further updates on or before it reports second quarter 2018 results.
First-Quarter Conference Call & Webcast

Synergy will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss first quarter 2018 results. Participants may access the conference call by dialing 877-407-3978 (US and Canada) or 412-902-0039 (International). Please let the operator know you would like to join the Synergy Pharmaceuticals call. To access the webcast as well as a PDF copy of the presentation, please visit the Investors section of Synergy’s website at www.synergypharma.com.

An audio replay of the conference call will also be available beginning approximately two hours after the call’s conclusion, and will remain available through May 24, 2018. The replay may be accessed by dialing 877-660-6853 (U.S. and Canada) or 201-612-7415 (International) and entering conference ID number 13668774. A replay of the webcast will also be available on the Investors section of Synergy’s website at www.synergypharma.com.

On May 10, 2018 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that management will present at the upcoming UBS Global Healthcare Conference taking place May 21-23, 2018 in New York, NY, USA (Press release, Zymeworks, MAY 10, 2018, View Source [SID1234526500]).

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The Company will present on Monday, May 21, 2018 at 2:00 p.m. ET. Interested parties can access a live webcast of the presentation via a link from Zymeworks’ website at View Source, which will also host a recorded replay available afterwards.

On May 10, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP), reported financial results for the first quarter ended March 31, 2018, and provided an update on the Company’s recent activities (Press release, Ziopharm, MAY 10, 2018, View Source [SID1234526499]).

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"The landscapes of CAR-T therapies and the use of cytokines are playing out as anticipated," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "Regarding CAR-T, we find the dominating concern among patients, physicians and payers is the high costs and lengthy manufacturing time. We are addressing these issues head on with Sleeping Beauty CAR-T cells manufactured in two days with our technology called ‘point-of-care,’ which we expect to enter the clinic this year."

Dr. Cooper continued, "Immune-stimulatory cytokines have gained significant momentum as potential combination therapies with immune checkpoint inhibitors. We are excited to build on our Controlled IL-12 platform and its ability to turn cold tumors hot as we explore more tumor types that can benefit from our ability to regulate IL-12."

Program Updates

Controlled IL-12

Ziopharm is advancing Ad-RTS-hIL-12 plus veledimex as a gene therapy to treat patients with recurrent glioblastoma (rGBM) as a monotherapy and in combination with an immune checkpoint inhibitor. Ad-RTS-hIL-12 plus veledimex is an adenoviral vector (Ad) administered via a single injection into the tumor and engineered to express human IL-12 (hIL-12), a powerful cytokine that has demonstrated a targeted, anti-tumor immune response with the ability to activate and recruit killer T cells to the tumor site. The expression of hIL-12 is controlled and modulated with the RheoSwitch Therapeutic System (RTS) by the small molecule veledimex, an activator ligand which is taken by mouth and crosses the blood-brain barrier.

The Company reported that it is focusing its resources on studies of Controlled IL-12 in additional tumor types to demonstrate the value of this platform technology and, as a result, the Company is pausing plans for the pivotal trial in rGBM.

Combination Trial with OPDIVO (nivolumab) in rGBM Initiated. Ziopharm has initiated a trial of adult patients with rGBM to evaluate a single dose of Ad-RTS-hIL-12 plus daily veledimex for 15 days in combination with OPDIVO (nivolumab), an immune checkpoint inhibitor targeting programmed death-1 (PD-1). In January, the protocol for this first-ever dosing combination was finalized with the U.S. Food and Drug Administration (FDA), and since then the Company has been working with clinical trial sites. Ziopharm expects the first patient to be dosed in this trial in the second quarter.

Additional Oncology Indications to be Explored Including in Combination with Immune Checkpoint Inhibitors. The Company previously has presented compelling data demonstrating that Ad-RTS-hIL-12 plus veledimex can turn cold tumors hot by recruiting and activating a sustained immune response that favorably changes the tumor microenvironment in different tumor types. Therefore, Ziopharm plans to explore the potential of the Controlled IL-12 platform by combining it with immune checkpoint inhibitors in multiple tumor types in addition to rGBM later this year.

Pivotal Trial in rGBM Paused. Ziopharm is pausing plans for its pivotal randomized control trial for Ad-RTS-hIL-12 plus 20mg dose of veledimex for the treatment of rGBM. The Company is progressing in resolving previously disclosed technical requirements related to Chemistry and Manufacturing Control (CMC), making this asset phase-3 ready for rGBM.

Expansion Substudy Added to Phase 1 Trial in rGBM. Ziopharm is initiating an expansion substudy in its Phase 1 trial to evaluate Ad-RTS-hIL-12 plus veledimex as a monotherapy to treat patients with rGBM. The Company is adding patients who have not received steroids for four weeks prior and have not been treated with bevacizumab for their disease to the cohort receiving a 20mg dose of veledimex. Previously, the Company has shown improvement in survival benefit in patients who received Ad-RTS-hIL-12 plus 20mg of veledimex with no steroids or low-dose steroids. The data from these patients will help further understand the benefits of IL-12 as a single-agent.

