Biomedical Advanced Research and Development Authority
Biomedical Advanced Research and Development Authority
News Article | May 18, 2017
Long-term follow-up data indicate that the tumor resolution was enduring and, moreover, that the mortality rate in the SGX942 1.5 mg/kg treatment group was lower (p=0.08) than the placebo group over the 12 months following completion of CRT. These data further support the safety and tolerability of SGX942 in this patient population. Potential ancillary benefits of utilizing SGX942 for the treatment of oral mucositis include the reduction of infection, the accelerated tumor resolution and the decreased mortality rate. Soligenix recently announced that it has received US Food and Drug Administration (FDA) clearance to advance the pivotal Phase 3 clinical trial and released the protocol study design for SGX942, following the completion of the Phase 2 follow-up visits late last year. The Phase 3 study utilizes the patient population at highest risk of severe oral mucositis as identified in the Phase 2 study (i.e., those receiving the most aggressive CRT). While the drug effect was 67% in the Phase 2 study, a much more conservative estimate was utilized in planning the Phase 3 study, yielding a study size of approximately 190 subjects. SGX942 will be administered in conjunction with the CRT, as a treatment for oral mucositis. "The long-term follow-up data further support the safety and tolerability of SGX942, consistent with the results from the previous Phase 1 study," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "The Phase 2 study also enabled a highly powered and efficient Phase 3 study to be designed, which will use duration of severe oral mucositis as the primary endpoint, while continuing to assess incidence of infection, tumor resolution status and survival as important safety endpoints. We look forward to starting the pivotal Phase 3 study this year." "The primary objective of the Phase 2 study was to demonstrate the safety and explore the efficacy of SGX942," stated Oreola Donini, PhD, Senior Vice President and Chief Scientific Officer of Soligenix. "The results from the follow-up evaluations indicate that SGX942 is safe and well tolerated and may have a number of additional benefits. The consistency between the clinical findings and the earlier nonclinical studies further demonstrates the applicability of dusquetide to a human clinical population in multiple indications, including reduction of infection. Accordingly, we will continue to explore expanding the IDR technology across additional indications, including antibiotic resistant and emerging infectious disease." The Phase 2 oral mucositis clinical study was partially funded with a grant from the National Institute of Dental and Craniofacial Research Small Business Innovation Research grant #1R43 DE024032-01 (Soligenix, Inc.). Dusquetide (the active ingredient in SGX942) is an IDR, a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body's reaction to both injury and infection towards an anti-inflammatory and an anti-infective response. IDRs have no direct antibiotic activity but, by modulating the host's innate immune system responses, increase survival after infections caused by a broad range of bacterial Gram-negative and Gram-positive pathogens. It also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- and/or radiation therapy. Preclinical efficacy and safety has been demonstrated in numerous animal disease models including mucositis, colitis, melioidosis, macrophage activation syndrome (MAS) and other bacterial infections. Some of these preclinical findings have been published in an article entitled "A novel approach for emerging and antibiotic resistant infections: Innate defense regulators as an agnostic therapy," available at the following link: http://dx.doi.org/10.1016/j.jbiotec.2016.03.032. SGX942 has demonstrated safety in a Phase 1 clinical study in 84 healthy human volunteers. Recently, SGX942 had positive results in an exploratory Phase 2 clinical study in 111 patients with oral mucositis due to CRT for HNC. Consistent with preclinical findings, SGX942 at a dose of 1.5 mg/kg demonstrated positive improvements in decreasing the duration of severe oral mucositis by 50% overall compared to the placebo group, from 18 days to 9 days (p=0.099). In patients at the highest risk of developing severe oral mucositis (i.e., those receiving concomitant cisplatin chemotherapy of 80-100 mg/m2 every third week), the reduction in the duration of severe oral mucositis was even more significant at 67% when treated with SGX942 1.5 mg/kg, from 30 days to 10 days (p=0.04). The p-values met the prospectively defined statistical threshold of p<0.1 in the study protocol. Additional observations included an improved tumor response to CRT at the one month follow-up visit, as well as decreases in mortality and infection rate. The study results are reviewed in "Dusquetide: A Novel Innate Defense Regulator Demonstrating a Significant and Consistent Reduction in the Duration of Oral Mucositis in Preclinical Data and a Randomized, Placebo-Controlled Phase 2 Clinical Study," published online in the Journal of Biotechnology and available at the following link: http://dx.doi.org/10.1016/j.jbiotec.2016.10.010. Long-term (12 month) follow-up data further indicated the safety and tolerability of SGX942 treatment, with a sustained trend towards reduced mortality and increased tumor resolution in the 1.5 mg/kg SGX942 treatment group compared to the placebo group. Opioid pain medication use was also seen to decrease over the course of CRT in the 1.5 mg/kg SGX942 treatment group at the point of highest oral mucositis risk, while it increased in the placebo group. Detailed clinical results from the Phase 2 study, as well as a review of the pathogenesis of oral mucositis and the mechanism of action of SGX942, are discussed here. The long-term follow-up results from the Phase 2 study are reviewed in, "Dusquetide: Reduction in Oral Mucositis associated with Enduring Ancillary Benefits in Tumor Resolution and Decreased Mortality in Head and Neck Cancer Patients", published online in Biotechnology Reports and available at the following link: https://doi.org/10.1016/j.btre.2017.05.002. The Phase 2 oral mucositis clinical study was partially funded with a grant from the National Institute of Dental and Craniofacial Research Small Business Innovation Research grant #1R43 DE024032-01 (Soligenix, Inc.). Drug products containing dusquetide have also received Fast Track Designations from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in HNC patients, and as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis. Orphan Drug Designations for use of dusquetide in the treatment of MAS as well as for the treatment of acute radiation syndrome have also been granted. In addition, dusquetide has been granted Promising Innovative Medicine designation in the United Kingdom by the Medicines and Healthcare Products Regulatory Agency for the treatment of severe oral mucositis in HNC patients receiving CRT. Dusquetide and related analogs have a strong intellectual property position, including composition of matter. