1stOncology™: Cancer Intelligence Service – Page 14979 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Momenta Pharmaceuticals Reports Second Quarter 2018 Financial Results

On August 9, 2018 Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA) reported its financial results for the second quarter ended June 30, 2018 and provided a corporate update (Press release, Momenta Pharmaceuticals, AUG 9, 2018, View Source [SID1234528838]).

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!

"The strategic review of our business announced earlier this year is progressing and we are actively evaluating options for our biosimilars business. We expect to complete this review and announce an outcome in the coming weeks," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals. "Our novel drug candidates for rare autoimmune diseases continue to advance. We remain on track to initiate two Phase 2 proof of concept studies of M281 in the fourth quarter of 2018 and a Phase 1/2 proof of concept study of M254, our hyper-sialylated IVIg candidate, in late 2018 or early 2019, pending regulatory feedback."

Second Quarter Highlights and Recent Events

Complex Generics:

Glatopa Products: a fully substitutable, AP-rated generic version of three-times-a-week COPAXONE 40 mg/mL and daily COPAXONE 20 mg/mL (glatiramer acetate injection) for patients with relapsing forms of multiple sclerosis developed in collaboration with Sandoz

In the second quarter of 2018, Momenta recorded $11.8 million in product revenues from Sandoz’s sales of Glatopa 20 mg/mL and 40 mg/mL products.

Enoxaparin Sodium Injection: First FDA-approved, substitutable generic LOVENOX (Enoxaparin Sodium Injection) used for the prevention and treatment of deep vein thrombosis developed in collaboration with Sandoz

In June 2018, Sandoz alerted its customers and the U.S. Food and Drug Administration (FDA) that it will be discontinuing the supply of the Enoxaparin Sodium Injection product.

Biosimilars:

M923: a fully-owned proposed biosimilar to HUMIRA (adalimumab)

· In January 2018, the Company announced that the Biologics License Application (BLA) for M923 is prepared to be filed with the FDA.

·In June 2018, Momenta announced that it had reached a contract amendment to an agreement with Human Genome Sciences, Inc. ("GSK"), the supplier of product for M923, in which Momenta will pay GSK $15.0 million by August 15, 2018 and $15.0 million by July 1, 2019 as part of Momenta’s contractual commitments for the purchase of product batches for M923 through 2022.

M834: a proposed biosimilar to ORENCIA (abatacept) being developed in collaboration with Mylan

·In late 2017, Momenta announced that M834 did not meet its primary pharmacokinetic endpoints in a Phase 1 study to compare the pharmacokinetics, safety and immunogenicity of M834 to US- and EU-sourced ORENCIA in normal healthy volunteers. Momenta and Mylan continue to investigate these results in order to determine the next steps for the program.

M710: a proposed biosimilar to EYLEA (aflibercept) candidate being developed in collaboration with Mylan

·In January 2018, Momenta and Mylan disclosed that M710 is a proposed biosimilar to EYLEA and announced an IND acceptance by the FDA. The two companies are in the process of initiating the pivotal clinical trial in patients and the trial is progressing according to plan.

Novel Drugs for Rare Autoimmune and Immune-mediated Diseases:

M281 (anti-FcRn): a fully human anti-neonatal Fc receptor (FcRn) aglycosylated immunoglobulin G (IgG1) monoclonal antibody (mAb)

·Momenta is finalizing its development strategy for M281 and planning two Phase 2 proof of concept clinical trials in the fourth quarter of 2018, pending regulatory feedback.

M230 (CSL730): a recombinant Fc multimer being developed in collaboration with CSL

·CSL’s Phase 1 study in healthy volunteers to evaluate the safety and tolerability of M230 is ongoing and is targeted to be completed in 2019.

M254 (hsIVIg): a hyper-sialylated immunoglobulin designed to be a higher potency alternative to intravenous immunoglobulin (IVIg) with the potential for enhanced safety and convenience

·The Company has successfully completed the IND-enabling toxicology study of M254 and is targeting the initiation of a Phase 1/2 proof of concept study in late 2018 or early 2019, pending regulatory feedback.

