On March 14, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a clinical-stage oncology company advancing novel therapeutics for patients with cancer and hematological diseases, reported that its oncology drug candidate PNT2258 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse large B-cell lymphoma (DLBCL) (Press release, ProNAi Therapeutics, MAR 14, 2016, View Source [SID:1234509844]). This is the second orphan drug designation obtained by ProNAi for PNT2258 for the treatment of DLBCL, following a similar grant by the European Commission in August 2015.

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"Achieving this regulatory milestone for PNT2258 is an important advancement in our registration-oriented development plan for this cancer drug in DLBCL, a disease for which there are limited treatment options, particularly in patients who relapse or do not respond to front-line therapies." said Dr. Nick Glover, President and CEO of ProNAi.

Orphan drug designation is typically granted for novel drugs or biologics that are intended to treat rare medical diseases or conditions that affect less than 200,000 people in the United States. The designation qualifies the sponsor for certain incentives including seven years of market exclusivity after a drug’s approval, tax credits for clinical research costs, and certain application fee waivers.

ProNAi has also previously received European Commission Orphan Drug Designation for PNT2258 for the treatment of patients with DLBCL. In addition to a 10-year period of market exclusivity in the EU following marketing authorization, receiving orphan drug designation provides other incentives for companies, including scientific advice and protocol assistance during the product’s development phase.

About PNT2258 and DLBCL
ProNAi is actively enrolling patients in "Wolverine", a Phase 2 trial evaluating PNT2258 for the treatment of relapsed or refractory DLBCL and in "Brighton", a Phase 2 trial evaluating PNT2258 for the treatment of Richter’s transformation.

PNT2258 is designed to target cancers that overexpress BCL2, an important and validated oncogene known to be dysregulated in many types of cancer. BCL2 overexpression is thought to be a key driver of DLBCL, an aggressive form of cancer that is the most prevalent form of Non-Hodgkin lymphoma (NHL), comprising approximately 30% of the annual NHL diagnoses in the United States according to the Leukemia & Lymphoma Society (2013).

DLBCL affects mostly middle aged and older adults and is aggressive but potentially curable. First-line treatment of intensive combination chemotherapy involving rituximab may cure approximately 67% of patients. If this fails, second-line treatment is typically platinum-based chemotherapy along with continued rituximab. In the event that a response is achieved with second-line treatment, patients may be given a hematopoietic stem cell transplant. If second-line treatment or the transplant fails, patients are left with few options and little hope of a curative therapy. The median survival for third-line DLBCL patients is less than a year.

On March 14, 2016 ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders and cancer, reported financial results for the fourth quarter ended December 31, 2015 and provided an update on the Company’s corporate and clinical development activities expected in 2016 (Press release, ChemoCentryx, MAR 14, 2016, View Source [SID:1234509537]).

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"2015 was a period of significant accomplishments for ChemoCentryx, highlighted by the positive clinical results which we announced earlier this year from the Phase II CLEAR trial in ANCA Vasculitis (AAV) with our lead drug candidate, CCX168. Premature death is among the many dangers of chronic high dose steroids in the standard of care for AAV. At ChemoCentryx, we think such risks are unacceptable, and simply cannot be ignored," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "The single biggest cause of first year mortality associated with AAV is steroid-induced infections. Adding to this, there are other dangers of chronic high dose steroid use, such as bone fractures, incipient diabetes, and neuropsychiatric problems. Our data showed that CCX168 can eliminate the need for chronic high dose steroid administration in patients with AAV, and it was correlated with a rapid and sustained remission of disease symptoms, as well as improved quality of life, as reported by patients themselves."

Dr. Schall continued, "The momentum of last year has continued into 2016 as we await several important milestones, including potentially the initiation of our Phase III development program for CCX168. We also await clinical data for CCX872 in pancreatic cancer. With multiple programs advancing in the clinic, we believe that this is one of the most exciting moments in ChemoCentryx’s history."