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, that mucositis affects approximately 500,000 people in the US per year and occurs in 40% of patients receiving chemotherapy. Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes. The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the US, with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation. Oral mucositis in HNC remains an area of unmet medical need where there are currently no approved drug therapies. Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our BioTherapeutics business segment is developing SGX301 as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma, our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203) and acute radiation enteritis (SGX201). Our Vaccines/BioDefense business segment includes active development programs for RiVax®, our ricin toxin vaccine candidate, OrbeShield®, our GI acute radiation syndrome therapeutic candidate and SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease. The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID) and the Biomedical Advanced Research and Development Authority (BARDA). For further information regarding Soligenix, Inc., please visit the Company's website at www.soligenix.com. This press release may contain forward-looking statements that reflect Soligenix, Inc.'s current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations and clinical trial enrollment. Statements that are not historical facts, such as "anticipates," "estimates," "believes," "hopes," "intends," "plans," "expects," "goal," "may," "suggest," "will," "potential," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of the Phase 3 clinical trial of SGX942 (dusquetide) as a treatment for oral mucositis in patients with head and neck cancer receiving chemoradiation therapy and the Phase 3 clinical trial of SGX301 (synthetic hypericin) for the treatment of cutaneous T-cell lymphoma. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission, including, but not limited to, Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/soligenix-announces-publication-of-its-phase-2-long-term-follow-up-results-of-sgx942-for-the-treatment-of-oral-mucositis-in-head-and-neck-cancer-patients-300459736.html
News Article | May 17, 2017
"This study is intended as a step towards improving transfusion therapy and transfusion safety in the U.S.," said Sherrill Slichter, MD, principal investigator from Bloodworks Northwest and lead investigator for the MIPLATE clinical trial. "We're excited to be a part of this study that hopefully will lead to measures that could further protect the nation's blood supply from certain complications and threats of blood transfusions." MIPLATE is a multi-center, controlled, randomized, non-inferiority study designed to evaluate the clinical effectiveness of Mirasol-treated apheresis Platelets in Plasma versus standard apheresis Platelets in Plasma in patients with hypoproliferative thrombocytopenia. This condition is characterized by low platelet count in patients with compromised bone marrow due to hematologic malignancies or treatments such as chemotherapy. "In addition to being designed to support our PMA application for U.S. approval, this study is an important part of our ongoing efforts to advance blood safety and patient care," said Palani Palaniappan, Executive Vice President of Innovation & Development, Terumo BCT. "With a better clinical understanding of technologies like the Mirasol PRT system in the U.S., we can bring our customers a safe, simple and effective solution to help protect their patients from pathogens." About Mirasol PRT System The Mirasol PRT system is CE marked for platelets, plasma, and whole blood and is in use in approximately 20 countries and 140 blood centers throughout Europe, the Middle East, Africa, Asia and Latin America. The system is for investigational use only in the U.S. and Canada and is available in other select countries. About Terumo BCT Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction technologies. We believe in the potential of blood to do even more for patients than it does today. This belief inspires our innovation and strengthens our collaboration with customers. This project has been funded in whole or in part with Federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority, under Contract No. HHSO100201600013C. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/terumo-bct-announces-enrollment-of-the-first-patient-in-its-us-clinical-trial-designed-to-study-platelets-treated-with-the-mirasol-pathogen-reduction-technology-prt-system-300458904.html
News Article | May 23, 2017