Second Quarter 2018 Financial Results

Revenue: In the second quarter of 2018, the Company recorded $11.8 million in product revenues from Sandoz’s sales of Glatopa 20 mg/mL and 40 mg/mL products compared to $19.1 million in profit share from Sandoz sales of Glatopa 20 mg/mL for the same period in 2017, net of a deduction of $0.6 million for reimbursement to Sandoz of the Company’s share of Glatopa-related legal expenses. The decrease in product revenues of $7.3 million, or 38%, was primarily due to lower net sales driven by market decline and Mylan N.V.’s entry into the COPAXONE market.

Research and development revenue for the second quarter of 2018 was $1.3 million compared to $4.4 million recorded in the same quarter last year. The decrease in research and development revenue of $3.1 million, or 70%, was primarily due to lower revenue recognized from the Mylan upfront payment as compared to the amount recognized in the 2017 period and lower reimbursable expenses for the Company’s complex generic programs.

Total revenues for the second quarter of 2018 were $13.0 million compared to $23.6 million for the same period in 2017.

Operating Expenses: Total GAAP operating expenses were $83.9 million in the second quarter of 2018.

Research and development expenses for the second quarter of 2018 were $31.3 million, compared to $39.1 million for the same period in 2017. The decrease of $7.8 million, or 20%, was primarily due to a decrease in external R&D expenses for M923 offset by increases in spending for M281 and M230.

General and administrative expenses for the second quarter of 2018 were $22.5 million, compared with $22.6 million for the same period in 2017.

During the second quarter, Momenta entered into a contract amendment to an agreement with GSK, the supplier of product for M923, for which Momenta will pay GSK $30.0 million as

part of Momenta’s contractual commitments for the purchase of product batches for M923 through 2022. The amount has been included in operating expenses for the quarter. As a result, second quarter non-GAAP operating expense was $78.0 million. Excluding the expense associated with the amendment of the M923 agreement, non-GAAP operating expense would be $48.0 million. Momenta previously provided guidance of $45 – $55 million for non-GAAP operating expense for the second quarter. Non-GAAP operating expense is total operating expenses (which excludes collaboration expenses reimbursable by Mylan), less stock-based compensation expense and collaborative reimbursement revenues. See "Non-GAAP Financial Information and Other Disclosures" and the table below entitled "Reconciliation of GAAP Results to Non-GAAP Financial Measures" for a reconciliation of GAAP operating expense to non-GAAP operating expense.

Net Loss: The Company reported a net loss of $69.9 million, or $0.91 per share for the second quarter of 2018 compared to a net loss of $36.9 million, or $0.50 per share for the same period in 2017.

Cash Position: At June 30, 2018, Momenta had $321.2 million in cash, cash equivalents and marketable securities compared to $346.0 million at March 31, 2018.

2018 Financial Guidance

Due to the ongoing strategic review of the business, Momenta’s 2018 operating expense guidance is no longer accurate and may continue to change subject to the outcome of the Company’s strategic review. Momenta plans to provide updated 2018 operating expense guidance for 2018 when it reports its third quarter 2018 financial results.

Non-GAAP Financial Information and Other Disclosures

Momenta uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Momenta believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Momenta’s operating performance as it excludes non-cash stock compensation expense and collaborative reimbursement revenues. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Momenta’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Momenta’s operating results as reported under GAAP. Momenta has not provided GAAP reconciliation for its forward-looking non-GAAP annual or quarterly operating expense because Momenta cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure. The Company has provided the estimated reconciling information

that is available without unreasonable effort in the section of this press release above entitled "2018 Financial Guidance."

