On May 9, 2016 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer, reported financial results for the first quarter ended March 31, 2016, and also provided an overview of certain corporate developments (Press release, Verastem, MAY 9, 2016, View Source;p=irol-newsArticle&ID=2166539 [SID:1234512149]).

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"To date in 2016, Verastem has announced two new clinical collaborations with world-class organizations, including Merck KGaA and Pfizer, and Washington University in St. Louis and Merck & Co., to further elucidate the potential of FAK inhibition to enhance the efficacy of PD-(L)1 inhibitors in patients with pancreatic and ovarian cancer," said Robert Forrester, President and Chief Executive Officer of Verastem. "The data generated from these trials will continue to inform the ongoing development of our anti-cancer therapeutics which reduce cancer stem cells and modulate the local tumor microenvironment to allow both cancer treatments and the immune system to do their job more efficiently. We’ve had a strong start to 2016 with the announcement of these clinical collaborations in addition to attracting key strategic hires on the development team, including Dr. Greg Berk as Chief Medical Officer and Dr. Toyin Shonukan as Vice President of Clinical Development, to oversee and execute on our ongoing and future studies. We are well financed with approximately $100 million in available capital and we look forward to keeping you updated in the coming quarters on our progress."

First Quarter 2016 and Recent Highlights:
Focal Adhesion Kinase Inhibition Program
Clinical Collaboration with Pfizer and Merck KGaA to Evaluate Combination of VS-6063 and Avelumab in Ovarian Cancer – In March 2016, the companies announced a clinical trial collaboration agreement to evaluate the combination of Verastem’s focal adhesion kinase (FAK) inhibitor VS-6063 and Pfizer and Merck KGaA’s anti-PD-L1 immunotherapy avelumab. Verastem has previously reported initial signs of clinical activity in patients with ovarian cancer when VS-6063 is used in combination with paclitaxel. Under the terms of the agreement, the parties will conduct a planned Phase 1/1b clinical trial evaluating escalating doses of the combination of VS-6063 and avelumab as a potential treatment option for patients with advanced ovarian cancer.

Washington University in St. Louis Initiated a Clinical Study of VS-6063 in Combination with Merck & Co.’s Pembrolizumab and Gemcitabine in Pancreatic Cancer – In January 2016, Verastem announced the initiation of a Phase 1 dose-escalation study at Washington University to evaluate its FAK inhibitor VS-6063 in combination with Merck & Co.’s anti-PD-1 immunotherapy pembrolizumab and gemcitabine in patients with pancreatic cancer. The trial builds upon preclinical research conducted by Dr. David Denardo, presented at several conferences in late 2015 and early 2016, demonstrating the ability of FAK inhibition to increase the efficacy of checkpoint inhibition in the reduction of tumor volume and overall survival in models of pancreatic cancer. This Phase 1 clinical trial is currently enrolling and is anticipated to enroll approximately 50 patients with advanced pancreatic cancer.

Presented Scientific Data Supporting FAK Inhibition in Combination with Immunotherapy at Key Medical Meetings – During the first quarter of 2016, Verastem presented data in support of its new development programs focused on advancing its FAK inhibitors in combination with immune-oncology agents and other current and emerging standard of care cancer treatments. Data were presented at several medical and scientific meetings, including the 2016 American Academy of Cancer Research (AACR) (Free AACR Whitepaper), the Society for Gynecologic Oncology’s 2016 Annual Meeting on Women’s Cancer, the Keystone Symposium on Cancer Pathology, the Keystone Symposium on Stem Cells and Cancer, and Immunotherapy World 2016.

Presented Clinical Data from the Window of Opportunity Study at iMig 2016 – In May 2016, the Company announced results from the ongoing open-label, single-center, neoadjuvant Window of Opportunity study evaluating tolerability, along with biomarker and tumor volume response to VS-6063 (400mg BID) following either 12 days (Cohort 1) or 35 days (Cohort 2) of treatment in surgically-eligible patients with malignant pleural mesothelioma. Data analysis from Cohort 1 and Cohort 2 showed that VS-6063 was generally well tolerated with early signs of tumor reduction observed, with six of the twenty patients demonstrating an encouraging tumor reduction after brief treatment with VS-6063.