Reltecimod (AB103) is a rationally designed peptide that binds to the CD28 co-stimulatory receptor to modulate the host's immune response to severe infections. By limiting, but not inhibiting, the body's acute inflammatory response, Reltecimod helps control the immune system in cases where an out of proportion response could otherwise quickly lead to morbidity and mortality. Reltecimod received Orphan Drug status from the FDA and EMA as well as Fast Track designation. NSTIs, commonly referred to as "flesh eating bacteria", represent the most severe, rare types of infections involving the skin, skin structure and soft tissues. NSTIs progress rapidly and often result in significant tissue destruction and systemic disease leading to multiple organ dysfunction, failure and death. Currently, there are no approved treatments for NSTIs - the standard of care includes prompt and repeated surgical debridement, aggressive resuscitation and physiologic support, in addition to antibiotics. The phase 3 ACCUTE (AB103 Clinical Composite endpoint StUdy in necrotizing soft Tissue infEctions) study is an ongoing randomized, placebo-controlled study, that plans to enroll 290 patients with NSTI at approximately 60 level 1 trauma sites in the U.S. Patients receive Reltecimod or placebo, administered as a single dose during or shortly after surgical debridement, in addition to standard of care treatment. The primary end point is a clinical composite that evaluates both the local and systemic components of this disease. Atox Bio is a late stage clinical biotechnology company with operations in the US and Israel that develops novel immune modulators for critically ill patients with severe infections. Atox Bio is exploring the potential of Reltecimod in NSTI ("Necrotizing Soft Tissue Infection" or "Flesh Eating Bacteria" infections) and additional critical care indications such as Acute Kidney Injury. Atox Bio is supported by an investment syndicate including SR One, OrbiMed and Lundbeckfonden Ventures. Atox Bio has an ongoing contract with the Biomedical Advanced Research and Development Authority (BARDA) supporting the development of Reltecimod in NSTI. Atox Bio was established by Prof. Raymond Kaempfer and Dr. Gila Arad from the Hebrew University of Jerusalem and Yissum. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/atox-bios-reltecimod-passes-futility-analysis-in-phase-3-accute-study-trial-continues-as-planned-300461771.html
News Article | May 18, 2017
VALENCIA, Calif. & PERTH, Australia--(BUSINESS WIRE)--Avita Medical Limited (ASX:AVH; OTCQX:AVMXY), a regenerative medicine company focused on the treatment of wounds and skin defects, today said it achieved both co-primary endpoints in its pivotal clinical trial which will soon be submitted for U.S. market approval of its ReCell® device to treat severe burns. The company also released supportive results from a previous burns trial in a dual data release. These data will be submitted to the U.S. Food and Drug Administration as part of an application to support Premarket Approval (PMA) for the ReCell® Autologous Cell Harvesting device. The Company is now focused on fulfilling requirements for remaining non-clinical data needed for the PMA submission, which is on track for mid-2017. Based on expected timelines, approval could occur by Q2 2018, the Company said. “ The positive results from both clinical trials clearly demonstrate the efficacy of ReCell® for treating burn injuries,” said Dr James Holmes, from Wake Forest Medical Center, North Carolina, who led the pivotal trial. “ Submission of the PMA, and FDA approval, will be the final steps on a long road to improve burn care. The addition of ReCell® to the U.S. burn surgeon's armamentarium is eagerly awaited and will undoubtedly advance burn care in the U.S.” The 30-patient trial was conducted at seven leading U.S. burn centers between 2015 and early 2017. Co-primary endpoints were designed to demonstrate the effectiveness of ReCell® when combined with widely meshed (expanded) skin grafting in the closure of deep-partial and full-thickness burn injuries. Within the trial design, treatment was randomly allocated to two separate parts of each patient’s burn wound such that a controlled comparison of outcomes could be made for conventional skin grafting versus the combination of ReCell® with a more expanded skin graft. Independent analyses of the study data showed that the co-primary endpoints have both been met. The first co-primary effectiveness endpoint gauged superiority of donor skin expansion, to resolve whether using ReCell® could lead to less donor skin being needed. This difference in donor skin expansion with ReCell® was found to be significant (p<0.001) and resulted in use of an average of just over 30% less donor skin than the Control and a commensurate reduction in donor site size. The second co-primary effectiveness endpoint explored the incidence of healing within 8 weeks, which was similar in wounds that received ReCell® compared to those that received control treatment. Healing with ReCell® was found to be statistically non-inferior relative to conventional treatment. Three secondary endpoints evaluated (1) patient preference of scar outcomes along with (2) patient- and (3) blinded-observer overall opinion ratings using a standardized scar assessment scale and no statistical difference was observed between ReCell® and Control on these measures. The Company said it was encouraged by the equivalent secondary endpoint data because more expanded meshed skin grafts are expected to result in a worse long-term scar outcome, but this was not the case with the adjunctive use of ReCell® with these autografts. The results of the co-primary endpoints suggest the use of ReCell®, relative to conventional autografting, can result in use of over 30% less donor skin to achieve comparable short-term healing and long-term scar outcomes. This is important for patients dealing with burn injuries, because the harvesting of donor skin adds discomfort and increases the effective size of their injury. The Biomedical Advanced Research and Development Authority (BARDA) has supported Avita’s late-stage clinical development of ReCell® through a USD 61.9m contract. The successful completion of this trial is a major Avita milestone under the BARDA contract, supporting the Department of Health and Human Services mission toward burn care preparedness in the response to a mass casualty event. The other U.S. trial was conducted between 2010 and 2014, and involved 101 burns patients treated under the same Investigational Device Exemption (IDE) as used for the PMA pivotal trial. Participants in this trial were treated under a different protocol that compared the effectiveness of the cell suspension alone to that of conventional meshed autografting on partial-thickness injuries. These data showed superiority in healing of donor sites used for ReCell®. It also showed superiority of scar outcomes in terms of scar height, in the straight comparison between ReCell® and autografting. The data did not show statistical non-inferiority of burn injury healing using ReCell® compared to standard treatment, although analysis of the group concluded this