Ziopharm Oncology Reports Fourth Quarter and Full Year 2017 Financial Results and Provides Corporate Update

On March 1, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP), a biotechnology company focused on development of next generation immunotherapies utilizing gene- and cell-based therapies to treat patients with cancer, reported its financial results for the fourth quarter and year ended December 31, 2017, and provided an update on the Company’s recent activities (Press release, Ziopharm, MAR 1, 2018, View Source [SID1234524332]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is an exciting year for Ziopharm and the clinical development of our two platform technologies, Controlled IL-12 and Sleeping Beauty," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "With Sleeping Beauty, we achieved major milestones in 2017 and are looking forward to what is ahead in 2018. We plan to move our point-of-care technology into the clinic and, for the first time ever, we intend to infuse patients with T cells genetically modified and manufactured without virus using the Sleeping Beauty platform within two days from harvesting the T cells from patients. Also, Sleeping-Beauty-modified T-cell receptors targeting neoantigens — the very mutations that cause solid tumors — will enter the clinic with Dr. Steven Rosenberg’s team at the National Cancer Institute."

Dr. Cooper continued, "With Controlled IL-12, in addition to investigating this cytokine as a monotherapy, we look forward to seeing data from our first combination of IL-12 and an immune checkpoint inhibitor, and exploring its impact on the care options for the treatment of patients with brain cancer. We believe Controlled IL-12 is a powerful platform that can turn cold tumors hot and has the potential for broad applicability across oncology."

Program Updates

Controlled IL-12

Ziopharm is advancing Ad-RTS-hIL-12 plus veledimex as a gene therapy product candidate to treat patients with recurrent glioblastoma (rGBM). Ad-RTS-hIL-12 is an adenoviral vector administered via a single injection into the tumor and engineered to conditionally express human IL-12, a powerful cytokine that has demonstrated a targeted, anti-tumor immune response. The expression of hIL-12 is controlled and modulated with the RheoSwitch Therapeutic System (RTS) by the small molecule veledimex, an activator ligand which crosses the blood-brain barrier.

Combination Trial with Checkpoint Inhibitor in rGBM Initiated. As announced earlier this year in January, Ziopharm has initiated a trial of adult patients with rGBM to evaluate a single dose of Ad-RTS-hIL-12 plus veledimex in combination with OPDIVO (nivolumab), an immune checkpoint inhibitor targeting programmed death-1 (PD-1).

Plans to Initiate Pivotal Trial in rGBM in Second Half of 2018. Ziopharm plans to initiate a pivotal trial for Ad-RTS-hIL-12 plus veledimex for the treatment of patients with rGBM in the second half of 2018. As previously disclosed, Ziopharm is currently in the process of completing Chemistry Manufacturing and Control technical requirements for a planned Phase 3 clinical trial, subject to regulatory approval.

Phase 1 Trial for Pediatric Brain 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.

Fourth Quarter Highlights

In November 2017, Ziopharm presented updated data from its Phase 1 trial of Ad-RTS-hIl-12 plus veledimex to treat patients with rGBM that supports a survival benefit and the underlying immune system mechanism at the 22nd Annual Meeting and Education Day of the Society for Neuro-Oncology.

The data showed median overall survival (mOS) of 12.5 months sustained for patients treated with Ad-RTS-hIL-12 plus 20 mg of veledimex (n=15) at a mean follow-up time of 11.1 months as of October 18, 2017. This mOS compares favorably to the 5 to 8 months survival established in historical controls for patients with rGBM. Additional highlights observed included:

