San Diego biotech offers anaphylaxis medicine without needles
Richard Lowenthal, president and chief executive officer of ARS Pharmaceuticals, holds a case for its product Neffy in San Diego. ARS Pharmaceuticals received FDA approval for Neffy, a nasal spray alternative to the EpiPen.
By NATALLIE ROCHA
The San Diego Union-Tribune (TNS)
Alyssa Bauder was just learning how to read, but she had already memorized the colors of food packaging that spelled danger for her: peanuts. The 6-year-old knew to stay away from chocolate dressed in orange or blue and yellow wrappers.
Then everything got bungled on Valentine’s Day. Her first grade class exchanged handfuls of candy all disguised in special pink and red designs.
When Alyssa got home, she tore away the pretty candy wrapper and took a bite of a mini chocolate bar not knowing it was a Butterfinger. Immediately, something didn’t feel right.
Was it her chest or her throat that felt funny?
She wanted water, but couldn’t move.
The bottle was right in front of her, but she was frozen.
Alyssa screamed for water from her parents, then started vomiting.
Alyssa had no words to describe the scary situation taking over her body: an anaphylactic reaction. Her parents had never seen this happen before and didn’t have anything on hand to make it stop.
The panic of an extreme allergic reaction, or anaphylaxis, is the reality for millions of kids and adults across the U.S.
Reactions are often triggered by food, medication or insect stings and can only be quelled by injecting epinephrine, also known as adrenaline, with a syringe or EpiPen. The needles, however, can add to the trauma of an attack and make patients hesitant to inject the medicine or even keep it on hand.
But, for the first time, injecting epinephrine isn’t the only option. A San Diego-based company, ARS Pharmaceuticals, has developed a potentially ground-breaking needle- free alternative: an epinephrine nasal spray. Doctors and patients say a needle-free option could help people react quicker and administer the medicine without fear.
Neffy – a name that’s intentionally easy to say and sounds kid-friendly – was recently approved by the U.S. Food and Drug Administration and is now available to people with severe allergies.
“Anaphylaxis is life-threatening and some people, particularly children, may delay or avoid treatment due to fear of injections,” said Dr. Kelly Stone, associate director of the Division of Pulmonology, Allergy and Critical Care in the FDA’s Center for Drug Evaluation and Research. “The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis. As a result, Neffy provides an important treatment option and addresses an unmet need.”
The device fits in the palm of your hand and can be sprayed into the nose using just three fingers. The medicine was also formulated in a way that doesn’t require the patient to inhale – it just has to get into the nose.
It’s the same exact delivery system as Narcan, the emergency nasal spray used to treat opioid overdoses. That’s no coincidence.
Richard Lowenthal, CEO of ARS Pharmaceuticals, said Neffy is the third product he has worked on that employs this nasal spray delivery system. Prior to ARS, he was part of the team that got Narcan approved as a nasal spray alternative to Naloxone injections.
Lowenthal saw how a severe food allergy affected the life of a close friend. The problem was right in front of him and it got the wheels turning on how to apply this drug technology to epinephrine.
Neffy has been in the works for about eight years, and it took some back and forth to get it through the FDA, an agency Lowenthal previously worked for. Epinephrine is not a new drug – the EpiPen auto- injector has been on the market for about three decades – but the agency wanted to see substantial data to back the novel delivery method.
Lowenthal said the ultimate goal is to make this treatment more accessible to the approximately 40 million people in the US who experience severe allergic reactions, based on insurance claims data. Out of that population, he said 3.3 million patients have an active prescription for epinephrine and only half of them consistently carry an epinephrine auto-injector.
But the fear of needles often leads many people to delay or not inject at all.
“If you’re that happy EpiPen user … that’s not the patient we’re interested in,” Lowenthal said. “We’re interested in the patients that have auto-injectors that are terrified to use them or don’t carry them.”
Lowenthal hopes a nasal spray like Neffy can minimize the trepidation around treating the source of allergic reactions.
“You get the same medicine, but you get it in a way where you’re not afraid, you’re not scared to do it and it’s very simple, easy and you can administer it very quickly,” Lowenthal said of Neffy. “And that’s the key to this disease – is the sooner you recognize that you’re having a reaction and you treat it and stop it.”
The trouble with delaying epinephrine
Since Alyssa Bauder’s first experience with anaphylaxis on Valentine’s Day, she has carried two epinephrine autoinjectors with her at all times.
What she didn’t expect to be part of her allergic reactions was the aftermath. She didn’t want to go to school, was terrified to eat and didn’t want to be away from her parents. As she got older, Bauder was scared to move away for college.
“I have very few childhood memories that are in a world before anaphylaxis, because I was only 6 years old when I had my first reaction,” Bauder said. “So much of my life has been colored by that experience. I always say, it’s kind of like living in a body that could betray me at any time.”
Now, a 27-year-old graduate student in Chicago, she’s studying the psychology and mental impacts of allergies on patients. She’s learned how to manage her allergies, but even as an adult, Bauder fears self-injecting with epinephrine.
Last year while having dinner with her boyfriend’s family, she finally had to face that reality. Bauder felt a weird sensation in her throat, then her stomach started to hurt, so she excused herself to the bathroom.
“Maybe it’s nothing,” she thought. Then her boyfriend walked in.
“I’m not really sure what’s going on,” she said. “It might be a reaction, but it might not be.”
“I think you should do your EpiPen,” he said.