outcome was due to post-operative care rather than treatment. The Company said this earlier work could support Avita’s PMA submission by providing additional safety and effectiveness information. Avita received support for this trial from the Armed Forces Institute of Regenerative Medicine (AFIRM), a six-way partnership among the U.S. Army, Navy, Air Force, Veterans Administration, the Defense Health Program and the National Institutes of Health. “ Both studies validate our broader view that this unique regenerative approach will transform the way burns are treated in the U.S., a sector that has been starved of innovation for many years,” said Andy Quick, Avita’s Senior VP of Clinical Development. “ The successful trial data represent years of hard work and commitment from patients and physicians across the U.S., all of whom we would like to thank for their participation. We look forward to publication of the complete data set in a peer-reviewed journal.” Avita CEO Adam Kelliher agreed, saying: “ This significant data readout, from a total of 131 burns patients evaluated under randomized controlled settings, takes us one step further along the approval pathway. The Avita team will now push ahead with completing and submitting the PMA, our next big milestone as we work towards launching ReCell® into the U.S. burns market.” Avita’s patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patient’s own skin. Our medical devices work by preparing a Regenerative Epithelial Suspension (RES™), an autologous suspension comprised of the patients’ own skin cells and wound healing factors that are necessary to regenerate natural healthy skin. This is then applied to the area to be treated. In all countries outside of Europe, our portfolio is marketed under the ReCell® brand to promote skin healing in a wide range of applications, including burns, chronic wounds and aesthetics. ReCell® is TGA-registered in Australia, and CFDA-cleared in China. In the United States, ReCell® is an investigational device limited by federal law to investigational and compassionate use. In Europe, our portfolio of medical device products received CE-mark approval as three tailored product presentations, with three individual brand names. ReCell® is designed for the treatment of burns and plastic reconstructive procedures; ReGenerCell™ has been formulated for chronic wounds, including leg and foot ulcers; and ReNovaCell™ is tailored for aesthetic applications, including the restoration of pigmentation. This letter includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “intend,” “could,” “may,” “will,” “believe,” “estimate,” “look forward,” “forecast,” “goal,” “target,” “project,” “continue,” “outlook,” “guidance,” “future,” other words of similar meaning and the use of future dates. Forward-looking statements in this letter include, but are not limited to, statements concerning, among other things, our ongoing clinical trials and product development activities, regulatory approval of our products, the potential for future growth in our business, and our ability to achieve our key strategic, operational and financial goal. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Each forward-looking statement contained in this letter is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the timing of regulatory approvals of our products; physician acceptance, endorsement, and use of our products; failure to achieve the anticipated benefits from approval of our products; the effect of regulatory actions; product liability claims; risks associated with international operations and expansion; and other business effects, including the effects of industry, economic or political conditions outside of the company’s control. Investors should not place considerable reliance on the forward-looking statements contained in this letter. Investors are encouraged to read our publicly available filings for a discussion of these and other risks and uncertainties. The forward-looking statements in this letter speak only as of the date of this release, and we undertake no obligation to update or revise any of these statements.
News Article | May 15, 2017
MISSION VIEJO, CA--(Marketwired - May 15, 2017) - Aeolus Pharmaceuticals, Inc. ( : AOLS), a biotechnology company developing compounds to protect against fibrosis, inflammation, nerve damage and infection announced today financial results for the three months ended March 31, 2017. The company reported net loss of $1,035,000 or $0.01 per share for the three months ended March 31, 2017. This compares to a net loss (before extraordinary items) of $629,000 or $0.01 per share for the three months ended March 31, 2016. The increase in net loss was primarily attributable to pre-IND work on AEOL 11114, the Company's compound in development for the treatment of Parkinson's disease and lower revenues from the Biomedical Advanced Research and Development Authority ("BARDA"). "During the quarter, we initiated a Phase 1 safety study of AEOL 10150 in healthy volunteers, completed manufacturing optimization and oral formulation development work on AEOL 11114 for Parkinson's disease, and reported positive results in an animal model of Cystic Fibrosis with inhaled AEOL 20415," stated John L. McManus, President and Chief Executive Officer. "Although we were disappointed with BARDA's notification in March that it had elected not to exercise additional options under the contract at this time, we are continuing work in process under the contract through its expiration in May 2019. In addition, we are submitting proposals to and have received indications of interest from other government agencies to expand the funding of AEOL 10150 as a medical countermeasure against the pulmonary effects of radiation exposure and sulfur mustard gas exposure." Results of Operations for the Three Months Ended March 31, 2017 Revenue for the three months ended March 31, 2017 was $129,000, which compares to $565,000 for the three months ended March 31, 2016. The revenue is from the BARDA Contract and the decline in revenue is primarily attributable to a lower level of activity under that contract. Research and Development ("R&D") expenses increased $93,000, or 19%, to $594,000 for the three months ended March 31, 2017 from $501,000 for the three months ended March 31, 2016. The increase is