An anti-tumor effect evident with centralized review of magnetic resonance imaging showing decreasing size of brain tumor lesions in patients;
Immunohistochemistry analyses from three of three patient biopsies greater than four months after completion of veledimex demonstrated that IL-12 results in an extensive infiltration of CD8+ T cells within the tumor;
These same biopsies showed sustained production of interferon-gamma, a cytokine crucial to arming an immune response in the tumor microenvironment;
Interferon-gamma was undetectable in the peripheral blood at the time of biopsies providing further evidence of an on-target T-cell response;
These same biopsies demonstrated evidence of an anti-tumor response;
These same biopsies showed upregulation of both PD-1 and PD-L1, which suggests added potential efficacy for combining Ad-RTS-hIL-12 plus veledimex with an immune checkpoint inhibitor;
Ratio of circulating killer CD8+ T cells to suppressor FOXP3+ T cells correlates with overall survival;
Patients in the trial who received low-dose systemic corticosteroids for peri-operative management have a much better survival rate than those who received higher doses of corticosteroids, as the latter presumably interferes with immune activation;
Ad-RTS-hIL-12 plus veledimex continues to be well tolerated, as adverse events (AE) in the trial were predictable and reversible, neurologic AEs were relatively mild and transient, and there were no drug-related deaths.
Adoptive Cell Therapies
Using Ziopharm’s non-viral approach leveraging Sleeping Beauty 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 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 harvesting T cells from the patient.

Phase 1 Trial of Sleeping Beauty-Modified TCRs to Treat Solid Tumors to Initiate in Second Half of 2018. The NCI anticipates initiation of a Phase 1 trial in the second half of 2018 to evaluate adoptive cell transfer (ACT)-based immunotherapies genetically modified using the Sleeping Beauty transposon/transposase system 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 ACT 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 cancer.

Phase 1 Trial of CD33-specific CAR+ T Therapy for Acute Myeloid Leukemia (AML). Enrollment is underway at MD Anderson Cancer Center 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 cetuximab-activated 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.

Fourth Quarter Update

Data supporting the third-generation point-of-care technology were presented at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017, where first- and second-generation Sleeping Beauty clinical trial data demonstrated tolerability, disease response including long-term survival, and sustained persistence of infused CD19-specific CAR+ T cells. Ziopharm also presented at ASH (Free ASH Whitepaper) preclinical data demonstrating that Sleeping Beauty can manufacture CAR+ T cells co-expressing mbIL15 in less than two days. In addition, preclinical data were also presented last month at the 2018 Keystone Symposia Emerging Cellular Therapies: T Cells and Beyond. These data further demonstrated that T cells expressing CD19-specific CAR with mbIL15 could be generated with the Sleeping Beauty system in less than two days and did not require ex vivo activation or propagation. Ziopharm observed in these trials that T cells designed to express mbIL15 showed greater persistence and more potent antitumor activity than comparator T cells without mbIL15.

Graft-versus-Host Disease (GvHD) Update
Following an in-depth review of Ziopharm’s research and development portfolio, management made the strategic decision to focus resources on developing Controlled IL-12 and Sleeping Beauty platforms for oncology indications and to stop development of engineered cell therapies for the treatment of GvHD. Ziopharm reverted its rights to the GvHD program to Precigen and is winding down its final clinical activities.

Fourth Quarter 2017 Financial Results

Net loss applicable to the common stockholders for the fourth quarter of 2017 was $18.3 million, or $(0.13) per share, compared to a net loss of $14.8 million, or $(0.11) per share, for the fourth quarter of 2016. The increase is primarily due to an increase in operating expenses of $2.3 million and an increase of $1.5 million related to the value of preferred stock dividends.
Research and development expenses were $11.2 million for the fourth quarter of 2017, compared to $9.4 million for the fourth quarter of 2016. The increase in research and development expenses for the three months ended December 31, 2017 is primarily due to expanded development in Ziopharm’s gene and cell therapy programs.
General and administrative expenses were $3.9 million for the fourth quarter of 2017, compared to $3.3 million for the fourth quarter of 2016.
Ziopharm ended the quarter with unrestricted cash resources of approximately $70.9 million.
As part of Ziopharm’s strategic co-development activities at MD Anderson Cancer Center, a prepayment of approximately $31.9 million remains available for programs to be conducted by the Company at MD Anderson Cancer Center under the current Research and Development Agreement.
Ziopharm believes its current resources will be sufficient to fund its currently planned operations into the fourth quarter of 2018.
ZIOPHARM Oncology, Inc.
Statements of Operations
(in thousands except share and per share data)

Three Months Ended Year Ended
December 31, December 31,
(unaudited) (audited)
2017 2016 2017 2016

