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Tocagen Reports First Quarter 2018 Financial and Business Results

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.

Syros Reports First Quarter 2018 Financial Results and Highlights Key Accomplishments and Upcoming Milestones

On May 10, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of genes, reported financial results for the quarter ended March 31, 2018 and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, MAY 10, 2018, View Source [SID1234526496]).

"Our priorities for 2018 are advancing our first-in-class drug candidates SY-1425 and SY-1365, leveraging our leading gene control platform to continue fueling our pipeline, and building on our strong financial position and company fundamentals," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "We have made terrific progress on all three fronts this year, presenting preclinical data that support the planned expansion of our Phase 1 trial of SY-1365 into ovarian cancer, entering into a collaboration with Incyte that applies our platform to diseases beyond our current areas of focus, and fortifying our financial position with a successful public offering. We continue to build momentum as we prepare for our planned data readouts in the fourth quarter of this year for both SY-1425 and SY-1365, and we remain focused on executing with excellence as we strive to build a great and enduring company with medicines that make a profound difference for currently underserved patients."

Upcoming Milestones

Syros plans to report initial clinical data in the fourth quarter of 2018 from cohorts in its Phase 2 trial evaluating SY-1425 in combination with azacitidine in RARA and IRF8 biomarker-positive newly diagnosed acute myeloid leukemia (AML) patients who are not suitable candidates for standard chemotherapy, and in combination with daratumumab in biomarker-positive relapsed or refractory AML and higher-risk myelodysplastic syndrome (MDS) patients. The primary objective of the trial is to evaluate the safety and efficacy of these combinations in biomarker-positive AML and higher-risk MDS patients. The Company announced today that it plans to add approximately 25 biomarker-negative newly diagnosed AML patients who are not suitable candidates for standard chemotherapy to its ongoing Phase 2 trial in order to support the development of a commercial companion diagnostic test for SY-1425. These patients will be treated with SY-1425 in combination with azacitidine.
Syros plans to open expansion cohorts in mid-2018 in its ongoing Phase 1 trial of SY-1365 in multiple patient populations with ovarian and breast cancer to evaluate SY-1365 as a single agent and in combination with standard-of-care therapies.
Syros plans to report clinical data in the fourth quarter of 2018 from the dose escalation portion of its ongoing Phase 1 trial of SY-1365 in advanced solid tumor patients.
Syros plans to select a new development candidate from its preclinical pipeline by the end of 2018.
Recent Platform and Pipeline Highlights

In April 2018, Syros and its collaborators at the Dana-Farber Cancer Institute presented new preclinical data on SY-1365 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago. SY-1365 demonstrated potent anti-tumor activity in multiple models of heavily pretreated ovarian cancer, inhibiting tumor growth in 10 of the 17 patient-derived xenograft models studied, including inducing complete regressions. These responses were observed irrespective of BRCA status or sensitivity to a PARP inhibitor. Preclinical studies also pointed to potential biomarkers of response to SY-1365.
First Quarter 2018 Financial Results

Cash, cash equivalents and marketable securities as of March 31, 2018 were $121.7 million, compared with $72.0 million on December 31, 2017. This increase in cash reflects aggregate gross proceeds of approximately $46.0 million from Syros’ underwritten public offering of common stock that closed in February 2018, $1.4 million in proceeds from a private placement of stock to Incyte Corporation concurrent with this public offering, and a $10.0 million upfront payment and $10.0 million in proceeds from the sale of Syros common stock received in January 2018 in connection with Syros’ entry into its collaboration with Incyte.

For the first quarter 2018, Syros reported a net loss of $14.5 million, or $0.48 per share, compared to a net loss of $11.5 million, or $0.49 per share, for the same period in 2017. Stock-based compensation included in the net loss was $1.7 million for the first quarter 2018, compared to $0.9 million for the same period in 2017.

Revenues were $0.4 million for the first quarter of 2018, as compared to $1.1 million for the same period in 2017. Revenues in the first quarter of 2018 were earned under Syros’ collaboration with Incyte, compared to revenues in the first quarter of 2017, which were earned from a research agreement with a multinational pharmaceutical company.
Research and development (R&D) expenses were $11.1 million for the first quarter of 2018, as compared to $9.6 million for the same period in 2017. This increase was primarily attributable to increased external research and development costs associated with Syros’ ongoing clinical trials. Stock-based compensation included in R&D expenses was $0.6 million for the first quarter 2018, compared to $0.3 million for the same period in 2017.
General and administrative (G&A) expenses were $4.1 million for the first quarter of 2018, as compared to $3.1 million for the same period in 2017. This increase was primarily attributable to an increase in employee-related costs, including salary, benefits and stock-based compensation, as well as legal and professional fees associated with entry into Syros’ collaboration with Incyte. Stock-based compensation included in G&A expenses was $1.1 million for the first quarter 2018, compared to $0.6 million for the same period in 2017.
Financial Guidance

Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to enable it to fund its planned operating expenses and capital expenditure requirements into 2020.