primarily attributable to manufacturing optimization and oral formulation development work for AEOL 11114. General and administrative ("G&A") expenses decreased $123,000, or 18%, to $570,000 for the three months ended March 31, 2017 from $693,000 for the three months ended March 31, 2016. The decrease is primarily attributable to lower accounting and legal fees related to SEC filing requirements. Results of Operations for the Six Months Ended March 31, 2017 Revenue for the six months ended March 31, 2017 was $212,000, which compares to $870,000 for the six months ended March 31, 2016. The revenue is from the BARDA Contract and the decline in revenue is primarily attributable to a lower level of activity under that contract. Research and Development ("R&D") expenses increased $89,000, or 9%, to $1,082,000 for the six months ended March 31, 2017 from $993,000 for the six months ended March 31, 2016. The increase is primarily attributable to manufacturing optimization and oral formulation development work for AEOL 11114. General and administrative ("G&A") expenses decreased $2,000 to $1,252,000 for the six months ended March 31, 2017 from $1,254,000 for the six months ended March 31, 2016. The decrease is primarily attributable to lower investor relations fees. As of March 31, 2017, the Company had approximately $981,000 in cash and cash equivalents and 152,085,825 common shares outstanding. The Company had accounts receivable of $603,000 and accounts payable of $638,000 on March 31, 2017. Aeolus has filed today with the SEC its Quarterly Report on Form 10-Q for the quarter ended March 31, 2017. Aeolus urges its investors to read this quarterly filing as well as its Annual Report on Form 10-K, also filed with the SEC, for further details concerning the Company. The Quarterly Report on Form 10-Q and the Annual Report on Form 10-K are also available on the Company's website, at www.aolsrx.com. AEOL 10150 is a superoxide dismutase (SOD) mimic being developed for the treatment/mitigation of lung damage from exposure to chemical and radiological insults and to reduce/prevent lung damage in patients with Idiopathic Pulmonary Fibrosis (IPF) and in cancer patients receiving radiation therapy. AEOL 10150 protects tissue from damage and increases survival in animal models of lung damage after exposure to radiation toxic chemicals, agents that induce inflammation, and trauma by mitigating and/or preventing cell death, inflammation and fibrosis through its action on oxidative stress (Reactive Oxygen Species, or "ROS") and regulation of growth factors and chemokines including PTEN, TGF-β1, HIF-1α, TNF-α and IL-6, as well as impacting subsequent signaling pathways associated with ROS production, apoptosis and fibrosis such as NADPH-oxidase (Nox-4), PTEN, PI3K/p-Akt and p53/Bax. AEOL 10150 has been shown to improve survival and mitigate pulmonary damage in rodent models of SMG, chlorine gas and radiation exposure and in a nonhuman primate (NHP) model of whole thorax lung irradiation (WTLI). Given these promising results, Aeolus is developing AEOL10150 for the mitigation and/or treatment of pulmonary injury resulting from SMG exposure. AEOL 10150 has performed well in animal safety studies, was well tolerated in two human clinical trials and is currently being tested in a third human study. Aeolus has received "Orphan Drug" designation for use in treating Lung ARS, Idiopathic Pulmonary Fibrosis and Amyotrophic Lateral Sclerosis and has active IND's for the Lung ARS and ALS indications. Preparations are currently underway to make IND filings for Idiopathic Pulmonary Fibrosis, Cancer Radiation Therapy and Pulmonary Effects of Sulfur Mustard Gas Exposure. Aeolus Pharmaceuticals is developing a platform of novel compounds, for use in biodefense, fibrosis, oncology, infectious disease and diseases of the central nervous system. Its lead compound, AEOL 10150, is being developed for the treatment of Idiopathic Pulmonary Fibrosis and as a treatment to reduce side effects caused by radiation toxicity and improve local tumor control in cancer therapy. These development efforts have been aided by substantial funding for toxicology, manufacturing, and preclinical and clinical studies from the US Department of Health and Human Services, for the development of AEOL 10150 as a medical countermeasure against chemical and radiological weapons, where its initial target indications are as a protective agent against the pulmonary effects of radiation exposure and sulfur mustard gas exposure. The Company is also developing AEOL 11114 as a treatment for Parkinson's Disease and AEOL 20415 as a treatment for cystic fibrosis and diseases that have developed a resistance to existing antibiotic and anti-viral therapies. For more information, please visit Aeolus's corporate website at www.aolsrx.com. The statements in this press release that are not purely statements of historical fact are forward-looking statements. Such statements include, but are not limited to, those relating to Aeolus' product candidates, as well as its proprietary technologies and research programs, a potential phase 1 study in healthy normal volunteers, the BARDA Contract, and the expected use of proceeds from the financing. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Aeolus' actual results to be materially different from historical results or from any results expressed or implied by such forward-looking statements. Important factors that could cause results to differ include risks associated with uncertainties of progress and timing of clinical trials, scientific research and product development activities, difficulties or delays in development, testing, obtaining regulatory approval, the need to obtain funding for pre-clinical and clinical trials and operations, the scope and validity of intellectual property protection for Aeolus' product candidates, proprietary technologies and their uses, and competition from other biopharmaceutical companies, and whether BARDA exercises one or more additional options under the BARDA Contract. Certain of these factors and others are more fully described in Aeolus' filings with the Securities and Exchange Commission, including, but not limited to, Aeolus' Annual Report on Form 10-K for the year ended September 30, 2016. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.