Collaboration revenue $ 1,597 $ 1,597 $ 6,389 $ 6,861

Operating expenses:
Research and development 11,181 9,389 45,084 157,791
General and administrative 3,852 3,319 14,798 14,377
Total operating expenses 15,033 12,708 59,882 172,168

Loss from operations (13,436 ) (11,111 ) (53,493 ) (165,307 )

Other income (expense), net 166 32 465 134
Change in fair value of derivative liabilities (3 ) (145 ) (1,295 ) (124 )
Net loss (13,273 ) (11,224 ) (54,323 ) (165,297 )
Preferred stock dividends (4,999 ) (3,532 ) (18,938 ) (7,123 )
Net loss applicable to common stockholders $ (18,272 ) $ (14,756 ) $ (73,261 ) $ (172,420 )


Basic and diluted net loss per share $ (0.13 ) $ (0.11 ) $ (0.53 ) $ (1.32 )

Weighted average common shares outstanding used
to compute basic and diluted net loss per share 140,644,238 130,524,204 136,938,264 130,391,463

ZIOPHARM Oncology, Inc.
Balance Sheet Data
(in thousands)
(audited)

December 31, December 31,
2017 2016

Cash and cash equivalents 70,946 81,053
Working capital 69,927 89,075
Total assets 105,606 106,348
Total stockholders’ (deficit) (96,806 ) (77,298 )

Conference Call and Slide Webcast

Ziopharm will host a conference call and webcast slide presentation today, March 1, 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 3782628. To access the slides and live audio 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 Ziopharm’s website for two weeks.

Varian to Present at Barclays Global Healthcare Conference

On March 1, 2018 Varian (NYSE: VAR) reported that Gary Bischoping, chief financial officer, and J. Michael Bruff, senior vice president of investor relations, will present at the Barclays Global Healthcare Conference in Miami, scheduled for 9:00 a.m. ET on March 14, 2018 (Press release, Varian Medical Systems, MAR 1, 2018, View Source [SID1234524331]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Information about the webcast of the company’s presentation will be available through a link on the company website at www.varian.com/inv­estors.

Sarepta Therapeutics Announces Fourth Quarter 2017 and Full-Year 2017 Financial Results and Recent Corporate Developments

On March 1, 2018 Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, today reported financial results for the three and twelve months ended December 31, 2017 (Press release, Sarepta Therapeutics, MAR 1, 2018, View Source [SID1234524329]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In 2017, Sarepta advanced our ambitious vision to make a profound difference in the lives of those with Duchenne muscular dystrophy (DMD) and furthered our position as an important global genetic medicine company in rare disease. We accelerated our industry-leading DMD pipeline composed of some 16 programs across our RNA-targeted, gene therapy and gene-editing platforms; raised substantial resources to invest in our programs; and delivered on our commitment to execute a successful launch for EXONDYS 51, our first genetic medicine therapy for DMD," said Douglas Ingram, Sarepta’s president and chief executive officer.

Mr. Ingram continued, "The exceptional 2017 performance of EXONDYS 51 and the advancement of our multi-platform pipeline reflects our leadership position in DMD and our commitment to turning our vision into reality. As we track into 2018, our commitment to the DMD community is unwavering, exemplified by our continued focus on the launch of EXONDYS 51, multiple important data read outs over the year, and our progress in bringing new therapies to the DMD community with a sense of urgency."

Financial Results

For the fourth quarter of 2017, on a GAAP basis, Sarepta reported a net loss of $24.0 million, or $0.37 per share, compared to a net loss of $88.5 million for the same period of 2016, or $1.62 per share. On a non-GAAP basis, the net loss for the fourth quarter of 2017 was $18.0 million, or $0.28 per share, compared to a net loss of $38.6 million for the same period of 2016, or $0.71 per share.

For the year ended December 31, 2017, on a GAAP basis, Sarepta reported a net loss of $50.7 million, or $0.86 per share, compared to a net loss of $267.3 million for the same period of 2016, or $5.49 per share. On a non-GAAP basis, the net loss for 2017 was $88.7 million, or $1.51 per share, compared to a net loss of $191.9 million for the same period of 2016, or $3.94 per share.