Conference Call and Webcast:

Syros will host a conference call today at 8:30 a.m. ET to discuss these first quarter 2018 financial results and provide a corporate update.

The live call may be accessed by dialing (866) 595-4538 for domestic callers or (636) 812-6496 for international callers and referencing conference ID number: 2967666. A live webcast of the conference call will be available online on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 90 days

Synlogic Announces Leadership Change

On May 10, 2018 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to probiotics to develop novel, living medicines, reported that Aoife Brennan, M.B., B.Ch., has accepted the position of Interim President and Chief Executive Officer of Synlogic, effective immediately (Press release, Synlogic, MAY 10, 2018, View Source [SID1234526495]).

This appointment follows the resignation of Jose Carlos Gutiérrez-Ramos, Ph.D., as Synlogic’s President and Chief Executive Officer and a member of its board of directors. Dr. Gutiérrez-Ramos will serve in an advisory capacity as needed.

"On behalf of the entire board I would like to thank JC for his contributions to Synlogic, particularly in transitioning the Company from a research organization to a publicly traded, clinical-stage company, and we wish him well in his future endeavors," said Peter Barrett, chairman of Synlogic’s board of directors. "Aoife has done a remarkable job advancing our development efforts, and our board has complete confidence in her ability to lead the company on an interim basis as we conduct our search for a permanent CEO to drive the next stage of Synlogic’s growth. To aid in this transition, I will assume the role of Executive Chairman of the Board and will support Aoife as we continue to execute on Synlogic’s strategic priorities and advance the Company’s research, preclinical activities and ongoing clinical trials, which remain on track per the Company’s prior guidance."

"JC leaves us with a strong, well-funded organization, compelling science and clinical programs, and a world-class team. We greatly appreciate his efforts to bring Synlogic from discovery to a clinical development company and his guidance as a mentor and leader," said Dr. Brennan. "I look forward to working with the leadership team and our board to continue to advance our mission to develop novel living medicines that will change the lives of patients with serious and life-threatening diseases."

Regulus Reports First Quarter 2018 Financial Results and Pipeline Progress

On May 10, 2018 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the first quarter ended March 31, 2018 and provided a pipeline update (Press release, Regulus, MAY 10, 2018, View Source [SID1234526494]).

"We are very pleased with the progress being made on advancing our pipeline, including the recent initiation of the multiple ascending dose (MAD) study for RGLS4326; the advancement of two new pre-clinical programs in important areas of unmet need; and the continued advancement of the RG-012 program," said Jay Hagan, President and Chief Executive Officer of Regulus. "These two new pre-clinical programs represent attractive areas of development for Regulus beyond our two chronic kidney disease programs."

Pipeline Update

RGLS4326 for autosomal dominant polycystic kidney disease (ADPKD): As previously announced, a Phase 1 MAD study was recently initiated in healthy volunteers. This trial was initiated based on data from the ongoing Phase 1 single ascending dose (SAD) trial, in which RGLS4326 has been determined to be well tolerated to date. The Phase 1 SAD study has completed dose escalation and continues in the planned follow-up phase, which is on-track for completion in the second half of 2018. Data from both studies will provide pharmacokinetics and safety data in advance of the Phase 2 proof-of-concept (POC) study estimated for initiation in the second half of 2019.

Pre-clinical programs: Based on robust human in vitro data and murine in vivo data, the Company announced today it is advancing programs in Hepatitis B virus and immunology (targets undisclosed).

RG-012 for Alport syndrome: The Phase 2 HERA study is ongoing and data from the Phase 1 renal biopsy study is anticipated by year-end 2018.

Financial Results

Cash Position: As of March 31, 2018, Regulus had cash, cash equivalents and short-term investments of $45.1 million.

Research and Development (R&D) Expenses: R&D expenses were $11.8 million for the quarter ended March 31, 2018, compared to $15.8 million for the quarter ended March 31, 2017. The decrease was primarily the result of a reduction in personnel-related costs subsequent to our May 2017 corporate restructuring and the wind-down of clinical activities related to the RG-101 program.

General and Administrative (G&A) Expenses: G&A expenses were $3.8 million for the quarter ended March 31, 2018, compared to $4.0 million for the quarter ended March 31, 2017.

