●risk of significant lawsuits, including stockholder litigation, which is common in the life sciences sector;
●our dependence on performance by third-party providers of services and supplies;
●the impact of development of competing therapies and/or technologies by other companies;
●risks related to our supply of drug product to Pfizer;
●risks related to our expectations with respect to the potential commercial value of our product and product candidates;
●risks relating to the compliance by Fiocruz, an arm of the Brazilian MoH, with its purchase obligations under our supply and technology transfer agreement, which may have a material adverse effect on us and may also result in the termination of such agreement;
●potential product liability risks, and risks of securing adequate levels of related insurance coverage;
●the possibility of infringing a third-party’s patents or other intellectual property rights and the uncertainty of obtaining patents covering our products and processes and successfully enforcing our intellectual property rights against third-parties;
●risks relating to changes in healthcare laws, rules and regulations in the United States or elsewhere; and
●the possible disruption of our operations due to terrorist activities and armed conflict, including as a result of the disruption of the operations of regulatory authorities, our subsidiaries, our manufacturing facilities and our customers, suppliers, distributors, collaborative partners, licensees and clinical trial sites.
Recent Company Developments
●On May 13, 2021, we signed a binding term sheet with Chiesi pursuant to which we amended the Chiesi Agreements in order to provide us with near-term capital. Chiesi agreed to make a $10.0 million milestone payment to us before the end of the second quarter in exchange for a $25.0 million reduction in a longer term regulatory milestone payment in the Chiesi EX-US Agreement. All other regulatory and commercial milestone payments remain unchanged. We also agreed to negotiate certain manufacturing related matters.
●On April 28, 2021, we, together Chiesi, announced the receipt from the FDA of a CRL in response to the PRX-102 BLA.
●On February 23, 2021, we, together with Chiesi, announced positive topline results from our phase III BRIGHT clinical trial of PRX-102 for the treatment of Fabry disease, or the BRIGHT study, designed to evaluate the safety, efficacy and pharmacokinetics of PRX-102 treatment, 2 mg/kg every four weeks, in up to 30 patients with Fabry disease previously treated with a commercially available ERT (agalsidase alfa – Replagal® or agalsidase beta – Fabrazyme®).
●On February 18, 2021, we announced the closing of a public offering of our common stock raising gross proceeds of approximately $40.2 million at a price equal to $4.60 per share, before deducting the underwriting discount and estimated expenses of the offering.
●On February 11, 2021, we announced an exclusive worldwide license agreement with SarcoMed for PRX-110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
We continue to actively advance all our clinical programs. We are in close contact with our principal investigators and clinical sites and our clinical research organizations, which are primarily located in the United States and Europe, and to date, our clinical trials have not been materially adversely affected by COVID-19. In light of recent developments relating to the COVID-19 pandemic, the focus of healthcare providers and hospitals on fighting the virus, and consistent with the FDA’s updated industry guidance for conducting clinical trials issued on March 18, 2020, we and our contract research organizations have made certain adjustments to the operation of our clinical trials in an effort to ensure the monitoring and safety of patients and minimize risk to trial integrity during the pandemic and generally, and we may need to make further adjustments in the future.