On May 10, 2016 Aradigm Corporation (NASDAQ: ARDM) (the "Company") reported financial results for the first quarter and three months ended March 31, 2016 (Press release, Aradigm, MAY 10, 2016, View Source [SID:1234512241]).

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Liquidity and Capital Resources

On April 22, 2016, the Company announced the pricing of $23 million of its senior convertible notes due 2021 and related warrants to purchase 263,436 shares of the Company’s common stock in a private placement conducted pursuant to Regulation D under the Securities Act of 1933, as amended. The initial conversion rate will be 191.9386 shares of common stock for each $1,000 principal amount of notes, which represents an initial conversion price of approximately $5.21 per share of common stock. Interest on the notes will be paid semi-annually in arrears at the rate of 9% per year. The warrants are exercisable at an exercise price of $5.21 per share beginning on the later of 180 days after the date of issuance or the date the Company issues a press release announcing data related to the ORBIT-3 and ORBIT-4 Phase 3 pivotal clinical trials in non-cystic fibrosis bronchiectasis (non-CF BE) patients with chronic respiratory infections with Pseudomonas aeruginosa treated with the Company’s investigational product Pulmaquin (proprietary formulation of inhaled ciprofloxacin). The first closing of the sale of the notes and warrants occurred on April 25, 2016, and the second closing is expected to occur immediately after the Company’s resale registration statement to be filed in connection with the offering has been declared effective, each subject to customary closing conditions.

The Company intends to use the net proceeds from the offering, estimated to be $20.7 million, to fund the current clinical development and regulatory submission for licensure of Pulmaquin and for general corporate purposes.

As of March 31, 2016, the Company reported cash and cash equivalents of $22.4 million which did not include the proceeds from the first closing of the private placement offering of $23 million senior convertible notes.

First Quarter 2016 Financial Results

The Company recorded $6,000 in revenue in the first quarter of 2016 compared with $8.8 million in revenue in the first quarter of 2015. The reduction in revenue occurred because the Company utilized in prior periods the full amount of the $65 million of Grifols-funded budget provided under the inhaled ciprofloxacin collaboration arrangement for funding the bronchiectasis program.

Total operating expenses for the first quarter of 2016 were $8.1 million, compared with total operating expenses of $9.9 million for the first quarter of 2015. The decrease in research and development expenses was due to lower contract manufacturing and clinical trial costs because the manufacturing, labeling and packaging expenses for clinical supplies and the enrollment activities of the Pulmaquin Phase clinical trials are complete, offset by higher employee-related expenses due to the higher number of employees and higher consulting expenses in support of the Pulmaquin bronchiectasis regulatory process towards US and EU approvals for market authorization. General and administrative costs were higher primarily due to increased non-cash stock compensation expense and slightly higher legal expense.

Net loss for the first quarter of 2016 was $8.1 million or $0.55 per share, compared with a net loss of $1.2 million or $0.08 per share in the first quarter of 2015. Net loss increased due to lower contract revenue of $8.7 million, partially offset by lower operating expenses of $1.8 million.

About Pulmaquin

Pulmaquin is a dual release formulation composed of a mixture of liposome encapsulated and unencapsulated ciprofloxacin. Ciprofloxacin, available in oral and intravenous formulations, is a widely prescribed antibiotic. It is used to treat acute lung infections and is often preferred because of its broad-spectrum antibacterial activity against various bacteria, such as Pseudomonas aeruginosa. Pulmaquin is being evaluated in two ongoing Phase 3 studies to determine its safety and effectiveness as a once-a-day inhaled formulation for the chronic treatment of patients with non-CF BE who have chronic lung infections with Pseudomonas aeruginosa.

Following Phase 2a development of the liposomal portion of Pulmaquin (Lipoquin) and Phase 1 development of Pulmaquin, the Phase 2b study ORBIT-2 with Pulmaquin was a 24-week multicenter, randomized, double-blind, placebo-controlled trial in 42 adult non-CF BE subjects. This study demonstrated a significant reduction in P.aeruginosa sputum activity (p=0.002) and a decrease in time to first exacerbation in the per protocol population (p=0.046) and the mITT (p=0.057) populations in the Pulmaquin treated subjects compared to placebo. Overall, the incidence of all treatment emergent adverse events was similar between groups. The most frequently reported treatment related adverse events (reported by ≥ 3 patients in either treatment group) included product taste abnormal and nausea in the Pulmaquin group and wheezing in the placebo group. No serious adverse events were considered treatment related. There were no deaths reported during ORBIT-2.