Pipeline Developments Across Key Therapeutic Areas

Orphan and Rare Diseases: CCX168 is an orally-administered complement inhibitor targeting the C5a receptor (C5aR), and is being developed for several rare disease indications, including ANCA-associated vasculitis (AAV) and atypical Hemolytic Uremic Syndrome (aHUS). CCX168 acts by blocking the destructive action of neutrophils that are activated as a consequence of the complement protein known as C5a binding to C5aR on neutrophils during autoimmune inflammation events.

CCXI reported positive top-line data from the Phase II CLEAR trial with CCX168 in 63 evaluable patients with AAV. The objective of the trial was to eliminate chronic high dose steroids from the standard of care (SOC) regimen in AAV and replace them with CCX168. Chronic high dose steroid administration is associated with premature death and a spectrum of other harmful side effects in AAV therapy.

The trial was successful in meeting its primary endpoint based on the Birmingham Vasculitis Activity Score response at week 12 in patients receiving CCX168, compared to those patients receiving the high dose steroid-containing SOC.

All CCX168 treatment groups demonstrated a numerically superior and statistically significant (P=0.002 for non-inferiority) clinical efficacy outcome when compared to SOC.

Treatment with CCX168 led to improvements in kidney function as measured by improvements in estimated glomerular filtration rate (eGFR) as well as beneficial changes in proteinuria, hematuria and renal inflammation (based on MCP-1).

Patient reported improvements in "Quality of Life" were also significantly improved in CCX168 treatment groups, but not in the SOC group.

Completed enrollment in the CLASSIC Phase II trial in AAV in North America.

Immuno-Oncology: CCX872 is a potent and selective inhibitor of the chemokine receptor known as CCR2, which is being evaluated in patients with non-resectable pancreatic cancer. In an ongoing, multi-center clinical trial with CCX872, 54 patients with non-resectable pancreatic cancer have been enrolled. The primary efficacy measurement of this study is progression-free survival after at least 24 weeks of treatment.

Completed enrollment in the Phase Ib clinical trial of CCX872 in patients with non-resectable pancreatic cancer; and

Presented combination data with check point and chemokine receptor inhibitors at Triple Meeting and Society of Immunotherapy for Cancer meeting showing synergistic effect with combination treatment in triple negative breast cancer models.

Anticipated Milestones

Orphan and Rare Diseases:

Report top-line results from the CLASSIC Phase II trial in patients with AAV in North America with CCX168 in mid-2016;

Conduct End of Phase II meetings with regulatory agencies to review CLEAR and CLASSIC Phase II clinical results in mid-2016;

Initiate Phase III development program with CCX168 for the treatment of AAV by the end of 2016; and

Report early results from the Phase II pilot study of CCX168 in aHUS patients who are on dialysis in 2016.

Immuno-Oncology:

Advance Phase Ib pancreatic cancer trial of CCX872 in combination with FOLFIRINOX; report initial overall response data in the second quarter of 2016 and initial progression free survival data in the second half of 2016.

Chronic Kidney Disease:

Conduct End of Phase II meeting with the FDA to review the Phase II data and discuss the Phase III clinical development program for CCX140 in diabetic nephropathy.

Fourth Quarter 2015 Financial Results

Cash, cash equivalents and investments totaled $76.3 million at December 31, 2015.

Research and development expenses were $8.2 million for the three months ended December 31, 2015 compared to $9.1 million reported for the same period in 2014. The decrease in research and development expense from 2014 to 2015 was primarily attributable to lower expenses associated with CCX168, the Company’s C5aR inhibitor, due to the completion of the Phase II CLEAR trial in the fourth quarter of 2015 and CCX140, the Company’s CCR2 inhibitor, due to the completion of the Phase II clinical trial in patients with diabetic nephropathy in 2014. These decreases were partially offset by higher expenses associated with CCX872, the Company’s second CCR2 inhibitor, reflecting continued patient enrollment in the ongoing pancreatic cancer trial.

General and administrative expenses were $3.4 million for the three months ended December 31, 2015 compared to $3.2 million for the comparable period in 2014. The increase from 2014 to 2015 was primarily due to increases in intellectual property related expenses and professional fees.