Development of VS-4718 Continues in Solid Tumors – Dosing continues in a Phase 1 dose escalation trial evaluating single-agent VS-4718 and a Phase 1 clinical trial evaluating VS-4718 in combination with gemcitabine and Abraxane is currently ongoing. Following results from the dose escalation trial, an expansion cohort of VS-4718 + Gemcitabine/Abraxane vs Gemcitabine/Abraxane alone in patients with pancreatic cancer is planned.

Dual PI3K/mTORC1/2 Inhibition Program
Confirmatory Recommended Phase 2 Dose and Expansion Cohorts – The maximum tolerated dose of single-agent VS-5584 has been reached in a Phase 1 study, and the recommended Phase 2 dose (RP2D) is being confirmed. Reductions in pharmacodynamic markers of PI3K and mTOR activity and clinical activity has been observed in some tumor types.

Corporate
Gregory I. Berk, MD Named Chief Medical Officer – In April 2016, the Company announced the appointment of Gregory I. Berk, MD as Chief Medical Officer. Dr. Berk, a highly accomplished physician and a well-regarded oncology veteran with more than 25 years of both industry and academic experience, will be responsible for leading the Company’s global clinical development strategy and clinical operations.

Announced Key Executive Management Appointments and Changes – In April 2016, the Company strengthened its management team through the appointment and promotion of several key individuals. Jonathan Pachter, PhD was promoted to Chief Scientific Officer, David Weaver, PhD was appointed Vice President, Translational Medicine, Joe Chiapponi, Vice President, Finance, was named Treasurer, Principal Accounting and Financial Officer and Oluwatoyin (Toyin) Shonukan, MD, has been appointed Vice President, Clinical Development. Dr. Shonukan most recently served as Senior Medical Director, Oncology Clinical Development at Vertex Pharmaceuticals and has held previous senior appointments at Millennium: The Takeda Oncology Company, Novartis Oncology and Eli Lilly.

First Quarter 2016 Financial Results
Net loss for the first quarter ended March 31, 2016 (2016 Quarter) was $8.3 million, or $0.22 per share, as compared to a net loss of $15.2 million, or $0.46 per share, for the first quarter ended March 31, 2015 (2015 Quarter). Net loss for the 2016 Quarter and 2015 Quarter, excluding non-cash stock-based compensation expense of $1.7 million and $2.9 million, was $6.6 million and $12.3 million, respectively.

Research and development expense for the 2016 Quarter was $4.2 million compared to $10.5 million for the 2015 Quarter. The $6.3 million decrease from the 2015 Quarter to the 2016 Quarter was primarily related to a decrease of $4.2 million in contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, a decrease in personnel related costs of $1.4 million, a decrease of approximately $550,000 in stock-based compensation, and a decrease of approximately $441,000 in travel, facilities and other research and development costs. These decreases were partially offset by an increase of approximately $276,000 in consulting fees.

General and administrative expense for the 2016 Quarter was $4.3 million compared to $4.7 million for the 2015 Quarter. The decrease of approximately $400,000 from the 2015 Quarter to the 2016 Quarter primarily resulted from approximate decreases in stock-based compensation expense of $734,000 and $148,000 in personnel related costs. These decreases were offset by an increase of approximately $411,000 in consulting and professional fees.

As of March 31, 2016, Verastem had cash, cash equivalents and investments of $99.5 million compared to $110.3 million as of December 31, 2015. Verastem used $10.8 million for operating activities during the 2016 Quarter settling one-time compensation payments, severance payments and paying down accounts payable and accruals.
The number of outstanding common shares as of March 31, 2016, was 36,992,418.

Financial Guidance
Based on current operating plans, we expect to have sufficient cash, cash equivalents and short-term investments to fund our research and development programs and operations into 2018.