Conference Call Information

Management will host a conference call and webcast today at 8:00 am ET to discuss these results and provide an update on the Company. A live webcast of the conference call may be accessed on the "Investors" section of the Company’s website, www.momentapharma.com. An archived version of the webcast will be posted on the Momenta website approximately two hours after the call.

To access the call you may also dial (877) 224-9084 (domestic) or (720) 545-0022 (international) prior to the scheduled conference call time and provide the access code 8687156. A replay of the call will be available approximately two hours after the conclusion of the call and will be accessible through August 16, 2018. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and provide the access code 8687156.

Molecular Templates, Inc. Reports Second Quarter 2018 Financial Results

On August 9, 2018 Molecular Templates, Inc. (Nasdaq:MTEM, "Molecular" or "Molecular Templates"), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the second quarter of 2018 (Press release, Molecular Templates, AUG 9, 2018, View Source [SID1234528837]). As of June 30, 2018, Molecular’s cash and cash equivalents totaled $41.6 million. Molecular’s current cash balance is expected to fund operations into 4Q2019.

"We have been very encouraged by the clinical results generated to date with MT-3724 in heavily-pretreated diffuse large B-cell lymphoma patients and we are excited to initiate multiple Phase II studies for this program by year-end, which will start yielding clinical results in 2019," said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Scientific Officer. "Furthermore, we expect to advance three new ETBs into clinical trials in the next twelve months."

Company Highlights and Upcoming Milestones

Corporate

At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2018, interim results from a Phase I and Phase Ib extension study of MT-3724 (an ETB targeting CD20) in B-cell non-Hodgkin’s lymphoma (NHL) patients were presented. These results included a preliminary objective response rate in diffuse large B-cell lymphoma (DLBCL) patients with low serum Rituxan levels at study entry (N=10) of 30%, with a disease control rate of 70%, including two stable disease patients that had tumor reductions of 47% and 49%.
Also at the ASCO (Free ASCO Whitepaper) annual meeting in June 2018 was a poster presentation of the pharmacokinetic profile of evofosfamide in patients with advanced pancreatic cancer. The data presented showed that there was a significant reduction in drug exposure observed between the Phase II ("404") study and the Phase III ("MAESTRO") trials, which used a new ethanol-based formulation of evofosfamide. Molecular plans to explore potential partnership opportunities for further development of evofosfamide.
MT-3724

Molecular expects to initiate Phase II combination studies with MT-3724 in earlier lines of DLBCL in 2H18.
Molecular also expects to provide an update on the Phase Ib extension study in 4Q18 and to start a Phase II monotherapy study at the end of 2018 which has the potential to be a pivotal study.
MT-4019

MT-4019 (an ETB candidate designed to target CD38-expressing myeloma cancer cells) has completed IND enabling studies.
Takeda and Molecular are evaluating CD38 ETBs and could potentially select a drug candidate for development by the end of 3Q18. If the two companies do not select a joint candidate for development, Molecular anticipates filing an IND application for MT-4019 in 3Q18 and initiating a Phase I clinical trial in 2H18.
Research

Molecular expects to file an IND application for an ETB targeting HER2 in 1Q19.
Molecular expects to file an IND application for an ETB targeting PD-L1 (with antigen seeding) in 3Q19.
Several other ETB candidates are in pre-clinical development, targeting both solid and hematological cancers.
Takeda Multi-Target Collaboration

In December 2017, Takeda selected two targets for further research using Molecular’s ETBs. This triggered $4.0 million in milestone payments, which were paid by Takeda in 2Q18.
Financial Results

The net loss attributable to common shareholders for the second quarter of 2018 was $9.7 million, or $0.36 per basic and diluted share. This compares with a net loss attributable to common shareholders of $4.5 million, or $20.76 per basic and diluted share, for the same period in 2017.

Revenues for the second quarter of 2018 were $1.4 million, compared to $42,000 for the same period in 2017. Revenues for the second quarter of 2018 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the second quarter of 2018 were $7.7 million, compared with $1.2 million for the same period in 2017. Total general and administrative expenses for the second quarter of 2018 were $3.7 million, compared with $2.4 million for the same period in 2017.