Net Revenues

For the three and twelve months ended December 31, 2017, the Company recorded net product revenues of $57.3 million and $154.6 million, respectively, which reflects sales from EXONDYS 51 compared to net revenues of $5.4 million for fourth quarter of 2016. The Company did not achieve any product revenues for the first three quarters of 2016. The increase primarily reflects increasing demand for EXONDYS 51 in the U.S.

Cost and Operating Expenses

Cost of sales (excluding amortization of in-licensed rights)

For the three and twelve months ended December 31, 2017, cost of sales (excluding amortization of in-licensed rights) were $3.5 million and $7.4 million, respectively, compared to $0.1 million for the same periods of 2016. The increase primarily reflects royalty payments to BioMarin Pharmaceuticals (BioMarin) as a result of the execution of the settlement and license agreements with BioMarin in July 2017 as well as higher inventory costs related to increasing demand for EXONDYS 51 during 2017. Prior to the approval of EXONDYS 51, the Company expensed related manufacturing and material costs as research and development expenses.

Research and development

Research and development expenses were $44.4 million for the fourth quarter of 2017, compared to $70.7 million for the same period of 2016, a decrease of $26.3 million. The decrease was primarily driven by an up-front payment of $40.0 million to Summit (Oxford) Ltd. (Summit) in the fourth quarter of 2016 partially offset by increased patient enrollment in the Company’s on-going late stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons. Non-GAAP research and development expenses were $41.8 million for the fourth quarter of 2017, compared to $27.8 million for the same period of 2016, an increase of $14.0 million. The increase was primarily due to increased patient enrollment in the Company’s on-going late-stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons.

Research and development expenses were $166.7 million for 2017, compared to $188.3 million for the same period of 2016, a decrease of $21.6 million. The decrease was primarily driven by lower manufacturing expenses due to the capitalization of inventory following the approval of EXONDYS 51 and $40.0 million and $7.0 million up-front payments, respectively, to Summit and University of Western Australia in 2016. The decreases were partially offset by a $22.0 million payment to Summit in 2017 as a result of achieving the milestone of the last patient being dosed in the safety arm cohort to the PhaseOut DMD study, increased patient enrollment in the Company’s ongoing late-stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons and increases in professional fees and compensation and other personnel expenses. Non-GAAP research and development expenses were $136.0 million for 2017, consistent with the same period of 2016.

Selling, general and administration

Selling, general and administrative expenses were $32.2 million for the fourth quarter of 2017, compared to $22.9 million for the same period of 2016, an increase of $9.3 million. Non-GAAP selling, general and administrative expenses were $27.3 million for the fourth quarter of 2017, compared to $16.1 million for the same period of 2016, an increase of $11.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administrative expenses for the fourth quarter were primarily driven by increases in professional services due to global expansion and compensation and other personnel expenses.

Selling, general and administrative expenses were $122.7 million for 2017, compared to $83.7 million for the same period of 2016, an increase of $39.0 million. Non-GAAP selling, general and administrative expenses were $97.9 million for 2017, compared to $60.7 million for the same period of 2016, an increase of $37.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administration expenses for the full year were primarily driven by increases in professional services due to global expansion, legal expenses and compensation and other personnel expenses.

EXONDYS 51 litigation and license charges and amortization of in-licensed rights

As a result of the execution of the settlement and license agreements with BioMarin in July 2017, the Company recorded $28.4 million in litigation and license charges. Additionally, the Company recognized an amortization of in-licensed rights of $1.1 million during 2017, primarily due to the BioMarin transactions.

Other Income (Loss)

Gain from sale of Priority Review Voucher

In connection with the completion of the sale of the Priority Review Voucher (PRV) in March 2017, the Company recorded a gain of $125.0 million from sale of PRV for 2017.

Interest expense and other, net

For the three and twelve months ended December 31, 2017, the Company recorded $2.7 million and $2.0 million, interest expense and other, net, respectively, compared to less than $0.1 million and $0.5 million, respectively, for the same periods of 2016. The year-over-year increases for both periods were primarily driven by accrued interest expense related to the convertible note that the Company issued in November 2017.