Revenue: Revenue was less than $0.1 million for the quarters ended March 31, 2018 and 2017.

Net Loss: Net loss was $16.0 million, or $0.15 per share (basic and diluted), for the quarter ended March 31, 2018, compared to a net loss of $20.0 million, or $0.38 per share (basic and diluted), for the quarter ended March 31, 2017.

Conference Call Details

Regulus will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss first quarter financial results and provide a general business update. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 8993969. To access the replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), conference ID 8993969. The webcast and telephone replay will be archived on the company’s website following the call

Radius Health Reports First Quarter 2018 Financial and Operating Results and Provides Business Update

On May 10, 2018 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), reported its financial results for the first quarter ended March 31, 2018 and provided a business update (Press release, Radius, MAY 10, 2018, View Source [SID1234526493]).

"The Company’s first quarter results highlight the strong performance of TYMLOS in the U.S. anabolic osteoporosis market, having captured one third of new patient starts with bone building anabolic therapy within less than a year of our commercial launch," said Jesper Hoeiland, President and Chief Executive Officer of Radius. "We remain focused on further increasing our market penetration and are committed to achieving leadership in the anabolic market with our differentiated and responsibly priced drug."

"I’m also very pleased that we made significant progress in advancing our clinical pipeline. Having finalized our development pathways for elacestrant and abaloparatide-patch, we remain on track with our preparations to launch these global pivotal studies," Mr. Hoeiland concluded.

TYMLOS (abaloparatide) injection

First quarter 2018 sales of TYMLOS in the U.S. were $14.5 million, an increase of 90% from the fourth quarter of 2017. TYMLOS prescriptions reached 31% of new anabolic patient starts (based on New Patients to Brand, NBRx PMOT) and 13% of the total U.S. anabolic osteoporosis market (based on Patient Months on Therapy, TRx PMOT) in the first quarter of 2018.

Less than a year after launch, TYMLOS has surpassed the level of U.S. commercial market access for the competing anabolic product, with 95% coverage in commercial plans. TYMLOS coverage in Medicare Part D plans has also increased to 43%. 263 million lives now have access to TYMLOS representing 88% of the total insured US population.

At the Academy of Managed Care Pharmacy (AMCP) Annual Meeting on April 25th, Radius presented two posters in support of the clinical and cost-effective value of treating earlier with TYMLOS to build bone followed by antiresorptive maintenance treatments. The findings demonstrate that sequential therapy of TYMLOS followed by generic alendronate was shown to improve outcomes at a lower total cost of care compared to teriparatide followed by generic alendronate for the treatment of US women at high risk for fracture. Further, sequential therapy with TYMLOS followed by generic alendronate was shown to improve outcomes at a lower total cost of care compared to starting with generic alendronate for women at high risk of fracture.

There was a 103% increase in the total number of U.S. physicians prescribing TYMLOS in the first quarter of 2018 versus the previous quarter. TYMLOS’ share of the total anabolic volume written by these physicians increased from 20% in the fourth quarter of 2017 to 38% in the first quarter of 2018. TYMLOS’ share of new prescriptions written by these physicians increased from 32% in the fourth quarter of 2017 to 49% in the first quarter of 2018.

The Company’s Awareness Trial and Usage Survey in the first quarter of 2018 showed TYMLOS reaching 80% of aided awareness, and a high intention by physicians to treat with TYMLOS, surpassing the competing anabolic product in the market.

Radius expects TYMLOS to capture on average 19-21% of the U.S. anabolic osteoporosis market in 2018 and that the U.S. anabolic market will continue its positive growth trajectory since TYMLOS was launched in May 2017, with an expected 5-7% volume increase.

A 5.9% price increase for TYMLOS took effect on February 22, 2018.
Pipeline Highlights

Abaloparatide -Transdermal Patch (abaloparatide-patch)

In Q1 2018, Radius finalized a development pathway for abaloparatide-patch after regulatory alignment with the FDA and entered into a scale-up and commercial supply agreement with 3M Company (3M).

The Company is on track with its ongoing efforts with partner 3M to increase manufacturing capacity to support the pivotal study and for clinical and non-clinical studies that will be included in a future New Drug Application (NDA) submission. The Phase 3 study of abaloparatide-patch is planned to start in mid-2019.
Abaloparatide – Subcutaneous (SC)

European MAA
In March 2018, Radius announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a negative opinion on the Company’s marketing authorization application (MAA) for abaloparatide-SC for the treatment of osteoporosis in postmenopausal women at increased risk for fracture. In April 2018, Radius submitted a request for re-examination of the CHMP’s opinion.
Male Osteoporosis Trial

In March 2018 Radius initiated the Phase 3 ATOM (Abaloparatide Treatment for Osteoporosis in Males) study of abaloparatide-SC for the treatment of osteoporosis in men, which, if successful, will form the basis of a supplemental NDA seeking to expand TYMLOS’ label. The study is a randomized, double-blind, placebo-controlled trial that will enroll approximately 225 men with osteoporosis at high risk of fracture. The study will include a primary endpoint of change in lumbar spine bone mineral density ("BMD") at 12 months versus placebo, and specialized high-resolution imaging of bone structure in a subset of the study participants.