Phase 1 for Pediatric Tumors Ongoing. Ziopharm is enrolling pediatric patients in its Phase 1 trial of Ad-RTS-hIL-12 with veledimex for the treatment of brain tumors at multiple U.S. sites.

ASCO 2018 Poster Title Announced. The company reported that a research poster entitled, "Demonstration of Anti-Tumor Immunity via Intratumoral Regulated Platform Ad-RTS-hIL-12 in Advanced Breast Cancer and Recurrent Glioblastoma Patients," has been accepted for presentation during the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. The presentation is scheduled for the Developmental Therapeutics-Immunotherapy poster session on Monday, June 4, 8 to 11:30 a.m. CT in Hall A. Abstract details will be made public on May 16, per ASCO (Free ASCO Whitepaper) embargo rules.

Sleeping Beauty and Adoptive Cell Therapies

Using Ziopharm’s non-viral approach leveraging Sleeping Beauty platform to genetically modify cells, the Company is developing chimeric antigen receptor (CAR) T-cell (CAR-T) and T-cell receptor (TCR) T-cell (TCR-T) therapies. These programs are being advanced in collaboration with Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation, and with MD Anderson Cancer Center, the National Cancer Institute and Merck KGaA, Darmstadt, Germany. This non-viral approach to genetically modifying T cells has the potential to reduce the costs of and expand access to this immunotherapy based on very rapid production and thus avoiding the need for centralized manufacturing.

Initiation of First Point-of-Care Clinical Trial Expected in Second Half of 2018. Ziopharm is advancing the Sleeping Beauty platform towards point-of-care manufacturing for the very rapid manufacturing of genetically modified CAR+ T cells, with Ziopharm’s first clinical trial utilizing this approach expected to begin in the second half of 2018. Ziopharm’s third-generation point-of-care trial intends to use the Sleeping Beauty platform to manufacture CAR+ T cells co-expressing membrane-bound interleukin-15, or mbIL15, within two days after genetically modifying T cells from the patient. The Company reported that the investigational new drug (IND) application has been submitted to the FDA by MD Anderson to initiate a Phase 1 trial to evaluate the point-of-care technology for the investigational treatment of CD19+ leukemia and lymphoma.

Phase 1 Trial of Sleeping Beauty-Modified TCRs to Treat Solid Tumors Expected to Initiate in Second Half of 2018. The NCI expects to initiate a Phase 1 trial in the second half of 2018 to evaluate adoptive cell transfer-based immunotherapies genetically modified using the Sleeping Beauty platform to express TCRs for the treatment of solid tumors. Ziopharm, Intrexon, and the NCI last year entered into a Cooperative Research and Development Agreement to develop and evaluate adoptive cell therapy for patients with advanced cancers using autologous peripheral blood lymphocytes genetically modified using the Sleeping Beauty system to express TCRs that recognize specific immunogenic mutations, or neoantigens, expressed within a patient’s solid tumor.

Phase 1 Trial of CD33-specific CAR+ T Therapy for Acute Myeloid Leukemia (AML). Enrollment is underway at MD Anderson in the Phase 1 adoptive cellular therapy clinical trial of CAR+ T-cell therapy in patients with refractory/recurrent AML that express CD33. This trial infuses autologous T cells genetically modified with lentivirus to express a CD33-specific CAR and a kill switch for elimination of genetically modified cells. Data from this trial are expected to serve as the basis for evaluating CD33 as a potential target for further development using non-viral manufacturing of T cells with Ziopharm’s point-of-care technology.

Corporate Update

Ziopharm appointed Robert Hadfield, who has financial, biotech and legal experience, to serve as general counsel. He joined the company from the Longwood Fund, a health care venture capital firm in Boston, and Flex Pharma before that, serving as general counsel in both. Mr. Hadfield received his J.D. from Georgetown University.

First-Quarter 2018 Financial Results

Net loss applicable to the common shareholders for the first quarter of 2018 was $21.1 million, or $(0.15) per share, compared to a net loss of $19.7 million, or $(0.15) per share, for the first quarter of 2017. The increased net loss is primarily due to a decrease in revenue recognized related to the implementation of ASU 2014-09 and an increase in operating expenses.
Research and development expenses were $10.2 million for the first quarter of 2018, compared to $12.0 million for the first quarter of 2017. The decrease in research and development expenses for the three months ended March 31, 2018 is primarily due to decreased manufacturing and preclinical activity related to our cell and gene therapy programs, as our trials prepare to be moved forward in the clinic.
General and administrative expenses were $6.2 million for the first quarter of 2018, compared to $3.6 million for the first quarter of 2017. The increase in general and administrative expenses for the three months ended March 31, 2018 is primarily due to severance and non-cash stock compensation expense.
The Company ended the quarter with unrestricted cash resources of approximately $51.1 million.
In addition, a prepayment of approximately $32.4 million remains for programs to be conducted by the Company at MD Anderson Cancer Center under the current Research and Development Agreement.
The Company believes its current resources will be sufficient to fund its currently planned operations into the second quarter of 2019.
Conference Call and Slide Webcast

