On August 8, 2018 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported a business update and financial results for the second quarter ended June 30, 2018 (Press release, Leap Therapeutics, AUG 8, 2018, View Source [SID1234528560]).

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"Over the past few months, we have seen patients in the studies of both of our antibody programs experience clinical responses in combination with checkpoint inhibitors, including in patients who would not be expected to respond to checkpoint inhibitors alone. We look forward to continuing to read out data throughout the second half of 2018 from these ongoing clinical trials, including from our study evaluating DKN-01 in combination with KEYTRUDA (pembrolizumab) in patients with advanced esophagogastric cancer at the European Society for Molecular Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting in October," commented Christopher K. Mirabelli, Ph.D, President and Chief Executive Officer of Leap Therapeutics. "In addition, we recently announced a TRX518 collaboration with Pfizer and EMD Serono to add to our existing DKN-01 collaborations with Merck and Roche."

Recent Highlights

Since the end of the first quarter, we have made substantial progress with the development of our product candidates:

· Presented clinical results from our study evaluating DKN-01 in combination with KEYTRUDA (pembrolizumab) in patients with advanced esophagogastric cancer.

· Combination has been well tolerated with no dose limiting toxicity.

· In the high-dose DKN-01 cohort of dose escalation, two of four evaluable patients naïve to anti-PD-1/PD-L1 therapy have had a partial response, with two other evaluable patients having a best response of stable disease.

· Both of the responding patients have a tumor phenotype which is typically less responsive to anti-PD-1 therapy.

· Currently enrolling expansion cohorts in patients who are naïve to anti-PD-1/PD-L1 therapy and patients who are refractory to anti-PD-1/PD-L1 therapy.

· The treatment-naïve cohort has passed the Simon Stage 2 threshold.

· Enrollment of the full study is expected during the first quarter of 2019.

· Additional data from this study will be presented at the ESMO (Free ESMO Whitepaper) 2018 Annual Meeting in October.

· Announced a collaboration agreement with Pfizer and Merck KGaA, Darmstadt, Germany to evaluate TRX518 in combination with BAVENCIO (avelumab) and cyclophosphamide chemotherapy.

· Under the terms of the collaboration, we will be conducting a Phase I/II clinical trial in advanced solid tumors including expansion populations in patients with relapsed/refractory ovarian, breast, and prostate cancers.

· The study is expected to begin enrolling patients in the first quarter of 2019.

· Presented initial data from our clinical trial evaluating TRX518 in combination with gemcitabine chemotherapy or in combination with KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab).

· The first two patients treated with the higher dose of TRX518 in combination with KEYTRUDA have experienced clinical benefit after the end of two cycles of therapy.

· Esophageal squamous cell carcinoma patient demonstrated a partial response with a 36% reduction in tumor volume.

· Ocular melanoma patient experienced stable disease with a 23% reduction in tumor volume.

· Seven patients have been treated with the lower dose of TRX518 in combination with OPDIVO.

· Urothelial carcinoma patient who had progressed while on KEYTRUDA has had a partial response with a 39% reduction in tumor volume after the end of two cycles.

· Four other patients experienced progressive disease, one patient was non-evaluable, and one patient has not yet had any on treatment disease evaluation.

·Enrollment continues in the dose escalation phase for both the KEYTRUDA and OPDIVO combinations and in the dose expansion phase for the gemcitabine combination.

Selected Second Quarter 2018 Financial Results

Net loss was $7.4 million for the second quarter of 2018, compared to $6.9 million for the same period in 2017.

Research and development expenses were $4.2 million for the second quarter 2018, compared to $4.9 million for same period in 2017. The decrease of $0.7 million was primarily due to a decrease of $0.4 million in manufacturing costs related to clinical trial material, a decrease of $0.2 million in clinical trial costs and a decrease of $0.2 million in consulting fees associated with research and development activities. These decreases were partially offset by an increase of $0.1 million in stock based compensation expense.

General and administrative expenses were $2.6 million for the second quarter 2018, compared to $2.1 million for the same period in 2017. The increase of $0.5 million in general and administrative expenses was primarily due to a $0.4 million increase in legal, audit and consulting fees associated with corporate and business development activities and an increase of $0.1 million of stock based compensation expense.