The Phase 3 clinical program for Pulmaquin in non-CF BE consists of two worldwide, double-blind, placebo-controlled pivotal trials (ORBIT-3 and ORBIT-4) that are identical in design except for a pharmacokinetics sub-study to be conducted in one of the trials. Each trial has enrolled patients (278 in ORBIT-3 and 304 in ORBIT-4) into a 48-week double-blind period consisting of 6 cycles of 28 days on treatment with Pulmaquin or placebo plus 28 days off treatment, followed by a 28 day open label extension in which all participants will receive Pulmaquin (total treatment duration approximately one year). The superiority of Pulmaquin vs. placebo during the double-blind period is being evaluated in terms of the time to first pulmonary exacerbation (primary endpoint), while key secondary endpoints include the reduction in the number of pulmonary exacerbations and improvements in the quality of life measures. Lung function is being monitored as a safety indicator.

Aradigm has been granted orphan drug designations for liposomal ciprofloxacin as well as for ciprofloxacin for inhalation for non-CF BE in the U.S. In addition, the U.S. Food and Drug Administration (FDA) has designated Pulmaquin as a Qualified Infectious Disease Product (QIDP). The QIDP designation is granted for treatment of non-CF BE patients with chronic lung infections with Pseudomonas aeruginosa. The QIDP designation made Pulmaquin eligible for Fast Track designation which was granted by the FDA in September 2014.

In 2013, Aradigm granted an exclusive, world-wide license for the Company’s inhaled liposomal ciprofloxacin product candidates for the indication of non-CF BE and other indications to Grifols S.A. More information on the terms of this license may be found in the Company’s Annual Report on Form 10-K for the year ended December 31, 2013 filed with the SEC on March 13, 2014.

About Non-Cystic Fibrosis Bronchiectasis

Non-CF BE is a severe, chronic and rare disease characterized by abnormal dilatation of the bronchi and bronchioles, frequently associated with chronic lung infections. It is often a consequence of a vicious cycle of inflammation, recurrent lung infections, and bronchial wall damage. Non-CF BE represents an unmet medical need with high morbidity and mortality that affects more than 110,000 people in the U.S. and over 200,000 people in Europe. There is currently no drug approved for the treatment of this condition.

On May 10, 2016 Celator Pharmaceuticals, Inc. (Nasdaq: CPXX) reported business highlights and financial results for the first quarter ended March 31, 2016 (Press release, Celator Pharmaceuticals, MAY 10, 2016, View Source [SID:1234512203]).

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"The first quarter of 2016 was transformative for Celator," said Scott Jackson, chief executive officer of Celator. "Our Phase 3 trial of VYXEOS demonstrated a statistically significant improvement in overall survival, among other benefits, in patients with high-risk acute myeloid leukemia (AML) and we plan to submit a New Drug Application (NDA) to the Food and Drug Administration (FDA) by the end of the third quarter of this year. We look forward to presenting additional data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting next month. In addition, following the positive Phase 3 results, we raised capital that we believe is sufficient to fund planned operations into 2018."

Celator anticipates achieving the following key objectives:

Continue to execute on the VYXEOS commercial readiness in the United States;
Submit the New Drug Application (NDA) for VYXEOS by the end of Q3 2016;
Continue to expand the clinical development of VYXEOS via investigator-initiated studies, oncology cooperative groups and company-sponsored studies;
Prescription Drug User Fee Act (PDUFA) date (assuming FDA grants priority review) by mid-2017; and
United States commercial launch (assuming FDA approval in mid-2017) in Q3 2017.
Recent Business and First Quarter 2016 Highlights

April – Celator announced that a late-breaking abstract was submitted to ASCO (Free ASCO Whitepaper) and accepted for an oral presentation on June 4, 2016 at 3:00 pm CT and will be presented by Dr. Jeffrey Lancet from H. Lee Moffitt Cancer Center & Research Institute.
March – Celator announced that Phase 3 trial data for VYXEOS in patients with high-risk AML demonstrated a statistically significant improvement in overall survival and induction response rate and meaningfully lower 60-day mortality. There was no substantial difference in Grade 3-5 adverse events between VYXEOS and the control arm (7+3 regimen). The company plans to submit an NDA for VYXEOS by the end of the third quarter.
March – Celator completed an underwritten public offering of 4,600,000 shares of the company’s common stock, resulting in net proceeds of approximately $40.6 million.
Financial Highlights