Net loss was $11.6 million for the fourth quarter of 2015 compared to $12.2 million in the same period in 2014.

Total shares outstanding at December 31, 2015 were approximately 44.2 million shares.

2016 Financial Outlook

The Company expects to utilize cash and cash equivalents between $40 million and $50 million in 2016.

About ANCA-Associated Vasculitis

Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, or AAV, is a type of rare autoimmune inflammation caused by auto-antibodies. AAV encompasses granulomatosis with polyangiitis (GPA, formerly known as Wegener’s granulomatosis), microscopic polyangiitis (MPA), eosinophilic polyangiitis (formerly Churg-Strauss syndrome) and renal limited vasculitis.

AAV represents a severe and often fatal autoimmune disease that is characterized by inflammation that can destroy different organ systems. AAV is the lead indication in the Company’s orphan and rare disease program with a clinical objective of eliminating chronic high dose steroids in the standard of care (SOC) regimen, essentially replacing steroids with the complement inhibitor CCX168.

AAV affects approximately 40,000 people in the US (with approximately 4,000 new cases each year) and greater than 75,000 people in Europe (with at least 7,500 new cases each year), and is currently treated with courses of immuno-suppressants (cyclophosphamide or rituximab) combined with high dose steroid administration. Following initial treatment, up to 30 percent of patients relapse within 6 to 18 months, and approximately 50 percent of all patients will relapse within 3 to 5 years.

Current standard of care (SOC) for AAV comprises high doses of chronic steroids combined with an immunosuppressant (either cyclophosphamide or rituximab). The SOC is associated with significant safety issues. For example, first year premature death is approximately 11 to 18 percent. The single major cause of premature mortality is not disease related adverse events, but rather infection that is thought largely to be a consequence of steroid administration. Indeed, the multiple adverse effects of courses of steroid treatment (both initial courses and those that are repeated as a consequence of relapse) are major causes of both short-term and long-term disease and death. Such therapy related adverse events contribute significantly to patient care costs, as well as to the diminution of quality of life for patients.

By damaging the body’s small blood vessels, AAV affects many organ systems, mostly the kidneys, eyes, lungs, sinuses and nerves. This damage is caused by the destructive activity of inflammatory leukocytes in the body, with neutrophils considered to be the terminal effector cell. In AAV, neutrophils are attracted to sites of vascular destruction as well as activated at those sites by the activity of the complement system product known as C5a and its receptor, C5aR, which is the target of CCX168. By blocking the C5aR, CCX168 is thought to reduce vasculitis by reducing neutrophil activation, accumulation, and adhesion, as well as vascular permeability.

About Pancreatic Cancer

It is estimated that over 337,000 cases of pancreatic cancer are diagnosed worldwide every year, accounting for 2.4 percent of all cancers. The incidence of pancreatic cancer in the US is about 45,000, with prevalence being only negligibly higher owing to the poor survival rates on current therapy. Current standards of care include surgical resection and chemotherapeutic regimens such as gemcitabine and FOLFIRINOX. These regimens are limited by marked toxicities. Almost 67 percent of cases are diagnosed in people aged 65 and over. In the United States, pancreatic cancer is the fourth most common cause of deaths due to cancer. Pancreatic cancer has a low survival rate regardless of stage of disease, with 93 percent of patients dying from their disease within five years.

On March 14, 2016 Vericel Corporation (NASDAQ: VCEL), a leading developer of patient-specific expanded cellular therapies for the treatment of severe diseases and conditions, reported financial results and business highlights for the fourth quarter and year ended December 31, 2015 (Filing, Q4/Annual, Vericel, 2015, MAR 14, 2016, View Source [SID:1234510447]).