About Focal Adhesion Kinase
Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for their ability to improve patient survival.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors

On May 09, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of molecular diagnostic testing products and life science tools for translational research, reported that Aetna has added the Prosigna Breast Cancer Gene Signature Assay to its Tumor Markers Clinical Policy Bulletin (Press release, NanoString Technologies, MAY 9, 2016, View Source [SID:1234512121]). Aetna and its more than 19 million members join other payors now covering Prosigna, collectively representing approximately 125 million covered lives throughout the United States.

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This positive coverage decision is in line with updated ASCO (Free ASCO Whitepaper) guidelines released in February, wherein Prosigna is considered medically necessary to assess the necessity of adjuvant chemotherapy in ER-positive, HER2-negative, node-negative breast cancer patients, when adjuvant chemotherapy is not precluded due to any other factor.

"Aetna is one of the nation’s largest health plans, and its decision to cover Prosigna is a clear sign of the expanded interest we are garnering from public and private payors alike," said Brad Gray, president and chief executive officer of NanoString Technologies. "Our team at NanoString will continue to work with national and regional payors to ensure their patients have access to this vital test. We estimate that approximately 80% of patients indicated for Prosigna testing are now covered."

The updated healthcare policy for Aetna, which now includes the Prosigna Assay, is available on its website: View Source

About the Prosigna Breast Cancer Prognostic Gene Signature Assay and nCounter Dx Analysis System
The Prosigna Assay provides a risk category and numerical score for assessment of the risk of distant recurrence of disease at 10 years in postmenopausal women with node-negative (Stage I or II) or node-positive (Stage II), hormone receptor-positive (HR+) breast cancer. Based on the PAM50 gene signature initially discovered by Charles Perou, Ph.D. and colleagues, the Prosigna Assay is an in vitro diagnostic tool that utilizes gene expression data weighted together with clinical variables to generate a risk category and numerical score to assess a patient’s risk of distant recurrence of disease. The Prosigna Assay measures gene expression levels of RNA extracted from formalin-fixed paraffin embedded (FFPE) breast tumor tissue previously diagnosed as invasive breast carcinoma.

The Prosigna Assay requires minimal hands-on time and runs on NanoString’s proprietary nCounter Dx Analysis System, which offers a reproducible and cost-effective way to profile many genes simultaneously with high sensitivity and precision.

The nCounter Dx Analysis System is a highly automated and easy-to-use platform that utilizes a novel digital barcoding chemistry to deliver high precision multiplexed assays. The system is available in the multi-mode FLEX configuration, which is designed to meet the needs of high-complexity clinical laboratories seeking a single platform with the flexibility to run the Prosigna Breast Cancer Assay and, when operated in the "Life Sciences" mode, process translational research experiments and multiplexed assays developed by the laboratory.

In the United States, the Prosigna Assay is available for diagnostic use when ordered by a physician. The Prosigna Assay has been CE-marked and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as Canada, Israel, Australia, New Zealand and Hong Kong.

In the U.S., the Prosigna Assay is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:

(1) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors or (2) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-positive (1-3 nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with four or more positive nodes.

On May 9, 2016 Dicerna Pharmaceuticals, Inc. (Nasdaq:DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, reported financial and operational results for the first quarter ended March 31, 2016 (Press release, Dicerna, MAY 9, 2016, View Source [SID:1234512150]).

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"We continue to achieve milestones across our portfolio of opportunities, including program launches with our DsiRNA-EX Conjugate technology, as well as our DCR-PH1 and DCR-MYC clinical programs," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "We look forward to sharing new preclinical data for our conjugate programs as well as further updates on our clinical programs at our upcoming Investor Day."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: Dicerna’s Dicer substrate short interfering RNA extended (DsiRNA-EX) Conjugates are proprietary RNAi therapeutic candidates rationally designed to enable convenient subcutaneous delivery for Dicerna’s emerging pipeline of liver-targeted RNAi investigational therapies. These conjugates do not involve lipid nanoparticles and are built on the DsiRNA-EX platform, using an extension to one end of the double-stranded DsiRNA molecule. These extensions are unique to Dicerna and utilize proprietary RNA structures that enable a differentiated and independent approach to subcutaneous delivery of RNAi-inducing therapeutics.