Cash, Cash Equivalents, Restricted Cash and Investments

The Company had $1.1 billion in cash, cash equivalents, restricted cash and investments as of December 31, 2017 compared to $329.3 million as of December 31, 2016, an increase of $760.5 million. The increase is primarily driven by the net proceeds from the Company’s equity and debt offerings, proceeds from the sale of the Company’s PRV and collection of accounts receivable related to EXONDYS 51 sales offset by up-front payments of $35.0 million related to the Company’s license and settlement agreements with BioMarin and a milestone payment of $22.0 million to Summit Therapeutics, and the use of cash to fund the Company’s ongoing operations.

Use of Non-GAAP Measures

In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements: non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for stock-based compensation, restructuring expenses, and other items.

1. Stock-based compensation expenses

Stock-based compensation expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially from period-to-period and depend significantly on factors that are not a direct consequence of operating performance that is within management’s control. Therefore, management believes that excluding these charges facilitates comparisons of the Company’s operational performance in different periods.

2. Restructuring expenses

Restructuring expenses have been excluded as the Company believes that adjusting for these items more closely represents the Company’s ongoing operating performance and financial results.

3. Other items

Management evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relates to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue on a regular basis. These other items include the aforementioned gain from the sale of the Company’s PRV and associated income taxes, upfront license and milestone payments to Summit, EXONDYS 51 litigation and license charges and amortization of in-licensed rights.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP to Non-GAAP Net Loss."

Recent Corporate Developments

— Sarepta Therapeutics Pre-Announces Fourth Quarter 2017 Revenue and Provides Full-Year 2018 Revenue Guidance for EXONDYS 51 (eteplirsen), Representing Approximately 100 Percent Year-over-Year Growth

— Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases

— Sarepta Therapeutics Elects Biopharmaceutical Veteran, Michael W. Bonney, to its Board of Directors

— Sarepta Therapeutics Announces Exercise of Initial Purchasers’ Option to Purchase Additional Convertible Senior Notes Due 2024

— Sarepta Therapeutics Prices $475 Million of Convertible Senior Notes Due 2024

— Sarepta Therapeutics Announces Proposed Offering of $375 Million of Convertible Senior Notes Due 2024

— Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051

— Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

— Sarepta Therapeutics and Nationwide Children’s Hospital Announce U.S. Food and Drug Administration (FDA) Clearance of the IND Application for the GALGT2 Gene Therapy Program

— Sarepta Therapeutics Signs Exclusive Global Collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD)

Conference Call

The Company will be hosting a conference call at 4:30 p.m. Eastern Time, to discuss these financial results and provide a corporate update. The conference call may be accessed by dialing 844-534-7313 for domestic callers and +1-574-990-1451 for international callers. The passcode for the call is 7396848. Please specify to the operator that you would like to join the "Sarepta Fourth Quarter and Full-Year 2017 Earnings Call". The conference call will be webcast live under the investor relations section of Sarepta’s website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About EXONDYS 51

EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Important Safety Information About EXONDYS 51

Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ³30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ³10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the full Prescribing Information.

Rigel Announces Two Fostamatinib Presentations at the 4th Biennial Summit of the Thrombosis & Hemostasis Societies of North America

On March 1, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), reported that fostamatinib data from its FIT Phase 3 extension study (FIT3) in patients with chronic immune thrombocytopenia (ITP) and preliminary data from its SOAR Phase 2 clinical study in patients with warm antibody autoimmune hemolytic anemia (AIHA) will be presented at the 4th Biennial Summit of the Thrombosis & Hemostasis Societies of North America on March 8 – 10, 2018 in San Diego, CA (Press release, Rigel, MAR 1, 2018, View Source;p=RssLanding&cat=news&id=2335578 [SID1234524328]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fostamatinib disodium is an oral investigational drug candidate designed to inhibit spleen tyrosine kinase (SYK), a key signaling component of the body’s immune process that leads to platelet destruction in ITP and red blood cell destruction in AIHA. SYK inhibition by fostamatinib can affect antibody-mediated platelet destruction, an underlying cause of ITP. Fostamatinib may also impair antibody-mediated red blood cell destruction in AIHA, a rare disease for which there are no approved therapies.