Cash, cash equivalents and marketable securities totaled $30.5 million at June 30, 2018. Research and development incentive receivables totaled $1.8 million. The Company believes that its current cash and cash equivalents and the anticipated receipt of the research and development incentive receivable will be sufficient to fund the Company’s operating expenses into the fourth quarter of 2019.

On August 8, 2018 Verastem, Inc. (Nasdaq: VSTM) (Verastem Oncology or the Company), focused on developing and commercializing medicines to improve the survival and quality of life of cancer patients, reported financial results for the quarter ended June 30, 2018 and provided an overview of certain corporate developments (Press release, Verastem, AUG 8, 2018, View Source [SID1234528554]).

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"During the second quarter of 2018, we’ve been actively preparing for the commercialization of duvelisib, our first-in-class, oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular lymphoma (FL)," said Robert Forrester, President and Chief Executive Officer of Verastem Oncology. "In advance of our target action date of October 5, 2018, we have been building our U.S. sales force and commercial capabilities in preparation for a potential product launch of duvelisib in the U.S. in 2018. On the financial front, we have significantly strengthened our balance sheet, ending June 30, 2018 with $168.7 million in cash and cash equivalents."

Second Quarter 2018 and Recent Highlights:

Corporate and Financial

Duvelisib NDA accepted by FDA with priority review – In April 2018, Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) accepted the duvelisib New Drug Application (NDA) for filing with Priority Review, with a target action date of October 5, 2018. In the accepted NDA, the Company is seeking full approval for duvelisib, its first-in-class investigational oral dual inhibitor of PI3K-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The duvelisib NDA is supported by clinical data from the randomized Phase 3 DUO study evaluating duvelisib as a monotherapy in patients with relapsed or refractory CLL/SLL, as well as the Phase 2 DYNAMO study evaluating patients with iNHL that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy. Both DUO and DYNAMO achieved their primary endpoints.
Hosted Analyst and Investor Day highlighting commercial potential of duvelisib – In early May 2018, Verastem Oncology hosted an Analyst and Investor Day in New York City titled, "Duvelisib: Harnessing the Power of Dual PI3K Inhibition." Key opinion leaders in the hematologic oncology field including Dr. Lori Kunkel, Former Chief Medical Officer, Pharmacyclics, Dr. Jennifer Brown, Dana-Farber Cancer Institute, Dr. Ian Flinn, Sarah Cannon Research Institute, Dr. Steven Horwitz, Memorial Sloan Kettering Cancer Center as well as Dr. Brian Koffman, Founder & Medical Director of the Chronic Lymphocytic Leukemia (CLL) Society, and a CLL patient, joined the Verastem Oncology executive leadership team for an in-depth discussion regarding the unmet need among CLL/SLL and FL patients, where PI3K-delta and PI3K-gamma inhibitors fit into the treatment paradigm, and the growing opportunity for duvelisib in CLL/SLL and FL, and beyond. The Company also provided an overview of its duvelisib commercial strategy and initiatives. The webcast is available within the "Media" section of the Company’s website at www.verastem.com.
Signed exclusive license agreement with Yakult Honsha Co., Ltd. (Yakult) to develop and commercialize duvelisib in Japan – In June 2018, Verastem Oncology announced its entry into an exclusive license and collaboration agreement with Yakult to develop and commercialize duvelisib for the treatment, prevention or diagnosis of all oncology indications in Japan. The transaction, which carries a total deal value of up to $100.0 million, includes a one-time upfront payment of $10.0 million and up to an additional $90.0 million if certain future pre-specified development, regulatory and commercial milestones are successfully achieved by Yakult. In addition, Verastem Oncology is also eligible to receive double-digit royalties based on future net sales of duvelisib in Japan. Pursuant to the agreement, Yakult has the right to develop and commercialize duvelisib in Japan at its own cost and expense. In addition, Yakult may fund certain global development costs on a pro-rata basis. Verastem Oncology retains all rights to duvelisib outside of Japan.
Strengthened the balance sheet through the sale of equity for net proceeds of approximately $105 Million – In May 2018, Verastem Oncology completed an underwritten registered offering of 8,944,444 shares of its common stock at a price to the public of $4.50 per share. The net proceeds to Verastem from the offering were approximately $38.3 million. In June 2018, Verastem Oncology completed a registered offering of 7,166,666 shares of its common stock at a price of $6.00 per share to funds managed by Consonance Capital. The net proceeds to Verastem Oncology from this offering were approximately $42.8 million. The Company also sold 6,314,410 shares of common stock under its at-the-market equity offering program for net proceeds of approximately $23.7 million.
Joined the Russell 3000 Index – In June 2018, the Company joined the broad-market Russell 3000 Index as part of the Russell US Indexes annual reconstitution.