The net loss attributable to common shareholders for the six months ended June 30, 2018 was $18.4 million, or $0.68 per basic and diluted share. This compares with a net loss attributable to common shareholders of $6.1 million, or $28.32 per basic and diluted share, for the same period in 2017.

Revenues for the six months ended June 30, 2018 were $1.8 million, compared to $1.9 million for the same period in 2017. Revenues for the six months ended June 30, 2018 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the six months ended June 30, 2018 were $14.4 million, compared with $2.3 million for the same period in 2017. Total general and administrative expenses for the six months ended June 30, 2018 were $6.6 million, compared with $4.2 million for the same period in 2017.

Fortress Biotech Reports Second Quarter 2018 Financial Results and Recent Corporate Highlights

On August 9, 2018 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the second quarter ended June 30, 2018 (Press release, Fortress Biotech, AUG 9, 2018, View Source [SID1234528835]).

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "During the second quarter, our Fortress Company subsidiaries reported significant value-driving milestones, including positive Phase 3 data from Avenue Therapeutics’ IV tramadol, which, if approved, would be the only Schedule IV intravenous opioid in the U.S. and could replace highly addictive Schedule II narcotics in many patients with moderate to moderately severe postoperative pain. Additionally, Cyprium Therapeutics was granted FDA Fast Track Designation for its CUTX-101 Copper Histidinate injection in patients with Menkes disease, a rare pediatric disease with no FDA-approved treatments. Also during the quarter, Mustang Bio expanded its infrastructure with the launch of a proprietary 27,000 sq. ft. CAR T cell manufacturing facility that will enable us to oversee product safety from needle-to-needle and help improve supply chain efficiencies from clinical development into commercialization."

Dr. Rosenwald continued, "We believe in the value proposition represented by our company and strive to protect the best interests of our shareholders and those of our subsidiaries. As we continue to build long-term value, our novel and efficient business model provides benefits for all stakeholders and offers unique synergies not typical of traditional biopharma companies."

Financial Results:

·As of June 30, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), cash deposits with clearing organizations and restricted cash totaled $151.8 million, compared to $168.3 million as of December 31, 2017, a decrease of $16.5 million year-to-date.
·Net revenue totaled $63.8 million for the second quarter of 2018, compared to $50.7 million for the second quarter of 2017. Total revenue as of June 30, 2018, includes $6.8 million of Fortress revenue, primarily from the sale of Journey Medical Corporation products, and $57.0 million of revenue from National Holdings Corporation1 ("National Holdings"). Total revenue as of June 30, 2017, included $4.4 million of Fortress revenue and $46.3 million of revenue from National Holdings.
·Research and development expenses were $17.5 million for the second quarter of 2018, of which $15.1 million was related to Fortress Companies. This compares to $11.7 million for the second quarter of 2017, of which $9.5 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in research and development were $0.8 million for the second quarter of 2018, compared to $2.4 million for the second quarter of 2017.
Research and development expenses from license acquisitions were nominal for the second quarter of 2018, compared to $1.8 million for the second quarter of 2017.
·General and administrative expenses were $13.1 million for the second quarter of 2018, of which $7.7 million was related to Fortress Companies. This compares to $11.1 million for the second quarter of 2017, of which $6.6 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.4 million for the second quarter of 2018, compared to $2.2 million for the second quarter of 2017.
·National Holdings’ operating expenses totaled $56.2 million for the second quarter of 2018, compared to $48.4 million for the second quarter of 2017.
·Net loss attributable to common stockholders was $21.6 million, or $0.50 per share, for the second quarter of 2018, compared to a net loss attributable to common stockholders of $17.4 million, or $0.43 per share, for the second quarter of 2017. For the first six months of 2018, net loss was $42.6 million or $0.99 per share, compared to $29.3 million or $0.73 per share in the first six months of 2017.

