ARS Pharmaceuticals Highlights Progress and Reports First Quarter 2023 Financial Results
FDA Advisory Committee Votes in Support of Favorable Benefit-Risk Profile for neffy® for the Treatment of Allergic Reactions (Type I), Including Anaphylaxis
New Drug and Marketing Authorization Applications for neffy Under Review with FDA and EMA, Respectively; FDA PDUFA Target Action Date Anticipated mid-2023
“For the millions of people living with Type I allergic reactions including anaphylaxis, we believe that neffy (nasal epinephrine spray), a small, needle-free and easy-to-use nasal spray, could provide a much-needed alternative to the currently approved epinephrine injection devices. Despite their effectiveness, these devices are limited in usage because many patients and caregivers do not carry, avoid using or hesitate to use them,” said
- FDA Advisory Committee Supports neffy Potential for the Treatment of Allergic Reactions (Type I), Including Anaphylaxis, Under Review with FDA and EMA: The
U.S.Food and Drug Administration’s (FDA) held a Division of Pulmonary-Allergy Drug Advisory Committee(PADAC) meeting on May 11, 2023, to review the New Drug Application (NDA) for neffy. The committee voted 16:6 in favor for adults, and 17:5 in favor for children (<18 years of age and ≥30 kg), that available data support a favorable benefit-risk assessment for neffy in the treatment of severe allergic reaction (Type I), including anaphylaxis, for adults and children who weigh more than 30kg. The PADAC decision was based on a review of comprehensive data from clinical studies developed in agreement with the FDA, which support a positive risk-benefit profile for intranasal (IN) epinephrine safety and effectiveness, compared to epinephrine injection. We believe the studies demonstrated and the majority of the Committee’s discussion supported that neffy shows:
- Comparable or greater pharmacodynamic (PD) response (systolic blood pressure and heart rate) vs. intramuscular injection that is observed even at 1 minute after dosing of neffy. Increases in systolic blood pressure and heart rate are the outcomes monitored by physicians to assess clinical response, and therefore ARS considered PD response to be a surrogate for efficacy. The majority conclusion of the Committee also suggests that PD was more important and informative given the high PK variability of injection products.
- Comparable or greater pharmacokinetic (PK) data (epinephrine levels in the blood) vs. intramuscular injection at all time points based on integrated data across the three primary clinical studies. This includes a “real-world” self-administration study in allergic reaction (Type I) patients, where PK was statistically greater than intramuscular injection during the early timepoints when clinical response is observed. Importantly, exposures with neffy in all clinical studies were less than the upper limit represented by EpiPen to ensure safety.
- Effective IN delivery of systemic epinephrine and PD response even with nasal congestion or runny nose (e.g., during allergic rhinitis or upper respiratory tract infection)
- Comparable safety to injection that is generally mild in nature without any meaningful nasal irritation or pain, without needle-related risks
- FDA PDUFA Target Action Date Anticipated Mid-2023: The Company’s new drug application (NDA) and marketing authorization application (MAA) for neffy for the emergency treatment of allergic reactions (Type I) including anaphylaxis in adults and children ≥30 kg (66 lbs), are under review by the FDA and the
European Medicines Agency(EMA), respectively. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date anticipated mid-2023. If approved, neffy would be the first non-injectable treatment available to patients with allergic reactions (Type I) including anaphylaxis.
First Quarter 2023 Financial Results
- Cash Position: Cash, cash equivalents and short-term investments were
$264.5 millionas of March 31, 2023, which ARS believes are sufficient to fund its current operating plan for at least three years.
- R&D Expenses: Research and development (R&D) expenses were
$6.6 millionfor the quarter ended March 31, 2023.
- G&A Expenses: General and administrative (G&A) expenses were
$12.2 millionfor the quarter ended March 31, 2023.
- Net Loss: Net loss was
$15.0 millionfor the quarter ended March 31, 2023.
About Type I Allergic Reactions including Anaphylaxis
Type I severe allergic reactions are serious and potentially life-threatening events that can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these reactions. While epinephrine autoinjectors have been shown to be highly effective, there are well published limitations that result in many patients and caregivers delaying or not administering treatment in an emergency situation. These limitations include fear of the needle, lack of portability, needle-related safety concerns, lack of reliability, and complexity of the devices. There are approximately 25 to 40 million people in
ARS is a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis. The Company is developing neffy® (also referred to as ARS-1), an intranasal epinephrine product in clinical development for patients and their caregivers with Type I allergic reactions including food, medications and insect bites that could lead to life-threatening anaphylaxis. For more information, visit www.ars-pharma.com.
Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, ARS’s projected cash runway; the anticipated timing for regulatory review decisions on neffy and the potential approval of neffy; the anticipated US launch of neffy, if approved, and the timing thereof; ARS’s strategy of pursuing potential strategic transactions or partnerships for neffy in
The forward-looking statements included in this press release are made only as of the date hereof. ARS assumes no obligation and does not intend to update these forward-looking statements, except as required by law.
ARS Investor Contact:
Condensed Consolidated Balance Sheets
(in thousands, except share and par value data)
|Cash and cash equivalents||$||87,862||$||210,518|
|Prepaid expenses and other current assets||2,801||3,319|
|Total current assets||267,350||277,700|
|Fixed assets, net||584||329|
|Liabilities, convertible preferred stock and stockholders’ equity|
|Accounts payable and accrued liabilities (including related party amounts of
|Lease liability, current||232||230|
|Contract liability, current||10||283|
|Total current liabilities||9,838||5,444|
|Lease liability, net of current portion||199||251|
|Contract liability, net of current portion||—||2,854|
|Commitments and contingencies|
|Additional paid-in capital||352,977||349,408|
|Accumulated other comprehensive gain||68||407|
|Total stockholders’ equity||261,155||272,886|
|Total liabilities, convertible preferred stock and stockholders’ equity||$||271,192||$||281,435|
Condensed Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share data)
|Three Months Ended
|Revenue under collaboration agreements||$||20||$||663|
|Research and development (including related party amounts of
|General and administrative (including related party amounts of
|Total operating expenses||18,733||7,762|
|Loss from operations||(18,713||)||(7,099||)|
|Other income (expense), net||3,752||(151||)|
|Change in unrealized gain on available-for-sale securities||(339||)||—|
|Net loss per share, basic and diluted||$||(0.16||)||$||(0.24||)|
|Weighted-average shares outstanding used in computing net loss per share, basic and diluted||94,227,313||30,369,413|
ARS Pharmaceuticals, Inc.