Thirty minutes after debating what to do and calling her parents for backup, Bauder reluctantly opened the cap of her emergency EpiPen and raised the needle above her thigh.
“I wasn’t necessarily afraid of the needle, but the whole idea of accepting that I was having an allergic reaction,” she said. “This EpiPen that I’ve carried for my entire life at that point … has always been in my back pocket and now is the time I’ve got to pull it out and use it.”
When patients wait too long to inject epinephrine, that’s when an allergic reaction can become more severe or even deadly, said Dr. Raffi Tachdjian, an immunologist and professor at UCLA. He works with patients from kids to adults, who experience severe allergies and is part of ARS Pharma’s speakers bureau, where he is compensated for speaking on behalf of the company.
In addition to needle phobia, he’s identified several hurdles that stop people from injecting epinephrine sooner.
Some people – especially parents caring for young kids – can’t always identify early symptoms like chest tightness or dizziness with anaphylaxis until more obvious signs like skin rashes or closing airways show up. Then, there’s the challenge of access to epinephrine – either because a patient doesn’t carry it with them or it hasn’t been prescribed.
“Generally speaking, that’s the million dollar question: when should someone know that it’s time to use epinephrine?” Tachdjian said. “If you’re thinking it, just go ahead and use it, because there’s no contraindication (or harm) for using epinephrine.”
Neffy is available by prescription and through the company’s website. The price is $25 to $199 for two single-use Neffy devices, depending on a patient’s insurance coverage. It’s free for some eligible patients through the company’s patient assistance program.
Lowenthal said Neffy comes with a little zip-up carrying case that can fit discreetly in a purse or get hooked onto a child’s backpack so they can access it quickly. He added that its shelf life is a little over two years and it’s not super sensitive to temperature changes. Typically, epinephrine auto-injectors last 18 to 20 months and are heat sensitive, particularly if the device is left in a car, Lowenthal said.
How does Neffy compare to current treatments?
ARS Pharma submitted clinical data to the FDA that examined how effective Neffy was in 59 healthy adults.
The study compared Neffy to two other approved treatments, an EpiPen auto-injector and an intramuscular epinephrine shot. Participants had their blood pressure and heart rate measured to gauge the effectiveness of each treatment because epinephrine raises both.
One dose of Neffy showed comparable results to the other drugs. Repeated doses of Neffy showed even stronger results for how quick and effective it was in patients.
The data looks very promising to Dr. Tina Sindher, a professor of allergy and immunology at Stanford, who also treats many children with food allergies. For instance, she was surprised to see that Neffy worked more quickly than current intramuscular epinephrine shots in the study.
Sindher – who is not affiliated with ARS Pharma – also liked that the study showed Neffy to be effective for people experiencing nasal polyps and congestion, since it’s delivered through the nose. She also finds it reassuring that Neffy delivers epinephrine using a device that is already FDA approved for Narcan.
Part of what makes this possible is Neffy’s dose of epinephrine is paired with intravail, which has been used alongside multiple other FDA-approved drugs. It essentially props the door open at the cellular level so the medicine can absorb easily into the body.
For those reasons Sindher said she’s open to trying Neffy with her patients, some of whom have already asked her about it.
However, Sindher said she would like to see more real world data from the company before she feels super confident in Neffy. The caveat to the promising data is that it was conducted in healthy adults, not those experiencing an anaphylactic reaction. She acknowledged that there are ethical barriers to collecting data for a new treatment like Neffy since anaphylaxis is considered life threatening. One way to overcome that and understand how Neffy works for people undergoing active anaphylaxis is by keeping it stocked in clinic settings under observation, she said.
“Still for me, I think I have to see it in action,” said Sindher. “I have to be able to use it for my patients, see how quickly they respond, look at how they feel about it, for me to start being comfortable.”
While Neffy creates a new option for patients, Sindher noted it will take time for there to be a visible industrywide move to the new treatment.
“Adopting new things is really hard, and I think before it becomes a big shift, the medical community and families and patients have to be educated, and more data is needed to show that indeed it is safe for anaphylaxis, and it works just as well, if not better,” she said.
Lowenthal, CEO of ARS, said the company is actively working on additional studies to get more data. Headquartered in Sorrento Valley, ARS grew its headcount in the last month from a staff of 60 fulltime employees to about 150, with many of the new positions in the sales department to support Neffy.
The company was founded in 2015 by Lowenthal, Robert Bell and Sarina Tanimoto. ARS entered the public market in 2022 through a reverse merger with another local biotech company, Silverback Therapeutics.
As Neffy rolls out over the next year, ARS Pharma is also working on getting the medicine approved in Europe and other countries that have expressed interest.
The company is also developing a Neffy dosage for young children and babies over the next couple of years. The current product is only available for adults and children who weigh at least 66 pounds.
Epinephrine is the only drug that has been approved by the FDA to treat anaphylaxis. But it has been about 30 years since a new epinephrine treatment, the auto-injector, became available to patients.
That’s why patients like Bauder – who has created an online community to share her experience of living with allergies – are heartened by the attention being put on this condition. She hasn’t tried Neffy yet, but the mere concept of not having an allergic reaction derail a celebration or cause a scene at dinner with friends is exciting to say the least.
“Something like Neffy has the ability to make the anaphylactic reaction feel less traumatic because we feel like we have more autonomy,” she said. “You have this safety net in your back pocket, but in a sense that it feels so much more accessible.”
K.C. Alfred / The San Diego Union-Tribune / TNS