News Article | May 15, 2017
CONCORD, Calif.--(BUSINESS WIRE)--Cerus Corporation (NASDAQ: CERS) announced today that the first patient has been transfused in Puerto Rico for the “INTERCEPT Blood System for Red Blood Cells in Regions at Potential Risk for Zika Virus Transfusion-Transmitted Infections (RedeS)” clinical trial to assess the safety and efficacy of the INTERCEPT Blood System for Red Blood Cells (RBCs) when compared to conventional RBCs in regions impacted by the Zika virus epidemic. RedeS is a two-stage study being conducted initially in Puerto Rico, a region significantly impacted by the Zika virus epidemic. The study is expected to be expanded to other areas at risk for transfusion-transmitted infections due to the Zika virus, such as Florida. The first stage of the trial is a double-blind, controlled, parallel group trial where 600 adult patients will be randomized to receive up to 28 days of transfusion support with INTERCEPT-treated RBCs or conventional RBCs, with a primary endpoint of hemoglobin increment following transfusion. In a second optional stage, up to 20,000 patients would receive RBC transfusion support with up to 50,000 RBC units in an open-label, single-arm treatment use study. The objective of the second stage is to provide early access to the INTERCEPT pathogen reduction system for RBCs in regions where a substantial proportion of the population has been infected or is at risk of infection by the Zika virus, and the risk of asymptomatic infection among qualified blood donors is recognized. “RedeS marks the first of three pivotal trials expected to support our planned submission to FDA for US licensure of the INTERCEPT Blood System for red cells,” said Richard Benjamin, Cerus’ chief medical officer. “It will lay the ground work for our subsequent anticipated U.S. Phase III trials designed to demonstrate safety and efficacy of INTERCEPT RBCs in cardiovascular surgery patients (the ReCePI study) and chronically transfused patients.” Study RBCs are currently being manufactured and supplied to participating Puerto Rican hospitals by Banco de Sangre de Servicios Mutuos. “We are proud to partner with Cerus by participating in the RedeS study to help move pathogen reduction technology one step closer for red cells,” said Jose O. Alsina, vice president and chief operating officer of Banco de Sangre de Servicios Mutuos, Puerto Rico’s largest blood bank. “Implementing the INTERCEPT Blood System for platelets and plasma allowed us to safely continue to accept donations from our local donor network during the Zika outbreak last year.” RedeS is funded as part of an agreement with the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. Cerus Corporation is a biomedical products company focused in the field of blood transfusion safety. The INTERCEPT Blood System is designed to reduce the risk of transfusion-transmitted infections by inactivating a broad range of pathogens such as viruses, bacteria and parasites that may be present in donated blood. The nucleic acid targeting mechanism of action of the INTERCEPT treatment is designed to inactivate established transfusion threats, such as Hepatitis B and C, HIV, West Nile Virus and bacteria, as well as emerging pathogens such as chikungunya, malaria and dengue. Cerus currently markets and sells the INTERCEPT Blood System for both platelets and plasma in the United States, Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. The INTERCEPT Blood System for Red Blood Cells is in clinical development. See www.cerus.com for information about Cerus. INTERCEPT and INTERCEPT Blood System are trademarks of Cerus Corporation. Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus’ products, prospects and expected results, including statements concerning potential expansion of the RedeS study to other areas at risk for transfusion-transmitted infections due to the Zika virus, and potential future manufacturing scale-up activities, in vitro studies and preparedness for a Phase III clinical trial in the continental U.S clinical trial activity and regulatory submissions. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation: risks associated with the uncertain nature of BARDA’s funding over which Cerus has no control as well as actions of Congress and governmental agencies which may adversely affect the availability of funding under the BARDA contract and/or BARDA’s exercise of any potential subsequent option periods, such that the anticipated activities that Cerus expects to conduct with the funds available from BARDA may be delayed or halted; the uncertain and time-consuming research and development processes that may be necessary prior to the commencement of a Phase III clinical trial; the risks that Cerus may be unable to meet FDA requirements to commence any Phase III clinical studies; the time-consuming clinical trials and regulatory processes that must be completed to obtain regulatory approval of the red blood cell system in a timely manner or at all; as well as other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including in Cerus‘ Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, filed with the SEC on May 4, 2017. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.