Scientific Presentations at Major Medical Meetings

Duvelisib

The efficacy of duvelisib monotherapy following disease progression on ofatumumab monotherapy in patients with relapsed/refractory CLL or SLL in the DUO crossover extension study – In June 2018, at both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting (ASCO 2018) and the European Hematology Association (EHA) (Free EHA Whitepaper) 2018 Annual Meeting (EHA 2018), Dr. Byrone Kuss, Flinders Medical Centre, and Dr. Peter Hillman, St. James University Hospital, respectively, presented additional data from the open-label, DUO crossover extension study where patients with radiologically confirmed progressive disease (PD) following treatment with ofatumumab in DUO were given the option to receive treatment with duvelisib. Among the 89 evaluable patients (median 3 prior therapies; range 2-8), duvelisib as a monotherapy achieved a 73% overall response rate (ORR) per investigators assessment in the extension study (95% confidence interval CI: 64,82); 5% complete response with incomplete marrow recovery (CRi), and 68% partial response (PR). The median progression-free survival (mPFS) for duvelisib in the DUO crossover extension study was 15 months (95% CI: 10,17). Notably, 83% of patients in the duvelisib arm post-crossover had >50% reductions in the size of their target nodal lesions. The safety profile of duvelisib as a monotherapy was manageable and consistent with what was observed in the Phase 3 DUO study. These data build upon the previously reported positive DUO results and further support duvelisib as an effective oral monotherapy treatment option for patients with relapsed or refractory CLL/SLL.
A Phase IB/II study of duvelisib in combination with Fludarabine (F), Cyclophosphamide (C), and Rituximab (R) (dFCR) for frontline therapy of younger CLL patients – At EHA (Free EHA Whitepaper) 2018, Dr. Matthew Davids, Dana-Farber Cancer Institute, presented data on the 31 patients evaluable for post-dFCR response. The ORR was 94%, with 26% (n=8) of patients achieving a complete response (CR) or CRi, and 68% achieving a PR. The best rate of minimum residual disease (MRD) negativity in the bone marrow (BM) in patients with at least one evaluation was 76% (22 of 29 patients). All patients who achieved CR/CRi at the primary endpoint also had BM-MRD negativity (26%). Among survivors, the median follow-up is 24.5 months (range 6.9-46 months). The two-year progression-free survival and overall survival rates for patients in the study were both 97%. Eight patients have now completed two years of duvelisib maintenance therapy. Based on these results the recommended Phase 2 dose of duvelisib in combination with FCR was 25mg twice daily. The most common all grade non-hematologic adverse events (AEs) were nausea (72%, all Grade 1/2), fatigue (69%, 3% Grade 3), fever (53%, all Grade 1/2), diarrhea (47%, 3% Grade 3), transaminitis (34%, 28% Grade 3/4), anorexia (34%, all Grade 1/2), vomiting (28%, all Grade 1/2), pruritus (16%, 3% Grade 3), arthritis (9%, all Grade 2) and Cytomegalovirus (CMV) reactivation (6%, both Grade 2). The most common all grade hematologic adverse events were thrombocytopenia (65%; 34% Grade 3-4), neutropenia (59%; 50% Grade 3-4), and anemia (38%, 16% Grade 3). Serious AEs included transaminitis (Grade ≥3), febrile neutropenia (n=6, all Grade 3), pneumonia (n=6, including 3 cases of PJP despite planned prophylaxis), and colitis (n=1 Grade 2, n=1 Grade 3). These results suggest that duvelisib in combination with FCR is an effective regimen for the initial therapy of younger, fit CLL patients and results in a high rate of BM-MRD negativity (76%), significantly higher than historical data with FCR.
The effect of duvelisib, a dual inhibitor of PI3K-δ,γ, on components of the tumor microenvironment in previously untreated follicular lymphoma patients – At both ASCO (Free ASCO Whitepaper) 2018 and EHA (Free EHA Whitepaper) 2018, Dr. Carla Casulo, University of Rochester, Wilmot Cancer Center, presented data from blood samples from healthy volunteers and FL patients treated in the CONTEMPO study. Samples collected both pre- and post-duvelisib treatment were analyzed. Ex vivo and in vitro PI3K-delta assays and PI3K-gamma assays, with PI3K-gamma-selective (idelalisib, TGR-1202, IPI-3063) and PI3K-delta-selective (IPI-549) inhibitors were compared. Collectively, the results of this analysis support the thesis that duvelisib disrupts PI3K-delta and PI3K-gamma function in FL patients, inhibiting the tumor microenvironment (TME) cancer-supportive macrophages and T-cells.
Duvelisib inhibition of chemokines in patients with CLL (DUO) and iNHL (DYNAMO) – At both ASCO (Free ASCO Whitepaper) 2018 and EHA (Free EHA Whitepaper) 2018, Dr. David Weaver, Verastem Oncology’s Vice President, Translational Medicine, presented data showing that PI3K-delta inhibition directly targets proliferation and survival of malignant leukemia and lymphoma cells, while PI3K-gamma inhibition modulates the TME through key support cells, including tumor-associated macrophages, nurse-like stroma and T-cells, and via soluble factors stimulating tumor growth, survival and migration. Serum samples from patients in the Phase 3 DUO study in relapsed/refractory CLL/SLL and the Phase 2 DYNAMO study in relapsed/refractory indolent non-Hodgkin lymphoma (iNHL) were collected at baseline and at infusion cycle date C2D1 and used for correlative studies of 24 chemokines, cytokines and serum factors. These data support the hypothesis that treatment with duvelisib results in significant reduction of chemokines potentially derived from the tumor cells and TME and that further investigation of the effects of duvelisib on TME pharmacodynamic markers is warranted.
Presented scientific data supporting immuno-oncology applications of duvelisib at the 3rd annual Advances in Immuno-Oncology Congress – In May 2018, Jonathan Pachter, Ph.D., Verastem Oncology’s Chief Scientific Officer, gave an oral presentation highlighting the unique potential of duvelisib, as a dual inhibitor of PI3K-delta and PI3K-gamma, to enhance the efficacy of immune checkpoint and co-stimulatory antibodies in preclinical models of both hematological malignancies and solid tumors. Dr. Pachter also moderated a round table discussion regarding novel checkpoint pathways and emerging strategies for combined modality treatment.
Defactinib