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Recent Business Highlights

During and since the fourth quarter of 2015, the company:
• Announced positive top-line results from the Phase 2b ixCELL-DCM clinical trial of ixmyelocel-T in patients with heart failure due to ischemic dilated cardiomyopathy;

• Received U.S. Food and Drug Administration (FDA) approval of the Epicel (cultured epidermal autografts) HDE supplement, which revised the Epicel product label to include pediatric patients and specify the probable survival benefit for adult and pediatric patients treated with Epicel, and allows the company to sell Epicel for profit on up to 360,400 grafts per year;

• Announced that the FDA has accepted for filing the BLA for MACI (matrix applied characterized autologous cultured chondrocytes), the company’s investigational third-generation autologous cultured chondrocyte implant intended for the treatment of symptomatic full-thickness cartilage defects of the knee in adult patients;

• Announced a long-term supply agreement with Matricel GmbH for the collagen membrane used in the production of MACI;

• Achieved 14% growth in total Carticel (autologous cultured chondrocytes) and Epicel net product revenues for 2015 over pro-forma Carticel and Epicel revenues for 2014, and 5% growth in total Carticel and Epicel net product revenues in the fourth quarter versus the fourth quarter of 2014;

• Achieved 60% and 24% growth in Epicel net product revenues for 2015 and the fourth quarter, respectively, versus the same periods in 2014; and

• Entered into a $10 million credit facility and $5 million term loan agreement with Silicon Valley Bank.

"2015 was an extremely productive year during which we completed our corporate transformation into a sustainable and growing commercial enterprise, substantially increased revenues and gross margins, and made significant progress on our clinical and regulatory objectives that we expect will drive current and long-term growth for the company," said Nick Colangelo, president and CEO of Vericel. "We believe that we have positioned the company as one of the leading cell therapy and regenerative medicine companies in the industry."

Financial Highlights

Total revenues for the fourth quarter and year ended 2015 were generated primarily from net sales of Carticel implants and surgical kits and Epicel, which were acquired on May 30, 2014 as part of the acquisition of Sanofi’s cell therapy and regenerative medicine business.

Total net revenues for the quarter ended December 31, 2015 were approximately $15.4 million and included approximately $11.3 million of net sales of Carticel implants and surgical kits and approximately $4.1 million of net sales of Epicel. Total Carticel and Epicel net product revenues in the fourth quarter increased approximately 5% over the same period in 2014.

Total net revenues for the year ended December 31, 2015 were approximately $51.2 million, including approximately $35.2 million of net sales of Carticel implants and surgical kits and approximately $15.2 million of net sales of Epicel. Total Carticel and Epicel net product revenues for 2015 increased approximately 14% over pro-forma Carticel and Epicel net product revenues for 2014. Total revenues for the quarter and year ended December 31, 2015 included approximately $0.1 million and $0.7 million of sales, respectively, from our Marrow Donation business which ceased operations in December, 2015.

Gross profit for the quarter and year ended December 31, 2015 was $8.2 million, or 53% of net product sales, and $24.7 million, or 48% of net product sales, respectively, compared to $8.0 million, or 54% of net product sales, and $11.5 million, or 40% of net product sales, for the quarter and year ended December 31, 2014, respectively.

Research and development expenses for the quarter and year ended December 31, 2015 were $7.4 million and $18.9 million, respectively, versus $5.8 million and $21.3 million for the same periods in 2014. The increase in research and development expenses in the fourth quarter is primarily due to additional research, development and regulatory costs incurred for the Biologics License Application (BLA) for MACI and Humanitarian Device Exemption (HDE) supplement submitted in December 2015 to revise the labeled indications for use of Epicel, which included $2.2 million in regulatory consulting expenses and a Prescription Drug User Fee Act (PDUFA) filing fee of $2.4 million paid in the fourth quarter of 2015.

The decrease in full-year research and development expenses is primarily due to a reduction in expenses associated with the ixCELL-DCM clinical trial, which completed enrollment in January 2015, and other clinical trial expenses, and a $3.2 million payment in 2014 to the former shareholders of Verigen pursuant to a settlement agreement that eliminated all future milestone payments related to the development and commercialization of MACI in the United States.

Selling, general and administrative expenses for the quarter and year ended December 31, 2015 were $5.7 million and $22.5 million, respectively, compared to $4.5 million and $13.8 million for the same periods in 2014. The increase in SG&A expenses is primarily due to Vericel being a commercial business for all of 2015 compared to only seven months in 2014, as well as an increase in sales and marketing expenses associated with Carticel and Epicel and strategic planning activities for MACI.