Ziopharm will host a webcast and conference call today, May 10, at 4:30 p.m. ET. The call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 4857096. To access the slides and live webcast or the subsequent archived recording, visit the "Investors & Media" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On May 10, 2018 Valeant Pharmaceuticals International, Inc. (NYSE/TSX: VRX) ("Valeant") reported that Joseph C. Papa, chairman and chief executive officer, and Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, are scheduled to participate at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas on May 16, 2018 at 10:40 a.m. PDT (1:40 p.m. EDT) (Press release, Valeant, MAY 10, 2018, http://ir.valeant.com/news-releases/2018/05-10-2018-133108200 [SID1234526498]).

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A live webcast and audio archive of the event will be available on the Investor Relations page of the Valeant web site at: http://ir.valeant.com/events-and-presentations/2018.

On May 10, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported financial results and business highlights for the first quarter ended March 31, 2018 (Press release, Tocagen, MAY 10, 2018, View Source;p=RssLanding&cat=news&id=2348569 [SID1234526497]).

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"We are off to a strong start this year and are well positioned to execute on our key priorities, including completing enrollment in our Phase 3 trial in recurrent brain cancer by year end," said Marty Duvall, chief executive officer of Tocagen. "In parallel, we are working to expand Toca 511 & Toca FC into newly diagnosed brain cancer while exploring its potential in other tumor types."

First Quarter 2018 and Recent Highlights

Entered into a license agreement with ApolloBio: In April 2018, Tocagen and ApolloBio entered a license agreement to develop and commercialize Toca 511 & Toca FC within the greater China region. Tocagen is eligible to receive up to $127 million in upfront payment, development and commercial milestones, plus additional double-digit tiered sales royalties. Upfront and near-term development milestone payments total up to $20 million. More details are available in the corresponding Form 8-K filed with the U.S. Securities and Exchange Commission (SEC).
Updated durable response data from Phase 1 recurrent high grade glioma (rHGG) resection study: Updated durable response data from the Phase 1 study involving patients with rHGG who received Toca 511 & Toca FC at the time of surgical resection were presented by trial investigators at the 2018 American Academy of Neurology (AAN) Annual Meeting and 2018 American Association of Neurological Surgeons (AANS) Annual Scientific Meeting. Tocagen previously presented data from this study as of August 15, 2017. In the presentations at AAN and AANS, updated data showed Toca 511 & Toca FC continue to demonstrate a favorable safety profile and all study responders remained alive and in complete response as of December 20, 2017. As of this cutoff date, the median duration of durable response had not yet been reached, with a median follow-up period of 37.4 months.
Presented preliminary Toca 6 Phase 1 data: At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018, research collaborators presented preliminary clinical data that suggest the potential feasibility of intravenous (IV) administration of Toca 511 from the ongoing Toca 6 Phase 1 trial of Toca 511 & Toca FC in patients with advanced solid tumors. Safety, tolerability and confirmation of vector deposition in metastatic tumors was demonstrated in five patients with advanced solid tumors who received Toca 511 intravenously. These data inform Tocagen’s plans to initiate additional studies evaluating the efficacy of Toca 511 & Toca FC in patients with advanced cancers.
First Quarter 2018 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $10.4 million for the quarter ended March 31, 2018, compared to $6.6 million for the quarter ended March 31, 2017. The increase in R&D expenses in 2018 was primarily driven by higher costs to support the expanded Toca 5 trial and increased activities in manufacturing of Toca 511 & Toca FC.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million for the quarter ended March 31, 2018, compared to $1.9 million for the quarter ended March 31, 2017. The increase in G&A expenses was primarily due to increased stock-based compensation expense and costs associated with being a public company during the first quarter of 2018.

Net Loss: Net loss was $12.9 million, or $0.65 per common share (basic and diluted), for the quarter ended March 31, 2018, compared to a net loss of $9.1 million, or $4.11 per common share (basic and diluted), for the quarter ended March 31, 2017. The 2018 calculation is based on 19.9 million average common shares outstanding for the first quarter of 2018, compared to 2.2 million average common shares outstanding for the first quarter of 2017.

Cash Position and Guidance

Cash, cash equivalents and marketable securities were $74.0 million at March 31, 2018 compared to $88.7 million at December 31, 2017. Subsequent to the close of the first quarter 2018, Tocagen signed a license agreement with ApolloBio and expects to receive $16 million by early in the third quarter of 2018, according to the terms of the licensing agreement. Tocagen reiterates its annual guidance and continues to estimate the total cash used in 2018 to fund operations, capital expenditures and debt amortization will not exceed $50 million.

About Toca 511 & Toca FC

Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprised of an investigational biologic, Toca 511 and an investigational small molecule, Toca FC. Toca 511 (vocimagene amiretrorepvec) is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered, extended-release formulation of the prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells in the tumor microenvironment resulting in anti-cancer immune activation and subsequent tumor killing.