Presented preliminary Phase 1 results from combination trial with defactinib, pembrolizumab and gemcitabine in advanced cancer – At ASCO (Free ASCO Whitepaper) 2018, Dr. Andrea Wang-Gillam presented a poster describing results from the ongoing Phase 1 study evaluating defactinib in combination with pembrolizumab and gemcitabine in patients with advanced cancer, including pancreatic cancer. The combination treatment appears to be well tolerated, the recommended Phase 2 dose was established, and the expansion phase of the study is now ongoing. Encouraging signs of clinical activity were observed in three pancreatic ductal adenocarcinoma (PDAC) patients treated beyond 250 days, including one patient with confirmed PR and two patients with stable disease. Meaningful reductions (57-96%) in the pancreatic cancer marker CA19-9 were also observed in all three patients. In addition, analysis of paired biopsies showed that the combination treatment induced desirable biomarker changes including increased proliferating CD8+ T-cells and reduced immunosuppressive Tregs and macrophages.
Presented scientific data supporting immuno-oncology applications of defactinib at the 3rd Annual Advances in Immuno-Oncology Congress – During Dr. Pachter’s oral presentation, he also provided an update on the scientific rationale and clinical progress of Verastem Oncology’s lead focal adhesion kinase (FAK) inhibitor, defactinib, in combination with PD-1 and PD-L1 inhibitors in solid tumors.
All posters and presentations are available within the "Media" section of the Company’s website at www.verastem.com.