News Article | May 3, 2017
CONCORD, Calif.--(BUSINESS WIRE)--Cerus Corporation (NASDAQ: CERS) today announced financial results for the first quarter ended March 31, 2017. “Coming out of our first quarter, we remain optimistic about our growth prospects over the next several years from major markets including the U.S., France and South Africa, but were not able to sign new contracts for the latter two geographies by now as we had previously expected. Therefore, though no less confident in our ability to capture this new business in the near to medium term, we believe it is prudent to adjust our 2017 product revenue guidance to a new range of $43 to 48 million dollars,” said William 'Obi' Greenman, Cerus’ president and chief executive officer. “In the U.S., we are seeing increasing awareness regarding the FDA’s pending final guidance on bacterial safety, and are encouraged by the urgency currently being expressed by blood centers to meet hospital demand for INTERCEPT platelets.” Product revenue recognized during the first quarter of 2017 was $7.0 million compared to $7.6 million during the same period in 2016. The Company experienced strong year-over-year growth for U.S. disposable kits, which was overshadowed by lighter demand for plasma products in EMEA and a weaker Euro compared to the U.S. Dollar. Reported product revenue was negatively affected by an approximate 3% weakening of the Euro compared to the U.S. dollar during the first quarter of 2017 as compared to the same period during 2016. The Company expects 2017 global product revenue in the range of $43 million to $48 million. Gross margins on product revenue for the first quarter of 2017 were 47%, compared to 44% for the first quarter of 2016. Gross margins for the first quarter of 2017 were positively impacted by a more favorable product mix, with higher gross margin platelet kits contributing proportionately more to sales during the first quarter of 2017 than in the prior year period. In addition, the Company realized manufacturing and inventory management efficiencies in the current period compared to the prior year period, which also contributed to the improved gross margins. Total operating expenses for the first quarter of 2017 were $22.8 million, compared to $18.7 million for the first quarter of 2016. Selling, general and administrative expenses increased primarily due to the Company’s increased commercial activity in the U.S. and to a lesser extent, the costs associated with administering the Company’s contract with the Biomedical Advanced Research and Development Authority (BARDA) for INTERCEPT red blood cell development. Research and development expenses increased as a result of increased costs associated with clinical development of the red blood cell system, pursuit of supplemental approvals for the platelet and plasma systems, and development activities under the BARDA agreement. Operating losses during the first quarter of 2017 were $18.1 million, compared to $15.3 million for the first quarter of 2016. Net loss for the first quarter of 2017 was $18.6 million, or $0.18 per diluted share, compared to a net loss of $16.9 million, or $0.17 per diluted share, for the first quarter of 2016. Net losses for the first quarter of 2016 were negatively impacted by non-cash income tax expense of $0.8 million. These tax items are largely the result of changes in the fair value of the Company’s marketable equity investment in Aduro Biotech, Inc. At March 31, 2017, the Company had cash, cash equivalents and short-term investments of $53.8 million compared to $71.6 million at December 31, 2016. At March 31, 2017, the Company had approximately $18.2 million in outstanding debt under its loan agreement with Oxford Finance. The Company will host a conference call and webcast at 4:15 p.m. Eastern time today to discuss its financial results and provide a general business overview and outlook. To access the live webcast, please visit the Investor Relations page of the Cerus website at http://www.cerus.com/ir. Alternatively, you may access the live conference call by dialing 866-235-9006 (U.S.) or 631-291-4549 (international). A replay will be available on the company’s website, or by dialing 855-859-2056 (U.S.) or 404-537-3406 (international) and entering conference ID number 41694810. The replay will be available approximately three hours after the call through May 17, 2017. Cerus Corporation is a biomedical products company focused in the field of blood transfusion safety. The INTERCEPT Blood System is designed to reduce the risk of transfusion-transmitted infections by inactivating a broad range of pathogens such as viruses, bacteria and parasites that may be present in donated blood. The nucleic acid targeting mechanism of action of the INTERCEPT treatment is designed to inactivate established transfusion threats, such as hepatitis B and C, HIV, West Nile virus and bacteria, as well as emerging pathogens such as chikungunya, malaria and dengue. Cerus currently markets and sells the INTERCEPT Blood System for both platelets and plasma in the United States, Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. The INTERCEPT Red Blood Cell system is in clinical development. See http://www.cerus.com for information about Cerus. INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation. Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus’ products, prospects and expected results, including statements concerning Cerus’ 2017 annual product revenue guidance and its expectations for U.S. revenue contribution in 2017 and the timing thereof; Cerus’ expectations regarding increased demand for INTERCEPT components in the U.S.; Cerus’ ability to capture new business in the near to medium; and the timing and likelihood of a future PMA submission to the FDA for the red blood cell system. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation: risks associated with the commercialization and market acceptance of, and customer demand for, the INTERCEPT Blood System, including the risks that Cerus may not meet its adjusted revenue guidance for 2017 and/or realize meaningful revenue contributions from U.S. customers in 2017 or otherwise, particularly since Cerus cannot guarantee the volume or timing of commercial purchases, if any, that its U.S. customers may make under Cerus’ commercial agreements with these customers; risks associated with Cerus’ lack of commercialization experience in the United States and its ability to develop and maintain an effective and qualified U.S.-based commercial organization, as well as the resulting uncertainty of its ability to achieve market acceptance of and otherwise successfully commercialize the INTERCEPT Blood System for platelets and plasma in the United States, including as a result of licensure requirements that must be satisfied by U.S. customers prior to their engaging in interstate transport of blood components processed using the INTERCEPT Blood System; risks related to Cerus’ ability to commercialize the INTERCEPT Blood System in the United States without infringing on the intellectual property rights of others; risks related to Cerus’ ability to demonstrate to the transfusion medicine community and other health care constituencies that pathogen reduction and the INTERCEPT Blood System is safe, effective and economical; the uncertain and time-consuming development and regulatory process, including the risks (a) that Cerus may be unable to comply with the FDA’s post-approval requirements for the INTERCEPT platelet and plasma systems, including by successfully completing required post-approval studies, which could result in a loss of U.S. marketing approval for the INTERCEPT platelet and/or plasma systems, (b) related to Cerus’ ability to expand the label claims and product configurations for the INTERCEPT platelet and plasma systems in the United States, which will require additional regulatory approvals and (c) that Cerus may be unable to file for CE Mark approval of the red blood cell system in Europe in the anticipated timeframe or at all, and even if filed, Cerus may be unable to obtain CE Mark approval, or any other regulatory approvals, of the red blood cell system in a timely manner or at all; risks related to adverse market and economic conditions, including continued or more severe adverse fluctuations in foreign exchange rates and/or weakening economic conditions in the markets where Cerus sells its products; Cerus’ reliance on third parties to market, sell, distribute and maintain its products; Cerus’ ability to maintain an effective manufacturing supply chain, including the ability of its manufacturers to comply with extensive FDA and foreign regulatory agency requirements; the impact of legislative or regulatory healthcare reforms that may make it more difficult and costly for Cerus to produce, market and distribute its products; risks related to future opportunities and plans, including the uncertainty of future revenues and other financial performance and results, as well as other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including Cerus’ Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 8, 2017. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.