1Fortress acquired approximately 56 percent of National Holdings in September 2016.

Recent Fortress and Fortress Company Highlights:

Fortress Biotech, Inc.

·In June 2018, data from a Phase 1 trial evaluating Fortress’ CNDO-109-activated allogeneic natural killer (NK) cells in acute myeloid leukemia (AML) patients were published in the journal Biology of Blood and Marrow Transplantation. The data demonstrated that CNDO-109-activated NK cells are safe, well tolerated and may be capable of extending complete remissions in high-risk AML patients.

Aevitas Therapeutics, Inc.

·In August 2018, Aevitas announced that it entered a sponsored research agreement with the laboratory of Wenchao Song, Ph.D., at the University of Pennsylvania to evaluate Aevitas’ adeno-associated virus ("AAV") gene therapy technology in Penn’s proprietary animal models of complement-mediated diseases.

Avenue Therapeutics, Inc.

·In May 2018, Avenue announced that its first pivotal Phase 3 trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 48 hours (SPID48) compared to placebo in patients with moderate to moderately severe postoperative pain following bunionectomy surgery. In addition, the trial met its key secondary endpoints and demonstrated a clear dose response. Avenue plans to initiate a second pivotal Phase 3 trial of IV tramadol in patients following abdominoplasty surgery in the second half of 2018.

Caelum Biosciences, Inc.

·In June 2018, Caelum announced a complete analysis of cardiac data from a Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory amyloid light chain ("AL") amyloidosis demonstrating CAEL-101’s potential to improve myocardial function as assessed by global longitudinal strain and generate a sustained decrease in N-terminal pro-brain natriuretic peptide levels in AL amyloidosis patients experiencing cardiac involvement. The data were presented by Columbia University at the American Society of Echocardiography 29th Annual Scientific Sessions.

Checkpoint Therapeutics, Inc.

·In April 2018, preclinical data was presented on Checkpoint’s BET inhibitor, CK-103, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. CK-103 demonstrated combinatorial effects in an in vivo model with anti-PD-1 antibodies, which may support the development of CK-103 as an anti-cancer agent alone and in combination with Checkpoint’s anti-PD-L1 antibody CK-301.

Cyprium Therapeutics, Inc.

·In July 2018, Cyprium announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to CUTX-101, a product candidate for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.

Mustang Bio, Inc.

·In May 2018, Mustang announced the publication of preclinical data in JCI Insight demonstrating that glioblastoma-targeted CD4+ CAR T cells mediate superior antitumor activity over CD8+ CAR T cells. The data, published by research partner City of Hope, will be applied in the ongoing Phase 1 trial of Mustang’s IL13Rα2-specific CAR T MB-101 in glioblastoma.
·In June 2018, Mustang opened a proprietary CAR T cell therapy manufacturing facility at UMass Medicine Science Park in Worcester, Mass. The facility will support the clinical development and commercialization of Mustang’s CAR T product candidates and enable proprietary cell therapy research.
·Also in June 2018, Mustang was added to the Russell 2000, 3000 and Microcap Indexes.
·In July 2018, Mustang completed a pre-Investigational New Drug (pre-IND) meeting with the FDA for MB-102 (CD123 CAR T). Based on the meeting, Mustang expects to file an IND in the fourth quarter of 2018 to support a Phase 1/2 trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome.

Sesen Bio Announces Vicinium Granted Fast Track Designation by FDA for Treatment of Non-Muscle Invasive Bladder Cancer

On August 9, 2018 Sesen Bio, Inc. (Nasdaq: SESN), a late-stage clinical company developing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Vicinium for the treatment of BCG-unresponsive high-grade non-muscle invasive bladder cancer (NMIBC) (Press release, Eleven Biotherapeutics, AUG 9, 2018, View Source [SID1234528823]). Vicinium, Sesen Bio’s lead product candidate, is currently being evaluated in a Phase 3 registration trial, the VISTA Trial, for the treatment of patients with high-grade NMIBC who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive.