Male osteoporosis is estimated to account for approximately 10% of the total treated osteoporotic patient population.
Elacestrant (RAD1901)

Based on EMA and FDA feedback, Radius announced in March 2018 that it will conduct a single, randomized, comparator controlled Phase 3 trial of elacestrant as a third-line monotherapy in approximately 300 patients with ER positive/HER2 negative advanced/metastatic breast cancer. Depending on the results, this study is expected to support applications for global marketing approvals for elacestrant. Patients in the study would be randomized to receive either elacestrant or an investigator’s choice of an approved hormonal agent and the primary endpoint of the study will be progression-free survival (PFS). Start-up activities for the randomized study are well underway and Radius will provide further study details when the Phase 3 trial is initiated, which the Company expects will be in the second half of 2018.
RAD140

Patient enrollment is ongoing in the Phase 1 study evaluating the safety and maximum tolerated dose of RAD140, a nonsteroidal selective androgen receptor modulator (SARM), in patients with hormone receptor-positive, locally advanced or metastatic breast cancer. The Company expects to provide an update on the RAD140 development program by the end of 2018.
Operational Activities

In March 2018, the Company consolidated operations in its headquarters in Waltham, Massachusetts and office in Wayne, Pennsylvania. As part of this consolidation, Radius’ Parsippany, New Jersey office will be closed.
Anticipated Upcoming Milestones

Elacestrant
Initiate a Phase 3 clinical trial as third-line monotherapy in advanced/metastatic ER-positive/HER2-negative breast cancer patients in the second half of 2018
Collaboration agreement for elacestrant combination therapy
RAD140
Continue enrollment in the Phase 1 study and provide a program update by the end of 2018

Abaloparatide
Initiate clinical bone histomorphometry study in the first half of 2018
Publication of ACTIVExtend Phase 3 data
Enter into a partnership for the potential commercialization of abaloparatide-SC outside the US and Japan
Expected Radius Presentations at Upcoming Conferences in 2Q 2018

On May 15-17, the Company will present and host one-on-one meetings at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas, Nevada.
On June 12-14, the Company will present and host one-on-one meetings at the Goldman Sachs Global Healthcare Conference in Palos Verdes, California.
First Quarter 2018 Financial Results

For the three months ended March 31, 2018, Radius reported a net loss of $61.6 million, or $1.37 per share, compared to a net loss of $56.9 million, or $1.32 per share, for the three months ended March 31, 2017.

For the three months ended March 31, 2018, Radius reported TYMLOS net product revenues of $14.5 million compared to zero TYMLOS revenue in the three months ended March 31, 2017.

Research and development expense for the three months ended March 31, 2018, was $22.9 million compared to $19.5 million for the three months ended March 31, 2017, an increase of $3.4 million, or 17%. This increase was primarily driven by a $2.5 million increase in abaloparatide-SC project costs, a $0.6 million increase in elacestrant project costs, a $0.3 million increase in RAD140 project costs, and a $0.1 million increase in abaloparatide-patch project costs. These increases were partially offset by a $0.9 million decrease in vasomotor project related spending. Additionally, there was an increase in headcount from 111 research and development employees as of March 31, 2017 to 131 research and development employees as of March 31, 2018.

For the three months ended March 31, 2018, selling, general and administrative expense was $48.0 million compared to $38.1 million for the three months ended March 31, 2017, an increase of $9.9 million, or 26%. This increase was primarily the result of a $6.6 million and $2.3 million increase in compensation and travel related expenses, respectively, due to an increase in headcount from 363 selling, general and administrative employees as of March 31, 2017 to 405 selling, general and administrative employees as of March 31, 2018.

As of March 31, 2018, Radius had $367.3 million in cash, cash equivalents and marketable securities. Based upon the Company’s cash, cash equivalents and marketable securities balance as of March 31, 2018, the Company believes that, prior to the consideration of proceeds from partnering and/or collaboration activities, it has sufficient capital to fund its development plans, U.S. commercial and other operational activities for not less than twelve months from the date of this press release.