News Article | April 21, 2017
The Global Translational Regenerative Medicine market is expected to grow significantly over the forecast period. The Global Translational Regenerative Medicine market was valued at $5.8bn in 2016. Visiongain forecasts this market to increase to $14.5bn in 2021. The market is estimated to grow at a CAGR of 19.9% in the first half of the forecast period and 17.7% from 2016 to 2027. How this report will benefit you Read on to discover how you can exploit the future business opportunities emerging in this sector. In this brand new report you find 316-page report you will receive 107 tables and 66 figures - all unavailable elsewhere. The 316-page report provides clear detailed insight into the Global Translational Regenerative Medicine market. Discover the key drivers and challenges affecting the market. By ordering and reading our brand new report today you stay better informed and ready to act. • Forecasts from 2017-2027 of the leading products in the Global Translational Regenerative Medicine market: - Osteocel Plus - Trinity ELITE - TEMCELL /Prochymal - Apligraf - Dermagraft - Epifix - ReCell - Neovasculgen - Glybera (alipogene tiparvovec) - IMLYGIC (talimogene laherparepvec) • SWOT and Porter's Five Force analysis of the translational regenerative medicine market Visiongain's study is intended for anyone requiring commercial analyses for the Translational Regenerative Medicine Market and leading companies. You find data, trends and predictions. To request a report overview of this report please email Sara Peerun at firstname.lastname@example.org or call Tel: +44-(0)-20-7336-6100 List of Organisations Mentioned in the Report Arthritis Research UK Associazione Infermieristica per lo Studio delle Lesioni Cutanee (AISLeC) [China] Australian Regenerative Medicine Institute Australian Sports Anti-Doping Authority (ASADA) Biomedical Advanced Research and Development Authority (BARDA) British Heart Foundation [UK] California Institute of Regenerative Medicine (CIRM) Cambridge Stem Cell Biology Institute [UK] Case Western Reserve University Catalan Institution for Research and Advanced Studies Center for Biologics Evaluation and Research (CBER) [US] CHA General Hospital [Korea] Cryocenter Saint Petersburg Drugs Controller General of India (DCGI) European Group for Blood and Marrow Transplantation (EBMT) European Medicines Agency Food and Drugs Agency (FDA) [US] Haute Autorité de santé [France] Heriot-Watt University Human Fertilisation and Embryology Authority (HFEA) Institute of Biomedical Research and Innovation Hospital [Japan] International Society for Stem Cell Research (ISSCR) Karolinska Institute [Sweden] Massachusetts General Hospital (MGH) Mayo Clinic [US] Medical Research Council [UK] MiMedx Ministry of Food and Drug Safety, MFDS) [Korea] Ministry of Health, Labour and Welfare (MHLW) [Japan] Ministry of Science and Technology [China] Moorfields Eye Hospital National Tissue Engineering Center (NTEC) [China] New York Blood Center Riken Center for Developmental Biology RUSH University Medical Center [US] Russian Ministry of Healthcare and Social Development Scottish Centre for Regenerative Medicine St. Jude's Children Research Hospital State Food and Drug Administration (SFDA) [China] SUNY Upstate Medical University The Genetico Center [Russia] The StemGen Organisation Therapeutics Goods Administration (TGA) [Australia] UH San Diego Sanford Stem Cell Clinical Center UK Medicines and Healthcare Products Regulatory Agency (MHRA) Universitat Autònoma de Barcelona [Spain] University College London University of Edinburgh MRC Centre for Regenerative Medicine [UK] University of Massachusetts (UMass) Memorial Hospital University of Modena Centre for Regenerative Medicine [Italy] University of Wisconsin US National Institute of Health Wake Forest Institute Wellcome Trust World Health Organization To see a report overview please email Sara Peerun on email@example.com