Second Quarter 2018 Financial Results

Net loss for the three months ended June 30, 2018 (2018 Quarter) was $18.4 million, or $0.30 per share, as compared to a net loss of $13.4 million, or $0.36 per share, for the three months ended June 30, 2017 (2017 Quarter). Net loss for the 2018 Quarter includes license revenue of $10.0 million, related to the upfront payment received in connection with the license and collaboration agreement with Yakult in June 2018. Cash used in operating activities, excluding the upfront payment from Yakult, was $20.3 million for the 2018 Quarter.

Research and development expense for the 2018 Quarter was $12.4 million compared to $9.0 million for the 2017 Quarter. The $3.4 million increase from the 2017 Quarter to the 2018 Quarter was primarily related to an increase of $1.6 million in contract research organization expense for outsourced biology, development and clinical services, which includes the Company’s clinical trial costs, an increase of $1.0 million in personnel related costs, and an increase of $0.4 million in stock-based compensation expense.

General and administrative expense for the 2018 Quarter was $15.8 million compared to $4.4 million for the 2017 Quarter. The increase of $11.4 million from the 2017 Quarter to the 2018 Quarter primarily resulted from increases in consulting and professional fees of $5.2 million, including $3.6 million related to commercial launch preparation activities, and an increase in personnel related costs of $4.4 million.

As of June 30, 2018, Verastem Oncology had cash and cash equivalents of $168.7 million compared to $86.7 million of cash, cash equivalents and investments as of December 31, 2017.

The number of outstanding common shares as of June 30, 2018 was 73,579,699.

Financial Guidance

Based on the Company’s current operating plans, assuming a favorable regulatory decision and estimated revenue, it expects to have sufficient cash and cash equivalents to fund operations into 2020.

About Duvelisib

Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib

Defactinib is an investigational inhibitor of focal adhesion kinase (FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.

On August 8, 2018 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported financial results for the three months and six months ended June 30, 2018 (Press release, Quanterix, AUG 8, 2018, View Source [SID1234528553]).

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"We are continuing to achieve strong momentum, as made evident by our performance in this second quarter, showing steady growth in terms of both product and company revenue," said Chief Executive Officer, President and Chairman, Kevin Hrusovsky. "We are at a pivotal point in our industry, where biomarkers are being recognized as increasingly representing a critical opportunity for accelerating drug approvals and helping to fuel demand for our technology for pharma services and drug development."

Second Quarter 2018 Financial Highlights
Key financial results for the second quarter are shown below:

Q2 revenue of $8.6M versus prior year Q2 of $5.2M, an increase of 66%.
Q2 product revenue was $5.2M versus prior year Q2 of $3.3M, an increase of 56%.
Q2 Service and Other revenue totaled $3.2M versus prior year Q2 of $1.6M, an increase of 97%.
YTD 2018 Financial Highlights
Key financial results for 2018 YTD are shown below:

YTD revenue of $16.2M versus prior year $10.6M, an increase of 53%.
YTD product revenue of $9.9M versus prior year $6.8M, an increase of 47%.
YTD Service and Other revenue of $5.7M versus prior year $3.3.M, an increase of 75%.
Second Quarter 2018 Business Highlights

SR-X platform momentum continues to build, with 22 assays now available
Cadence of publications continued to increase with 40 new publications featuring Simoa technology in Q2 alone, bringing total to >250
Continued commercial acceleration in Pharma Services, including significant build out of CLIA lab capabilities to handle increased sample volume
Major presence at American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2018 Annual Conference, broadening reach into oncology and liquid tumor biopsy market
Kevin Hrusovsky invited to speak at PULSE: The Atlantic Summit on Healthcare
Attendance at 10 industry tradeshows and conferences in North America and Europe spanning multiple therapeutic areas
Created new integrated marketing and inside sales lead generation team to fuel accelerated pipeline development
Developed and began promoting a major webinar scheduled in September on "Disrupting Drug Development with Digital Biomarkers" featuring Kevin Hrusovsky, CEO and Chairman of Quanterix, Dr. Henrik Zetterberg of the University of Gothenburg, and Dr. Andy Nixon of Duke University
Conference Call
In conjunction with this announcement, Quanterix Corporation will host a conference call on August 8, 2018, at 4:30 p.m. EDT to discuss the Company’s financial results and business outlook. To access this call, dial (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 4299369.