Dicerna is enhancing and extending the potency and reach of its DsiRNA-EX Conjugate technology, and has now achieved an IC50 level as low as 0.1 mg/kg in mice, generating greater than 90% gene knockdown at a dose of 1 mg/kg. Dicerna believes that potency in this range will translate into simple, infrequent single-shot dosing regimens in patients.
Dicerna will present extensive preclinical data for its DsiRNA-EX Conjugate programs, including non-human primate data against multiple gene targets, at the Company’s first Investor Day on June 29, 2016 in New York City.
Dicerna is on track to launch three DsiRNA-EX Conjugate programs in 2016, including an ongoing subcutaneous program for the treatment of primary hyperoxaluria that is advancing into IND-enabling studies.
Dicerna’s emerging library of DsiRNA-EX Conjugate molecules encompasses subcutaneously administered inhibitors for more than a dozen liver disease gene targets at various levels of optimization. These gene targets are in the fields of rare diseases as well as chronic liver disease, cardiovascular disease, and viral infectious disease. We expect these emerging programs will enable Dicerna to build a deep and broad pipeline of liver-targeted therapies, and create extensive opportunity for partnership and collaboration leading to additional program launches.
Rare Disease Program Update

DCR-PH1: DCR-PH1 is an intravenously infused DsiRNA-EX-based therapeutic candidate for primary hyperoxaluria type 1 (PH1), a severe, rare genetic disease of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, DCR-PH1 knocked down the activity of the HAO1 gene transcript that encodes for the enzyme glycolate oxidase, thereby significantly reducing the production of oxalate, the key mediator of disease pathology in PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit. In non-human primate studies, a single dose of DCR-PH1 led to an average of 84% knockdown of the HAO1 gene transcript. The DCR-PH1 clinical program consists of the following studies:

PHYOS: During the fourth quarter of 2015, Dicerna initiated PHYOS (Primary Hyperoxaluria Observational Study), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is designed to measure biomarkers implicated in PH1 and to identify patients who may be eligible for the Phase 1 trial of DCR-PH1.
Healthy Volunteer Study: Dicerna continues to enroll in DCR-PH1-102, a Phase 1 dose escalation trial of DCR-PH1 in healthy volunteers. The primary objective of this study is to determine the safety profile of DCR-PH1 in healthy volunteers in order to support dosing of PH1 patients in the United States. This trial was initiated in the fourth quarter of 2015.
PH1 Patient Study: During the second quarter of 2016, Dicerna initiated DCR-PH1-101, a Phase 1 dose escalation trial in PH1 patients, in Germany. Dicerna anticipates initiating additional sites in Europe and the United States during the second-half of 2016. The primary objective of this study is to determine the safety and tolerability of single ascending doses of DCR-PH1, and secondary objectives include measurement of the drug’s PK properties, and corresponding PD measurements of oxalate (associated with disease progression) and glycolate (a marker of DCR-PH1 activity). Dicerna expects to complete this trial in the first half of 2017.
Oncology Program Update

DCR-MYC: DCR-MYC is a potent and specific inhibitor of MYC, an oncogene frequently amplified or overexpressed in a wide variety of tumor types, including hepatocellular carcinoma (HCC). MYC has long been considered "undruggable" with small molecule and antibody technologies. DCR-MYC is a Dicer substrate short interfering RNA (DsiRNA)-based therapeutic formulated in Dicerna’s EnCore lipid nanoparticle for delivery to solid tumors. In preclinical studies, DCR-MYC knocked down MYC transcript levels and significantly reduced tumor volume in multiple mouse tumor models, including models of HCC. DCR-MYC is currently being tested in two ongoing clinical trials.