Fostamatinib Oral Presentation
Date and Time: Saturday, March 10, 10:15am PT
Location: Marriott Grand Ballroom 10, Marriott Marquis, San Diego
Title: Platelet Responses in Placebo Patients with Long-Duration Chronic Immune Thrombocytopenia Initiating Fostamatinib in the 049 Open-Label Extension Study

Fostamatinib Poster Presentation
Date and Time: Thursday, March 8, 3:30-4:15pm PT
Location: Pacific Ballroom, Marriott Marquis, San Diego
Title: Fostamatinib, a Spleen Tyrosine Kinase Inhibitor, is Active in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia: Results of the SOAR Phase 2, Multicenter, Open-Label Study
ePoster #35 (Electronic poster)

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the US and can be a severe, debilitating disease. To date, there are no FDA approved disease-targeted therapies for AIHA, despite the tremendous medical need that exists for these patients.

Radius Health Reports Fourth Quarter and Full Year 2017 Financial and Operating Results and Provides Business Update

On March 1, 2018 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), today reported its financial results for the fourth quarter and full year ended December 31, 2017, and provided a business update (Press release, Radius, MAR 1, 2018, View Source [SID1234524327]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our fourth-quarter and full year results highlight the great performance of TYMLOS which we expect to become the leader in the anabolic market," said Jesper Høiland, President and Chief Executive Officer of Radius.

"I’m also very pleased to announce that in January we aligned with the FDA on a regulatory path for our investigational abaloparatide patch, a novel technology with potential to provide a convenient, alternative treatment option for osteoporosis patients. This innovation is aimed at expanding both the abaloparatide and total anabolic addressable markets. We are also excited to announce that we have entered into a commercial supply agreement with 3M Company under which 3M has agreed to exclusively manufacture commercial supplies of abaloparatide patches," Mr. Høiland continued.

"Following the strong single-agent activity seen in our elacestrant Phase 1 study, we have finalized our global development strategy based on recent feedback from the EMA and the FDA. We expect to initiate a global, randomized, comparator-controlled Phase 2 study of elacestrant as a third-line monotherapy for ER-positive/HER2-negative advanced or metastatic breast cancer in the second half of 2018. Depending on the study results, this single trial could support applications for global marketing approvals."

"I believe our clinical progress and operational performance during 2017 have built solid momentum for a strong 2018 for Radius," Mr. Høiland concluded.

TYMLOS (abaloparatide injection)

Fourth-quarter sales of TYMLOS in the U.S. (the second full quarter since its launch) were $7.7 million. Radius received FDA approval for TYMLOS on April 28, 2017 for the treatment of postmenopausal women with osteoporosis at high risk of fracture and began shipments to wholesalers at the end of May 2017.
Nine months after launch, TYMLOS has surpassed the level of commercial market access for the competing anabolic product, with approximately 259 million covered lives and 93% coverage in commercial plans. TYMLOS coverage in Medicare Part D plans has also increased to 41%.

TYMLOS market share continued to increase in the fourth quarter to approximately 32% of new patients starting anabolic therapy (NBRx) and 13% of total prescriptions in the anabolic market. The anabolic market also showed growth in 2017, for the first time since 2012.

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

In the fourth quarter, a labeling supplement for TYMLOS was submitted to the FDA with the positive 43-month data from the ACTIVExtend study, which showed continued significant risk reduction in fractures in patients who were transitioned to receive an additional 24 months of bisphosphonate therapy.
Pipeline Updates

Abaloparatide-Transdermal Patch (TD)

In January 2018, Radius met with the FDA to align on a regulatory development path for the abaloparatide patch. The FDA agreed that, depending on the study results, a single, randomized, open label, active-controlled, non-inferiority (to abaloparatide-SC) study of up to 500 patients with postmenopausal osteoporosis at high risk of fracture would be sufficient to gain approval for the abaloparatide patch. The primary endpoint in the study will be change in lumbar spine bone mineral density (BMD) at 12 months. The Company expects to initiate this pivotal trial in mid-2019.