A live webcast will be accessible on the Investors section of Quanterix’ website: View Source The webcast will be available on the Company’s website for one year following completion of the call.

On August 8, 2018 OncoPep, Inc. reported the initiation of a Phase 1b clinical trial evaluating the safety and tolerability of PVX-410, an investigational multi-peptide cancer vaccine, for patients with smoldering multiple myeloma (SMM). Smoldering multiple myeloma is an early precursor to a rare blood cancer known as multiple myeloma, which affects plasma cells (Press release, OncoPep, AUG 8, 2018, View Source [SID1234528552]). The open-label investigator-sponsored study, led by Noopur Raje, M.D. at Massachusetts General Hospital, will assess two different combinations of the study drugs; a combination of PVX-410 along with an investigational histone deacetylase (HDAC 6) inhibitor, citarinostat (CC-96241, Celgene) and a triple combination of the PVX-410 vaccine, citarinostat, and lenalidomide.

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"Currently, there are no active treatment options for smoldering multiple myeloma and standard care for patients diagnosed with SMM is ‘watchful waiting’," said Dr. Raje, Director of the Center for Multiple Myeloma at the Massachusetts General Hospital Cancer Center and Professor of Medicine at Harvard Medical School. "This Phase 1b clinical trial will further evaluate the safety and tolerability of PVX-410 in combination with the HDAC 6 inhibitor, citarinostat, with or without the immunomodulatory drug, lenalidomide, in hopes that the combination will supplement a targeted immune-mediated attack against MM cells."

The study is expected to enroll approximately 20 patients at multiple trial sites, including Massachusetts General Hospital. More information on the trial can be found at clinicaltrials.gov, identifier number NCT02886065.

"OncoPep’s Phase 1b clinical trial of PVX-410 in SMM is an important advance for the multiple myeloma cancer community and we are pleased to again be working with Dr. Raje’s team," said Doris Peterkin, Chief Executive Officer of OncoPep. "Given the extremely limited therapeutic options available for patients diagnosed with SMM who are likely to progress to multiple myeloma, we believe PVX-410 has the opportunity to make a significant impact on this patient population."

About Smoldering Multiple Myeloma
Smoldering multiple myeloma (SMM) is a plasma cell proliferative disorder with a high risk of progression to multiple myeloma (MM). It is estimated that SMM accounts for approximately 15% of all newly diagnosed cases of MM, and the annual risk of progression from SMM to symptomatic MM requiring treatment is estimated to be 10%. The current standard of care for SMM is watchful waiting, and approaches that intend to delay or prevent progression to symptomatic MM are needed.

About PVX-410
PVX-410 is a novel investigational therapeutic cancer vaccine currently in Phase 1b clinical trials in smoldering multiple myeloma and triple negative breast cancer. PVX-410 consists of four peptides from unique regions of three tumor -associated antigens which may act to help stimulate an immune response to the targeted tumor cell. PVX-410 was granted orphan drug designation from the U.S. Food and Drug Administration in 2013 for the treatment of multiple myeloma.

On August 8, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended June 30, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 8, 2018, View Source [SID1234528551]).

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"Rocket made significant progress on our clinical, regulatory and corporate initiatives in the second quarter," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We are pleased with the positive clinical data from our FA program that were presented at ASGCT (Free ASGCT Whitepaper) and look forward to additional data over the next 12-18 months. The momentum has continued with recent regulatory designations for FA, including Rare Pediatric Disease from the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) by the European Medicines Agency (EMA). These positive steps set the stage nicely for a global registrational study in 2019."

"Our additional gene therapy pipeline programs for devastating rare diseases remain on track. These include our products for Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and our undisclosed adeno-associated viral vector (AAV) program. We expect to disclose the indication and share preclinical data from our AAV program later this year, and clinical data on up two programs in 2019. Our progress to date has been a true collaboration between the Rocket team, our partners, physicians, and the patients we serve. We look forward to meeting the milestones ahead."