Cash Position: Cash and cash equivalents as of March 31, 2016 were $67.5 million, compared to $23.3 million as of December 31, 2015. The increase was primarily due to the underwritten public offering that netted approximately $40.6 million during the first quarter of 2016 and $9.8 million in net proceeds from the issuance of common stock through an at-the-market equity offering program. Management believes that the cash and cash equivalents at March 31, 2016 will be sufficient to meet estimated working capital requirements and fund planned operations into 2018.
R&D Expenses: Research and development expenses were $2.7 million for the three months ended March 31, 2016 consistent with the same period in 2015.
G&A Expenses: General and administrative expenses were $2.5 million for the three months ended March 31, 2016, as compared to $1.8 million for the same period of 2015. The increase was primarily attributable to increases in compensation, pre-launch commercial activities, public company expenses and professional fees.
Net Loss: Net loss increased to $5.5 million for the three months ended March 31, 2016, from $4.7 million for the same period in 2015.

On May 10, 2016 Repros Therapeutics Inc. (Nasdaq:RPRX) reported financial results for the first quarter ended March 31, 2016 (Press release, Repros Therapeutics, MAY 10, 2016, View Source [SID:1234512321]).

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Financial Results

Net loss for the three month period ended March 31, 2016 was ($4.8) million, or ($0.20) per share, as compared to a net loss of ($8.5) million, or ($0.35) per share, for the same period in 2015. The decrease in net loss for the three month period ended March 31, 2016 as compared to the same period in 2015 was primarily due to decreases in clinical development expenses related to the Company’s enclomiphene product candidate, research and development payroll and benefits expenses and legal expenses, partially offset by an increase in clinical development expenses related to Proellex.

Research and development ("R&D") expenses decreased 49%, or $3.5 million, to $3.8 million for the three month period ended March 31, 2016, as compared to $7.3 million for the same period in the prior year. R&D expenses related to the clinical development of enclomiphene decreased 71%, or approximately $2.9 million, for the three month period ended March 31, 2016 as compared to the prior year period, primarily due to the submission of the NDA to the FDA in the first quarter of 2015. R&D expenses related to the clinical development of Proellex increased 41%, or approximately $336,000, from the 2015 period to the 2016 period, due to increased expenses associated with our ongoing Phase 2B clinical trials for the treatment of uterine fibroids. Payroll and benefits expenses decreased 45%, or approximately $580,000, to $712,000 for the three month period ended March 31, 2016 as compared to $1.3 million for the same period in the prior year. Included in payroll and benefits expense is a charge for non-cash stock-based compensation of $193,000 for the three month period ended March 31, 2016 as compared to $574,000 for the same period in the prior year. Additionally, salaries for the three month period ended March 31, 2016 were $422,000 as compared to $579,000 for the same period in the prior year. Operating and occupancy expenses decreased 37%, or approximately $423,000, to $716,000 for the three month period ended March 31, 2016 as compared to $1.1 million for the same period in the prior year, primarily due to decreased legal expenses.

General and administrative ("G&A") expenses decreased 9%, or approximately $109,000, to $1.1 million for the three month period ended March 31, 2016 as compared to $1.2 million for the same period in the prior year. Payroll and benefits expense decreased 17%, or approximately $123,000, to $620,000 for the three month period ended March 31, 2016 as compared to $743,000 for the same period in the prior year. Included in payroll and benefits expense is a charge for non-cash stock based compensation expense of $323,000 for the three month period ended March 31, 2016 as compared to $448,000 for the same period in the prior year. Additionally, salaries for the three month period ended March 31, 2016 were $257,000 as compared to $255,000 for the same period in the prior year. G&A operating and occupancy expense increased 3%, or approximately $14,000, to $476,000 for the three month period ended March 31, 2016 as compared to $462,000 for the same period in the prior year primarily due to an increase in legal expenses.

Interest income increased to $17,000 for the three month period ended March 31, 2016 as compared to $1,000 for the same period in the prior year. The increase was primarily due to higher yields for the three month period ended March 31, 2016 as compared to the prior year.

Liquidity and Capital Resources

The Company had cash and cash equivalents of $16.0 million as of March 31, 2016 as compared to $21.4 million as of December 31, 2015. Net cash of approximately $5.3 million and $8.8 million was used in operating activities during the three month periods ended March 31, 2016 and 2015, respectively. The major use of cash for operating activities for the three month period ended March 31, 2016 was to fund our clinical development programs and associated administrative costs. No cash was used in investing activities during the three month period ended March 31, 2016 and no cash was provided by financing activities during the three month period ended March 31, 2016.

As of March 31, 2016, the Company had 24,318,111 shares of common stock outstanding.

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.