In addition, on February 27, 2018, Radius entered into a scale-up and commercial supply agreement with 3M in which 3M agreed to exclusively manufacture commercial supplies of abaloparatide patches. As part of the agreement, in addition to per unit transfer prices, Radius will pay 3M a mid-to-low single digit royalty on worldwide net sales of abaloparatide patches and reimburse 3M for certain capital expenditures incurred to establish commercial supply of abaloparatide patches.
Abaloparatide — Subcutaneous (SC)

European MAA

Radius’ European Marketing Authorization Application (MAA) for abaloparatide-SC for the treatment of postmenopausal women with osteoporosis is under review by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). On December 15, 2017, the CHMP issued a third Day-180 List of Outstanding Issues. As part of its ongoing risk-benefit assessment, the CHMP informed the Company that it intends to refer the MAA to a scientific advisory group for additional advice. The Company expects an opinion from the CHMP regarding the MAA in the first half of 2018.
Male Osteoporosis Trial

In the fourth quarter, the Company gained agreement with the FDA on the design of a clinical trial in men with osteoporosis, which, if successful, will form the basis of an sNDA seeking to expand the use of TYMLOS to treat men with osteoporosis at high risk for fracture. The study will be a randomized, double-blind, placebo-controlled trial that will enroll approximately 225 men with osteoporosis. The primary endpoint is change in spine BMD at 12 months compared with placebo. The study is expected to be initiated in the first quarter of 2018.
Elacestrant (RAD1901)

An update on data as of October 30, 2017 from the Phase 1 clinical study was presented at the San Antonio Breast Cancer Symposium in December 2017, with the elacestrant 400mg single agent showing an objective response rate of 27.3%, a clinical benefit rate at 24 weeks of 47.4% and median progression free survival of 5.4 months in heavily treated, ER-positive/HER-2 negative advanced breast cancer patients. The results showed that elacestrant was well tolerated with low grade nausea and dyspepsia being the most commonly reported adverse events.

In October 2017, elacestrant received an FDA Fast Track designation. In February 2018, we received scientific advice from the EMA regarding a potential single-arm monotherapy Phase 2 trial of elacestrant in patients with ER-positive/HER2-negative advanced or metastatic breast cancer, and we had a meeting with the FDA regarding the registrational pathway for elacestrant at which we confirmed FDA’s guidance for a single-arm study and gained alignment on an alternative potential comparator study design. Based on feedback from the EMA and the FDA, we now intend to conduct a single, randomized, controlled Phase 2 trial of elacestrant as a third-line monotherapy in approximately 300 patients with ER positive/HER2 negative advanced/metastatic breast cancer. Patients in the study would be randomized to receive either elacestrant or the investigator’s choice of an approved hormonal agent and the primary endpoint of the study will be progression-free survival (PFS). The study would also include a planned interim PFS analysis. We believe that, depending on results, this single trial would support applications for global marketing approvals for elacestrant as a third-line monotherapy, including potentially accelerated approval for elacestrant in the United States. We will provide further study details when the Phase 2 study is started, which we expect will be in the second half of 2018.

Following a strategic review in December 2017, the elacestrant development program for vasomotor symptoms was discontinued.
RAD140

In September 2017, a Phase 1 study of RAD140, a nonsteroidal selective androgen receptor modulator (SARM), was initiated in patients with hormone receptor-positive, locally advanced or metastatic breast cancer. The clinical trial is designed to evaluate the safety and maximum tolerated dose of RAD140 in approximately 40 patients. We expect to provide an update on our RAD140 development program by the end of 2018.
Corporate Update

In November 2017, we announced the appointment of Joseph Kelly as Senior Vice President of Sales and Marketing, and the promotion of Amanda Mott to Senior Vice President of Market Access.