Recent Pipeline and Corporate Updates

Rare Pediatric Disease Designation for FA. In July 2018, the Company was notified that it received Rare Pediatric Disease designation from the FDA for RP-L102 for the treatment of FA Type A. The FDA defines a "rare pediatric disease" as a serious and life-threatening disease that affects less than 200,000 people in the U.S. that are aged between birth to 18 years. The Rare Pediatric Disease designation program allows for a Sponsor who receives an approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
Advanced Therapy Medicinal Product Classification for FA. In June 2018, the Company was notified that the EMA classified RP-L102 as an ATMP. The ATMP classification recognizes and defines medicines for human use that are considered gene-, tissue- or cell-based therapies. The key benefit of ATMP classification is the early involvement and guidance from the EMA’s Committee of Advanced Therapies, which is the regulatory reviewing body for gene therapies.
Phase 1/2 data of RP-L102 in FA shows promising engraftment and chromosomal stability leading to improved bone marrow functionality. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May 2018, updated data from the ongoing Phase 1/2 clinical trial of RP-L102 was presented and included data from four patients that have been followed for 12-24 months and a fifth patient, treated with transduction-enhanced RP-L102, that was followed for two months. All patients demonstrated continued improvement in engraftment following administration of RP-L102 with sustained phenotypic reversals and earlier evidence of gene correction seen in higher-dosed patients. The progressive increases of corrected versus non-corrected peripheral blood leukocytes indicate the potential of RP-L102 to restore the functionality of bone marrow hematopoietic stem cells. The one patient that received transduction enhanced RP-L102 showed the highest transduction efficiency seen to date in all five patients treated, with a preliminary drug product vector copy number (VCN) of ~2.5 – 3, and a cell dose considered below the threshold level of 500,000K CD34+/kg. Rocket plans to engage with regulatory authorities to progress RP-L102 towards a potential global registrational study in 2019.
Stanford University research collaboration. In May 2018, Rocket and the Stanford University School of Medicine announced a strategic collaboration to support the advancement of FA and PKD gene therapy research. Under the terms of the collaboration agreement, Stanford will serve as a lead clinical trial research center in the U.S. for the planned FA registrational trial and would also be the lead site for PKD clinical trials. The project will also separately evaluate the potential for non-myeloablative, non-genotoxic antibody-based conditioning regimens as a future development possibility that may be applied across bone marrow-derived disorders.
Strengthened management team with addition of former FDA Director of the Office of Orphan Products Development (OOPD). Gayatri R. Rao, M.D., J.D., was appointed Vice President, Regulatory Policy and Patient Advocacy, in May 2018. Dr. Rao most recently served as Director of the OOPD within the FDA for the last five years where she was responsible for implementing statutory programs focused on promoting the development of medical products for rare diseases. In her new role at Rocket, Dr. Rao will support the development of global regulatory policies and strategies, patient advocacy initiatives, and rare disease natural history studies.
Anticipated Milestones

Preclinical data and disclosure of the AAV-based gene therapy program (4Q18)
Investigational Medicinal Product Dossier (IMPD) filing in Spain for the LAD-I program (4Q18)
IMPD filing in Spain for the PKD program (Early 2019)
Additional FA patient data (Next 12-18 months)
Investigational New Drug (IND) application filing in the U.S. for the AAV-based program (2019)
IND application filing in the U.S. for the FA program (2019)
September Conferences

Citi’s 13th Annual Biotech Conference – September 5-6, 2018 in Boston, MA
Morgan Stanley 16th Annual Global Healthcare Conference – September 12-14, 2018 in New York, NY
Oppenheimer Specialty Pharma & Rare Disease Fall Summit – September 25-26 in New York, NY
Jefferies Gene Therapy and Editing Summit – September 27, 2018 in New York, NY
Second Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2018, were $171.5 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $10.8 million and $16.5 million for the three and six months ended June 30, 2018, compared to $2.8 million and $5.1 million for the three and six months ended June 30, 2017.
G&A expenses. General and administrative expenses were $4.1 million and $12.8 million for the three and six months ended June 30, 2018, compared to $0.7 million and $1.3 million for the three and six months ended June 30, 2017.
Net loss. Net loss was $15.8 million and $31.1 million or $(0.40) and $(0.82) per share (basic and diluted) for the three and six months ended June 30, 2018, compared to $3.3 million and $6.2 million or $(0.49) and $(0.91) per share (basic and diluted) for the three and six months ended June 30, 2017.
Shares outstanding. Approximately 39.5 million shares of common stock were outstanding as of June 30, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of June 30, 2018, will be sufficient to run its operations into 2020.