Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies, today announced new two-year data that suggest long-term efficacy of daily treatment with PALFORZIA™ [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in patients with peanut allergy. Patients dosed daily in the study also showed ongoing immunomodulation and higher rates of desensitization that increased over time compared to those patients who were administered non-daily dosing. Furthermore, the majority of patients in the daily dosing groups reported lower adverse event (AE) rates compared to those in the non-daily dosing groups, with an overall safety profile of long-term daily dosing with PALFORZIA that was better than non-daily dosing.
These two-year results from ARC004, a follow-on study to the pivotal phase 3 PALISADE trial, were presented during the digital European Academy of Allergy and Clinical Immunology (EAACI) Congress and are available for on-demand viewing at EAACI scientific programme website.
“We’re excited that these two-year data provide not only further insight into the long-term efficacy and safety profile of PALFORZIA resulting from continued daily treatment, but also show that patients exhibit the long-term effects of modulating the immune system,” said Daniel Adelman, M.D., Chief Medical Officer of Aimmune. “These are important data that reinforce and build on the findings from our pivotal PALISADE trial. We look forward to continuing to evaluate these patient groups over time to assess how daily treatment with PALFORZIA may result in further disease modification. We are grateful to the patients who continued their daily treatment to allow us to report these results with a high study completion rate.”
A greater proportion of patients who continued daily long-term dosing with PALFORZIA in ARC004 completed the study compared to those patients in the non-daily treatment groups. Furthermore, after two years of daily treatment with PALFORZIA, more than 80% of patients undergoing the food challenge demonstrated desensitization to 2000 mg peanut protein (4043 mg cumulatively [equivalent to ~14 peanut kernels]). Desensitization rates associated with PALFORZIA treatment were higher in the daily dosing groups compared to the non-daily dosing groups; within the daily dosing groups, desensitization rates improved with duration of therapy.
Changes in immunologic measures showed ongoing immunomodulation during the first two years of treatment with PALFORZIA with further modulation of immunologic and improvement in clinical parameters in subsequent years anticipated to occur.
Patients in the two daily dosing groups also reported fewer treatment-related adverse events (TRAE) when adjusted for the number of patient-years of exposure compared to the three non-daily dosing groups. Adverse events in the daily dosing groups were mostly mild to moderate with only three patients reporting severe events observed in over 100 patient-years of exposure. The overall rate of AE-related discontinuations was low but slightly higher in the longer-term non-daily dosing groups. Across treatment groups, PALFORZIA appeared to be well tolerated and was consistent with previous studies with no new safety concerns.
“The findings from ARC004 suggest that long-term daily dosing with PALFORZIA lead to higher rates of desensitization and ongoing immunomodulation, underscoring that for at least the first two years of treatment, daily dosing during maintenance is the optimal approach for this patient population,” continued Dr. Adelman.
PALFORZIA was approved by the U.S. Food and Drug Administration (FDA) in January 2020 as an oral immunotherapy for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut in patients aged 4 through 17 years with a confirmed diagnosis of peanut allergy. In Europe, Aimmune’s marketing authorization application for AR101 is under review with the European Medicines Agency (EMA). A Swiss Agency for Therapeutic Products (SwissMedic) review of AR101 also is ongoing.
ARC004 was an open-label follow-on study to the phase 3 PALISADE trial and assessed the safety and efficacy of extended daily and non-daily dosing of PALFORZIA across 351 peanut-allergic patients aged 4-17 years. Participants who received placebo (AR101-naïve) in PALISADE received active treatment with AR101, and eligible participants who received AR101 in PALISADE (AR101 continued) were assigned to either daily or non-daily continued treatment regimen with different durations. A total of 261 AR101-treated participants completed the ACR004 study.
PALFORZIA is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut. PALFORZIA is approved for use in patients with a confirmed diagnosis of peanut allergy. Initial Dose Escalation may be administered to patients aged 4 through 17 years. Up-Dosing and Maintenance may be continued in patients 4 years of age and older.
PALFORZIA is to be used in conjunction with a peanut-avoidant diet.
Limitations of Use: Not indicated for the emergency treatment of allergic reactions, including anaphylaxis.
IMPORTANT SAFETY INFORMATION
PALFORZIA can cause anaphylaxis, which may be life threatening and can occur at any time during PALFORZIA therapy.
Prescribe injectable epinephrine, instruct and train patients on its appropriate use, and instruct patients to seek immediate medical care upon its use.
Do not administer PALFORZIA to patients with uncontrolled asthma.
Dose modifications may be necessary following an anaphylactic reaction.
Observe patients during and after administration of the Initial Dose Escalation and the first dose of each Up-Dosing level, for at least 60 minutes.
PALFORZIA is available only through a restricted program called the PALFORZIA REMS.
PALFORZIA is contraindicated in patients with uncontrolled asthma, or with a history of eosinophilic esophagitis and other eosinophilic gastrointestinal disease.
WARNINGS AND PRECAUTIONS
PALFORZIA can cause anaphylaxis, which may be life threatening. PALFORZIA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the PALFORZIA REMS because of the risk of anaphylaxis. Only prescribers, healthcare settings, pharmacies, and patients certified and enrolled in the REMS Program can prescribe, receive, dispense or administer PALFORZIA.