"The granting of this designation is an important milestone for Sesen Bio, and we believe it exemplifies the urgent need for a new treatment option for people with NMIBC for whom bladder removal is the recommended course after BCG," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "We are highly encouraged by the differentiated product profile of Vicinium in NMIBC, with a unique mechanism of action, positive three-month data presented earlier this year and favorable tolerability in patients treated to-date. With Fast Track designation, we look forward to determining the optimal registration path and assessing the opportunity for accelerated approval to bring Vicinium to patients as quickly as possible."

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious or life-threatening conditions and fill an unmet medical need. Fast Track designation allows for frequent communication and interactions with the review team at the FDA throughout the drug development and review process, with the goal of providing faster drug approval and greater patient access.

Enrollment is complete in the Phase 3 VISTA Trial and the company expects to report 12-month efficacy results in mid-2019.

About Vicinium
Vicinium, also known as VB4-845, is Sesen Bio’s lead product candidate and is a next-generation antibody-drug conjugate (ADC), developed using the company’s proprietary Targeted Protein Therapeutics platform, for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC). Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered peptide linker to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently conducting the Phase 3 VISTA Trial, designed to support the registration of Vicinium for the treatment of high-grade NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Twelve-month data from the trial are anticipated in mid-2019. Additionally, Sesen Bio believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

Dova Pharmaceuticals Reports Second Quarter 2018 Operating and Financial Results

On August 9, 2018 Dova Pharmaceuticals, Inc. (NASDAQ: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for rare diseases where there is a high unmet need, reported its operating and financial results for the second quarter ended June 30, 2018 (Press release, Dova Pharmaceuticals, AUG 9, 2018, View Source [SID1234528822]).

"In the second quarter, we achieved our most significant milestone in Dova’s corporate history with the approval and launch of DOPTELET in the United States" said Alex C. Sapir, President and Chief Executive Officer of Dova. "We are pleased with the feedback we are hearing from physicians both in terms of how the drug is performing clinically, as well as the reimbursement support their patients are receiving through DOVA1SOURCE. This has translated to a high level of payer approval, as over 80% of referrals have been approved. In addition, we remain well-positioned financially, with approximately $135M in cash and equivalents to fund the operational success of the company for the foreseeable future."

DOPTLET Launch Highlights

·On May 21st, DOPTELET was approved by the U.S. Food and Drug Administration (FDA) for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure. DOPTELET was launched in the United States on June 4th.

·A total of 148 health care professionals have prescribed DOPTELET to their patients since launch with an increasing number using DOPTELET for multiple patients within their practice.

· For prescriptions that have completed the adjudication process with payers, the Company has seen greater than 80% of those prescriptions approved by the payer with an average approval time of 6.9 days.

·The Company has made significant progress in its outreach efforts to target prescribers having reached 62% of top hepatologists an average of 3.1 times since launch.

· Pivotal Phase 3 data for DOPTELET were published in Gastroenterology (View Source). The data were also highlighted at several key global scientific conferences including Digestive Disease Week (DDW) 2018, the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), and the 64th International Society on Thrombosis and Haemostasis (ISTH).

Other Important Highlights for the Quarter

·On April 27th, the Company submitted a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for DOPTELET for the treatment of thrombocytopenia in adult patients with CLD who are scheduled to undergo a procedure. The EMA has granted a Standard Review Assessment with a targeted decision date of July 2019.

· A supplemental New Drug Application (sNDA) for the treatment of patients with chronic immune thrombocytopenia (ITP) who have had an inadequate response to a previous treatment remains on track for submission to the FDA in the third quarter of 2018.

· The Company has initiated a Phase 3 clinical trial for the treatment of patients with chemotherapy-induced thrombocytopenia (CIT).