Loss from operations for the quarter and year ended December 31, 2015 was $5.0 million and $16.7 million, respectively, compared to $2.3 million and $23.5 million for the same periods a year ago. The operating loss for the quarter ended December 31, 2015 included $2.2 million for MACI BLA and Epicel HDE supplement regulatory consulting expenses and a $2.4 million PDUFA filing fee for MACI. Excluding these one-time expenses, the company would have had an adjusted operating loss of $0.4 million in the fourth quarter. Material non-cash items impacting the operating loss for the quarter and year included $0.6 million and $2.7 million, respectively, of stock-based compensation expense and $0.4 million and $1.6 million, respectively, in depreciation and amortization expense.

Other income (expense) for the quarter and year ended December 31, 2015 was less than $0.1 million and $0.3 million, respectively, compared to less than ($0.1) million and $3.6 million for the same periods in 2014. The change in other income for the quarter is primarily due to a decrease in the fair value of warrants in the fourth quarter of 2015 compared to the same period in 2014. The decrease in other income for the full year 2015 is primarily due to a bargain purchase gain of $3.5 million recognized in 2014 and a decrease in the fair value of warrants in 2015 compared to 2014.

Vericel reported a net loss for the quarter and year ended December 31, 2015 of $4.9 million, or $0.28 per share, and $16.3 million, or $0.97 per share, respectively, compared to a net loss of $2.4 million, or $0.17 per share, and $19.9 million, or $2.23 per share, for the same periods in 2014.

As of December 31, 2015, the company had $14.6 million in cash and cash equivalents compared to $30.3 million in cash and cash equivalents at December 31, 2014.

On March 14, 2016 Immunomedics, Inc., (Nasdaq:IMMU) reported that the Company is terminating the Phase 3 PANCRIT-1 trial with yttrium-90-labeled (90Y) clivatuzumab tetraxetan in patients with metastatic pancreatic cancer who had received at least two prior therapies, one of which must have been a gemcitabine-containing regimen (Filing, 8-K, Immunomedics, MAR 14, 2016, View Source [SID:1234509542]).

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The decision to terminate the trial early is based on the recommendation from the independent Data and Safety Monitoring Board (DSMB), following a planned interim analysis of data on overall survival (OS) after more than 50% of the required 371 deaths had occurred. The interim analysis showed that the treatment arm of 90Y-clivatuzumab tetraxetan combined with low-dose gemcitabine and best supportive care did not demonstrate a sufficient improvement in OS as compared to placebo plus low-dose gemcitabine and best supportive care.

"Given the significant unmet medical need in pancreatic cancer, we are disappointed that clivatuzumab tetraxetan did not produce the desired outcome and will review our future strategy for this antibody with key opinion leaders," remarked Cynthia L. Sullivan, President and Chief Executive Officer. "We would like to thank our clinical investigators and their patients and families for participating in this study. Pancreatic cancer is a challenging disease to treat but we remain fully committed to bringing innovative products, such as IMMU-132 (antibody-drug conjugate, sacituzumab govitecan) and (E1)-3s (T-cell redirecting immuno-oncology bispecific antibody), to address this unmet need," Ms. Sullivan added. "Both these other products have shown activity in pancreatic cancers, either clinically (IMMU-132) or preclinically [(E1)-3s]," Ms. Sullivan commented further.

On March 11, 2016 PharmaMar reported that the creation of a network of centres of reference and adopting measures that promote research and access to treatments are two key aspects for improving the prognosis of patients with soft tissue sarcoma (STS), an uncommon type of cancer that originates in the tissues that connect, support and surround other body structures, such as muscles, fat, blood vessels, nerves, tendons and lining of the joints (Press release, PharmaMar, MAR 11, 2016, View Source [SID:1234510444]).