Anaphylaxis has been reported during all phases of PALFORZIA dosing, including Maintenance and in subjects who have undergone recommended Up-Dosing and dose modification procedures.
Do not initiate PALFORZIA treatment in a patient who has had severe or life-threatening anaphylaxis within the previous 60 days. PALFORZIA may not be suitable for patients with certain medical conditions that may reduce the ability to survive anaphylaxis, including but not limited to markedly compromised lung function, severe mast cell disorder, or cardiovascular disease. In addition, PALFORZIA may not be suitable for patients taking medications that can inhibit or potentiate the effects of epinephrine.
All Initial Dose Escalation doses and the first dose of each Up-Dosing level must be administered in a certified health care setting.
Patients may be more likely to experience allergic reactions following PALFORZIA administration in the presence of cofactors such as exercise, hot water exposure, intercurrent illness (e.g., viral infection), or fasting. Other potential cofactors may include menstruation, sleep deprivation, nonsteroidal anti-inflammatory drug use, or uncontrolled asthma. Patients should be proactively counseled about the potential for the increased risk of anaphylaxis in the presence of these cofactors. If possible, adjust the time of dosing to avoid these cofactors. If it is not possible to avoid these cofactors, consider withholding PALFORZIA temporarily.
Uncontrolled asthma is a risk factor for a serious outcome, including death, in anaphylaxis. Ensure patients with asthma have their asthma under control prior to initiation of PALFORZIA.
PALFORZIA should be temporarily withheld if the patient is experiencing an acute asthma exacerbation. Following resolution of the exacerbation, resumption of PALFORZIA should be undertaken cautiously. Re-evaluate patients who have recurrent asthma exacerbations and consider discontinuation of PALFORZIA.
Eosinophilic Gastrointestinal Disease
Discontinue PALFORZIA and consider a diagnosis of eosinophilic esophagitis in patients who experience severe or persistent gastrointestinal symptoms, including dysphagia, vomiting, nausea, gastroesophageal reflux, chest pain, or abdominal pain.
Gastrointestinal Adverse Reactions
Gastrointestinal adverse reactions were commonly reported in PALFORZIA-treated subjects, and dose modification should be considered for patients who report these reactions. For severe or persistent gastrointestinal symptoms consider a diagnosis of eosinophilic esophagitis.
The most common adverse events reported in subjects treated with PALFORZIA (incidence ≥ 5% and ≥ 5% than placebo) are abdominal pain, vomiting, nausea, oral pruritus, oral paresthesia, throat irritation, cough, rhinorrhea, sneezing, throat tightness, wheezing, dyspnea, pruritus, urticaria, anaphylactic reaction, and ear pruritus.
Please see full Prescribing Information, including Boxed WARNING, and Medication Guide at www.PALFORZIA.com.
For more information about PALFORZIA, please call 1-844-PALFORZ (1-844-725-3679) or visit www.PALFORZIA.com.
Aimmune Therapeutics, Inc. is a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies. With a mission to improve the lives of people with food allergies, Aimmune is developing and commercializing oral treatments for potentially life-threatening food allergies. The Company’s Characterized Oral Desensitization ImmunoTherapy (CODIT™) approach is intended to provide meaningful levels of protection against allergic reactions resulting from accidental exposure to food allergens by desensitizing patients with defined, precise amounts of key allergens. Aimmune has one FDA-approved medicine for peanut allergy and other investigational therapies in development to treat other food allergies. For more information, please visit www.aimmune.com.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Aimmune’s expectations regarding its evaluation of the potential benefits of daily treatment with PALFORZIA, including benefits related to changes in immunologic measures;; and Aimmune’s expectations regarding potential applications of the CODIT approach to treating life-threatening food allergies. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: the risk that the COVID-19 worldwide pandemic may continue to negatively impact the business, research and clinical operations of Aimmune or its partners; Aimmune’s or any of its collaborative partners’ ability to initiate and/or complete clinical trials; the unpredictability of the regulatory process; the possibility that Aimmune’s or any of its collaborative partners’ clinical trials will not be successful; Aimmune’s dependence on the success of PALFORZIA; Aimmune’s reliance on third parties for the manufacture of Aimmune’s products and product candidates; possible regulatory developments in the United States and foreign countries; and Aimmune’s ability to attract and retain senior management personnel. These and other risks and uncertainties are described more fully in Aimmune's most recent filings with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended March 31, 2020. All forward-looking statements contained in this press release speak only as of the date on which they were made. Aimmune undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
This press release concerns PALFORZIA, which has been approved for marketing by the FDA in the United States and has not been approved for marketing by the EMA or Swissmedic. PALFORZIA in Europe is currently limited to investigational use, and no representation is made as to its safety or effectiveness for the purposes for which it is being investigated.
PALFORZIA™, AIMMUNE®, AIMMUNE THERAPEUTICS® and CODIT™ are trademarks of Aimmune Therapeutics, Inc.