Phase 1 DCR-MYC Trial in Solid Tumors

Having established safety at 1.0 mg/kg, Dicerna has initiated two expansion cohorts of this Phase 1 clinical trial. The first expansion cohort is enrolling patients with low-to-intermediate grade pancreatic neuroendocrine tumors (PNETs) having failed one or two lines of prior therapy. The second expansion cohort will enroll patients who will undergo pre- and post-treatment biopsies in order to directly assess molecular markers of RNAi activity against the MYC transcript. Direct observation of RNAi activity of DCR-MYC, combined with observations both of anti-tumor activity and inhibition of FDG uptake in tumors, will establish proof-of-concept for the RNAi-based mechanism of action of DCR-MYC. The Company expects to have these proof-of-concept data in 2016.
Phase 1 DCR-MYC Trial in Hepatocellular Carcinoma (HCC)

In December 2014 Dicerna initiated a Phase 1b/2 clinical trial of DCR-MYC in patients with advanced HCC. As of April 26, 2016, 19 patients have been treated with DCR-MYC in six cohorts. The current dosing level is 0.85 mg/kg. While an objective response based on modified Response Evaluation Criteria in Solid Tumors (mRECIST) has not been observed, a reduction in circulating alpha-fetoprotein level, a marker associated with anti-tumor activity, has been observed. The trial continues with dose escalation. Dicerna expects to have preliminary data from the HCC trial by the end of 2016.
Corporate Update

Dicerna announces today that it plans to host an Investor Day on June 29, 2016 in New York City to present new preclinical data for its emerging pipeline of DsiRNA-EX Conjugate programs, as well as an update on its PH1 and oncology clinical programs.

Financial Results

Cash Position – As of March 31, 2016, the Company had $80.6 million in cash and cash equivalents and held-to-maturity investments as compared to $94.6 million in cash and cash equivalents and held-to-maturity investments as of December 31, 2015. In addition, the Company had $1.1 million of restricted cash, which reflects collateral securing its lease obligations.
R&D Expenses – Research and development (R&D) expenses for the first quarter were $11.3 million, compared to $8.7 million for the same period in 2015. The increase in R&D expenses was due primarily to increased expenses related to discovery and early development of future programs including development of the DsiRNA-EX Conjugate delivery platform, increases in clinical activities from initiating additional studies in PH1, increased employee-related expenses primarily due to additional hiring during the period, along with stock-based compensation, and increased occupancy costs.
G&A Expenses – General and administrative (G&A) expenses for the first quarter were $4.5 million, compared to $5.4 million for the same period in 2015. The decrease in these costs was primarily due to stock-based compensation and payroll related cost savings, and a decrease in costs related to the facility move incurred in the first quarter of 2015.
Net Loss – Net loss for the first quarter was $15.7 million compared to a net loss of $14.1 million for the same period in 2015.
For more detailed information and analysis see the Company’s Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on May 9, 2016.

Guidance

Based on Dicerna’s current cash position and operating plan, the Company reiterates its expectation that it has sufficient cash to fund operations for at least the next 12 months. This estimate assumes no additional funding from new partnership agreements or debt or equity financing events.

On May 9, 2016 Cellular Biomedicine Group, Inc. (Nasdaq:CBMG) ("CBMG" or the "Company"), a clinical-stage biomedicine firm engaged in the development of effective stem cell therapies for degenerative diseases and immunotherapies for cancer, reported financial results for the first quarter ended March 31, 2016 and provided business highlights (Press release, Cellular Biomedicine Group, MAY 9, 2016, View Source [SID:1234512205]).

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"In the first quarter of 2016, we made several key clinical and business advancements that we expect will promote long-term growth and strengthen our presence in the cancer and osteoarthritis markets," commented Tony (Bizuo) Liu, CBMG’s Chief Executive Officer. "We reported encouraging Phase IIb data from our most advanced stem cell program for autologous adipose-derived mesenchymal progenitor cell (haMPC) ReJoin therapy for Knee Osteoarthritis (KOA), and launched a Phase I clinical trial for an off-the-shelf allogeneic haMPC AlloJoinTM therapy for KOA. The closing of a $43.13 million strategic investment through April 2016 brings us closer to reaching our objectives of launching multi-site clinical trials in immuno-oncology cell therapies in China, to file INDs and launch clinical trials for AlloJoinTM therapy for KOA patients in the US and to further strengthen our pipeline. We are determined to build on the momentum from the first quarter and believe that we can accomplish meaningful progress on our 2016 operating objectives."