Upcoming Milestones

Abaloparatide-SC
– Initiate a male osteoporosis study in the first quarter of 2018
– Receive Committee for Medicinal Products for Human Use (CHMP) opinion regarding the EMA’s review of the abaloparatide-SC MAA 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
Elacestrant
– Initiate a potentially pivotal Phase 2 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

RAD140
– Continue enrollment in the Phase 1 study and provide a program update by the end of 2018.
Expected Radius Presentations at Investor Conferences in 1H 2018:

On March 12-14, the Company will present and host one-on-one meetings at the 38th Cowen Annual Healthcare Conference in Boston, Mass.
On May 8-9, the Company will present and host one-on-one meetings at the Deutsche Bank Annual Healthcare Conference in Boston, Mass.
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, NV.
On June 12-14, the Company will present and host one-on-one meetings at the Goldman Sachs Global Healthcare Conference in Palos Verdes, CA.
Fourth Quarter 2017 Finance Review

For the three months ended December 31, 2017, Radius reported a net loss of $71.0 million, or $1.59 per share, compared to a net loss of $52.7 million, or $1.22 per share, for the three months ended December 31, 2016.

For the three months ended December 31, 2017, Radius reported TYMLOS net product revenues of $7.7 million, which reflects the second full quarter of recorded sales. Radius had no revenue in the three months ended December 31, 2016 as the FDA approved TYMLOS on April 28, 2017.

Research and development expense for the three months ended December 31, 2017, was $22.9 million compared to $25.6 million for the three months ended December 31, 2016, a decrease of $2.7 million or 10.5 percent, primarily attributable to a decrease in R&D costs associated with the development of TYMLOS and elacestrant.

Selling, general, and administrative expense for the three months ended December 31, 2017, was $50.7 million compared to $27.5 million for the three months ended December 31, 2016, an increase of $23.2 million or 84.4 percent primarily due to personnel, promotional, and consulting expenses related to the launch of TYMLOS.

Full Year 2017 Finance Review

For the twelve months ended December 31, 2017, Radius reported a net loss of $254.2 million, or $5.80 per share, compared to a net loss of $182.8 million, or $ 4.24 per share, for the twelve months ended December 31, 2016.

For the twelve months ended December 31, 2017 Radius reported TYMLOS net product revenues of $12.1 million. Radius had no revenue in the twelve months ended December 31, 2016 as the FDA approved TYMLOS on April 28, 2017.

Research and development expense for the twelve months ended December 31, 2017, was $83.1 million compared to $107.4 million for the twelve months ended December 31, 2016, a decrease of $24.3 million or 22.6 percent primarily due to the reduction in R&D expenses associated with the elacestrant projects and the development of TYMLOS.

Selling, general, and administrative expense for the twelve months ended December 31, 2017, was $186.7 million compared to $77.5 million for the twelve months ended December 31, 2016, an increase of $109.2 million, or 140.9%, primarily attributable to personnel and other support costs resulting from the commercial launch of TYMLOS.

As of December 31, 2017, Radius had $430.3 million in cash, cash equivalents and marketable securities. Based upon our cash, cash equivalents and marketable securities balance, we believe that, prior to the consideration of proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for not less than twelve months from the date of this press release.

Webcast and Conference Call

In connection with today’s reporting of Fourth Quarter and Full Year Financial Results, Radius will host a conference call and live audio webcast at 4:30 p.m. ET on Thursday, March 1, 2018 to discuss the commercial outlook for TYMLOS, review the financial results and provide a Company update.

Webcast Information:

Date: Thursday, March 1, 2018

Time: 4:30 p.m. ET

Live webcast: View Source

A replay of the webcast will be available on the Company’s website, www.radiuspharm.com in the Investor section under Events and Presentations for 7 days following the live webcast.

Conference Call Information:

Domestic Dial-in Number: (866) 323-7965

International Dial-in Number: (346) 406-0961

Conference ID: 3484256

For those unable to participate in the conference call or webcast, a replay will be available beginning March 1, 2018, at 7:30 p.m. ET until March 8, 2018 at 7:30 p.m. ET. To access the replay, dial (855) 859-2056 for U.S. or (404) 537-3406 for International. The replay pin number is 3484256.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com, where a webcast replay will be also available for 14 days. The full text of the announcement and financial results will also be available on the Company’s website.