· The Company repaid, in full, the secured promissory note that was issued to Eisai on March 31, 2016 of $31.1 million. The Company refinanced a portion of the note by entering into a Loan and Security Agreement with Silicon Valley Bank (SVB) for $20.0 million on April 17, 2018. The loan matures on April 17, 2021 unless a specified revenue milestone is achieved in which case the maturity date will be extended to April 17, 2022.

·Nancy J. Wysenski, an industry veteran with over 30 years of commercial and sales leadership, joined the Company’s Board of Directors. As the former Chief Commercial Officer at Vertex, she was responsible for launching Incivek, a treatment for hepatitis C, which is considered by many to be the most successful drug launch in U.S. history.

Dova will provide an update and additional details on DOPTELET’s launch activities during today’s call as well as at its upcoming Investor and Analyst Day scheduled for September 20, 2018 in New York City, New York. To RSVP for this event, please email John Woolford at [email protected].

Second Quarter and Financial Results

Dova reported a net loss of $20.0 million for the second quarter of 2018, compared to a net loss of $5.5 million for the same period in 2017.

For the second quarter of 2018, Dova reported net product sales from DOPTELET of $2.0 million. The Company recognizes revenue using the sell-in methodology when products are delivered to its specialty pharmacy partners. The majority of net sales recognized in the quarter were related to the initial stocking of DOPTELET at the specialty pharmacies. In addition, in March 2018, Dova entered into an exclusive distribution agreement with Shanghai Fosun Pharmaceutical Industrial

Development Co., Ltd., (Fosun Pharma Industrial). Dova received a $5.0 million upfront payment from Fosun Pharma Industrial of which $2.6 million was recognized as revenue during the second quarter of 2018. There were no product sales or other revenue in the second quarter of 2017.

Cost of product sales for the second quarter were $0.5 million, of which approximately $0.3 million consisted of a one-time stock-based compensation charge.

Research and development expenses were $4.5 million in the second quarter of 2018, compared to $3.3 million for the same period in 2017. The increase was primarily due to the initiation of Phase 3 clinical trials to evaluate DOPTELET for patients with thrombocytopenia undergoing surgery regardless of disease etiology and chemotherapy-induced thrombocytopenia.

Selling, general and administrative expenses were $18.6 million in the second quarter of 2018, compared to $1.9 million for the same period in 2017. The increase was primarily due to building Dova’s commercial infrastructure to support the launch of DOPTELET, increased corporate infrastructure, and additional costs associated with operating as a public company.

As of June 30, 2018, Dova had $134.7 million in cash and equivalents compared to $94.8 million as of December 31, 2017.

Company to Host Conference Call

Dova will host a conference call today, August 9, 2018 at 4:30 p.m. ET to discuss second quarter 2018 financial results and recent operational highlights. A question-and-answer session will follow Dova’s remarks.

To participate on the live call, please dial 866-550-8145 (domestic) or +1-430-775-1344 (international) and provide the conference ID 9093837 five to 10 minutes before the start of the call.

A live audio webcast of the call will also be available via the "Investor Relations" page of the Dova website, www.dova.com. Please log on through Dova’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Dova’s website for 90 days following the call.

Indication and Important Safety Information

INDICATION

DOPTELET (avatrombopag) is indicated for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease. Portal vein thrombosis has been reported in patients with chronic liver disease treated with TPO receptor agonists. In the ADAPT-1 and ADAPT-2 clinical trials, there was 1 treatment-emergent event of portal vein thrombosis in a patient (n=1/430) with chronic liver disease and thrombocytopenia treated with DOPTELET.

Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency).

DOPTELET should not be administered to patients with chronic liver disease in an attempt to normalize platelet counts.

CONTRAINDICATIONS:

None

ADVERSE REACTIONS

Most common adverse reactions (> 3%) were: pyrexia, abdominal pain, nausea, headache, fatigue, and edema peripheral.

Please see full Prescribing Information for DOPTELET (avatrombopag) www.doptelet.com