First Quarter 2016 Financial Performance

Cash Position: Cash and cash equivalents as of March 31, 2016 were $15.7 million compared to $14.9 million as of December 31, 2015. We had an increase in cash generated from financing activities due to a private placement financing in February 2016 for gross proceeds of approximately $5 million.
Net Cash Used in Operating Activities: Net cash used in operating activities for the first quarter of 2016 was $3.58 million, compared to $2.41 million for the same period in 2015.
Revenue: Revenues in the first quarter of 2016 were $488,491 compared to $603,390 for the same period in 2015. The revenue in first quarter 2016 is solely comprised of the hospital’s technology services related to cell therapy treatments.
G&A Expenses: General and administrative expenses for the first quarter of 2016 were $2.8 million compared to $2.7 million for the same period in 2015.
R&D Expenses: Research and development expenses for the first quarter of 2016 were $2.40 million, compared to $1.46 million for the same period a year ago. The increase was primarily attributable to the increase of the Company’s immunotherapy research and development team and expenses related to clinical trials.
Net Loss: Net loss allocable to common stock holders was $4.2 million, compared to $4.3 million for the same period in 2015.
Business & Technology Highlights of 2016 To Date

Announced encouraging 48-week clinical data from the Phase IIb trial of the Company’s ReJoin haMPC therapy for Knee Osteoarthritis (KOA), revealing increase of patient’s knee cartilage volume and relief of pain;
Launched an investigator initiated Phase I clinical trial of an off-the-shelf allogeneic adipose-derived mesenchymal progenitor cell (haMPC) AlloJoinTM therapy for KOA patients in China;
Advanced the Company’s cash position following the closing of an agreement with Wuhan Dangdai Science & Technology Industries Group Inc. to invest up to $43.13 million for 2.27 million shares of the Company’s common stock, representing a 16.2% post-money stake investment as of April 15, 2016.

PRM-151: Lead Product Candidate

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Promedior’s lead product, PRM-151, is a recombinant form of human pentraxin-2 protein (rhPTX-2) formulated for intravenous injection (Company Pipeline, Promedior, MAY 9, 2016, View Source [SID:1234512082]). Promedior is initially focusing the clinical development of PRM-151 on rare systemic fibrotic diseases, such as Idiopathic Pulmonary Fibrosis (IPF) and myelofibrosis. Highlights of PRM-151’s clinical development include:

· A Phase 1a clinical study in healthy subjects and IPF patients demonstrated that PRM-151 was safe and well-tolerated
· A Phase 1b randomized, double-blind, placebo-controlled, multiple ascending dose study in IPF patients demonstrated that PRM-151 was generally safe and well‐tolerated and resulted in a mean improvement in Forced Vital Capacity (FVC) at 8 weeks after dosing for only two weeks, whereas patients receiving placebo had a decline in FVC. These data were · presented at the American Thoracic Society Annual Meeting on May 22, 2013.

· A Phase 2 clinical trial to evaluate PRM-151 in patients with myelofibrosis is ongoing. This trial is a multi-center, two-stage, adaptive design study to determine the efficacy and safety of PRM-151 as a single agent or added to a stable dose of ruxolitinib in patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), or Post-Essential Thrombocythemia MF (post-ET MF). Data were presented at the 2014 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the 2014 European Hematology Association (EHA) (Free EHA Whitepaper) meetings in June. Positive preliminary data demonstrated biologic activity with improvements across clinically relevant measures, including bone marrow fibrosis, hemoglobin, platelets, spleen volume, and symptoms. Clinical data showed improvements in four independent treatment groups of myelofibrosis patients who received PRM-151 weekly or monthly, either as a single agent or in patients with no further improvements on a stable dose of ruxolitinib1. Importantly, PRM-151 demonstrated safety and tolerability both alone and in combination with ruxolitinib, with no evidence of the myelosuppression commonly observed with other treatments. This recent data in myelofibrosis demonstrates the potential of Promedior’s immuno-oncology approach in fibrotic cancers.

PRM-151 has demonstrated efficacy in multiple preclinical models of fibrotic disease, including the reduction of established pulmonary fibrosis.