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This is one of the conclusions of the renowned international oncologists during the presentation of the Soft Tissue Sarcoma: Evidence and Experience seminar. This meeting, organised by PharmaMar, will bring leading world experts in this disease to Barcelona on 11 and 12 March from, among other countries, the United Kingdom, France, Italy, Germany, Spain and the United States, to analyse the latest advances in its diagnosis and treatment.

"We are proud to lead this meeting, where the most influential oncologists in the world in sarcoma management share opinions, present new case reports, discuss the latest advances and the most effective treatments for these patients", explained Mr Luis Mora, General Manager of the PharmaMar Oncology Unit.

Incidence of sarcoma

According to figures quoted by Prof Jean-Yves Blay, Chair of the European Organisation for the Research and Treatment of Cancer (EORTC), STS represents less than 1% of adult cancersi. "There are more than 50 histological subtypes of soft tissue sarcoma. The most common are leiomyosarcoma and liposarcoma. In Europe, the incidence of this type of tumour in adults (not including gastrointestinal stromal tumour) is from 4 to 5 cases per 100,000 inhabitantsii and approximately 2 half of the patients diagnosed with soft tissue sarcoma have developed metastases or are expected to do so", said Prof Blay.

To prevent patients reaching the expert sarcoma team after having been operated on, measures are required to guarantee an appropriate diagnosis and treatment agreed upon by a multidisciplinary team. Dr Javier Martín Broto, Chair of the Spanish Sarcoma Research Group (GEIS), maintains that this coordination requires the creation of a network of centres of reference to guarantee that patients with suspected soft tissue sarcoma are diagnosed and treated by teams of experts in the disease. "Studies show that patients who are diagnosed and treated in a centre of reference specialising in soft tissue sarcoma survive for longer than those diagnosed and treated in a centre without such specialisation", he explained.

This is precisely one of the lines of research of the Spanish Sarcoma Research Group which, according to Dr Martin Broto, has been collaborating for years with the Ministry of Health for the recognition of sarcoma hospitals and CSUR (Centres, Services and Units of Reference) teams. In his opinion, "we are currently in the final phase and we expect these centres to be made official in the near future".

The Spanish Sarcoma Research Group was created in 1994 and has led 47 research projects that have included thousands of patients. In the last few years, it has sponsored a large number of international clinical trials and its protocols are used worldwide.

Advances in the treatment of soft tissue sarcoma

Professor George Demetri, Director of the Centre for Sarcoma and Bone Oncology at the Dana-Farber Cancer Institute (Boston), presented the latest research projects related to the treatment of sarcoma, including the study conducted with trabectedin (Yondelis) in the United States, in which the compound was shown to reduce the risk of disease progression versus conventional treatment. Yondelis is developed and marketed by PharmaMar in Europe, while Janssen Products, L.P. has the rights to develop and sell Yondelis in the rest of the world except Japan, where PharmaMar has signed a licensing agreement with Taiho Pharmaceutical. 3

"Since Yondelis was first approved in Europe in 2007, approximately 50,000 patients in 80 countries have benefited from this therapy in all its indications", said Prof. Demetri, who explained that "advanced soft tissue sarcoma is a complex set of uncommon diseases that are virtually always life-threatening for patients with advanced stage disease. Patients need new treatment options that are more effective and well tolerated, and renewed hope has come with new drug approvals in the past decade based on sophisticated scientific research".

According to the data provided by this expert, the Phase 3 multicentre trial in the United States, Brazil and Australia is one of the largest conducted to date in sarcoma patients. This study showed a significant improvement in disease control (called "progression-free survival") with trabectedin versus dacarbazine in patients with advanced liposarcoma (LPS) or leiomyosarcoma (LMS) after failure of prior standard therapy with an anthracycline and at least one other chemotherapy.

All the experts present highlighted the importance of these advances in the treatment of sarcoma, a rapidly progressing form of cancer that requires new treatment options. "In soft tissue sarcoma, disease stabilisation is useful for evaluating the success of the treatment in advanced stage patients. The data from the latest clinical trials with Yondelis offer a more hopeful future for patients and a path forward for even more research to improve clinical outcomes", concluded Dr Demetri.