IntraBio, Inc.

Novartis used one of its priority review vouchers (PRV) to speed the September approval of Kisqali in early breast cancer, the FDA said Monday. The FDA did not disclose where this PRV came to Novartis from, but the Swiss biotech has publicly disclosed an industry-leading seven PRVs , three of which were acquired on the open market for between $21 million and $130 million each. The use of a PRV sped up the supplemental approval by about four months. Novartis noted that this latest nod for Kisqali roughly doubles the treatable adjuvant population and includes patients whose cancer has not yet spread to their lymph nodes. Novartis did not immediately respond to a request for comment. The FDA on Monday also said it awarded two other PRVs for the approvals of two drugs to treat the rare condition Niemann-Pick disease type C. Those PRVs went to IntraBio’s Aqneursa and Zevra Therapeutics’ Miplyffa , both of which won approval late last month. The flurry of activity around the rare pediatric PRV program comes as Congress has only passed a short-term reauthorization of the program, running up to Dec. 20. The program still needs to be reauthorized for a longer extension or else it will sunset. The House in late September unanimously passed a wide-ranging bill via voice vote that will reauthorize the rare pediatric PRV program through 2029. To get it across the finish line, however, the Senate and the Biden administration will have to act in the so-called “lame-duck” period, which will occur after the election and before the next incoming administration takes over. The FDA, meanwhile, told industry recently that if it’s unable to grant a request for a rare pediatric disease designation by Dec. 20, “a marketing application for that drug cannot be eligible for a voucher under the current provisions of the law.”

“Our intent was basically to hit the ground running on the day of approval and make the medicine available on approval—and we’ve been able to accomplish that,” Karl Odquist, IntraBio's head of trade and commercial operations, told Fierce Pharma. With its first approval in hand, privately held IntraBio is embarking upon the difficult task of marketing a drug for an ultra-rare disease. Still, with months of prep work and outreach under its belt, the company is already proving it can hit the ground running.IntraBio last week scored an FDA nod for its drug levacetylleucine, now known as Aqneursa, as the second therapy to treat neurological symptoms of the lysosomal storage disorder Niemann-Pick disease type C (NPC).IntraBio’s green light came right after Zevra Therapeutics scored a thumbs up for its own NPC treatment Miplyffa. Zevra has said it plans to launch its drug in a matter of months, whereas IntraBio’s Aqneursa was made available immediately upon approval. Prior to the twin approvals, there had been “a lot of hope in the pipeline,” but also “a lot of setbacks” and “near misses” for patients living with NPC, Taylor Fields, chief development officer and president of product development at IntraBio, said in an interview.That instilled a sense of urgency in the company, which has endeavored to make it as easy as possible for the NPC community to access Aqneursa following the med’s approval, she said.“We wanted to make sure that we understood the urgency, we could be there immediately for [the community] and focus all of our efforts into ensuring—as soon as we were able to provide them with what they were looking for—that we did that,” Fields said.Given the size of the NPC community, “you can get to know the vast majority of the patients and the [healthcare professionals] by name,” IntraBio’s chief operating officer, Mark Lubkeman, added during the interview. He noted that Aqneursa’s launch is more about education than it is a strict marketing push. Since Aqneursa’s approval and launch last week, IntraBio’s field medical directors have been “highly active” with those HCPs to help them understand IntraBio’s drug and the benefits it can have for patients, Lubkeman said.“We’re really about getting the science out there and the benefits of the science to this community,” he said. NPC is an inborn metabolic disorder in which the lysosome has problems clearing cholesterol and other lipids in cells, subsequently hamstringing the ability of mitochondria to produce cellular energy, Fields explained.Aqneursa combats this issue by crossing the blood brain barrier, entering cells in high doses and normalizing energy production, she said, adding that IntraBio’s drug has also been shown to dampen neuroinflammation, in turn helping to prevent secondary cell death.To smooth access to Aqneursa, IntraBio has rolled out an assistance program dubbed Aqneursa Cares for both patients and doctors that includes financial support, third-party resources and educational materials.On the financial side, IntraBio’s head of trade and commercial operations, Karl Odquist, pointed to the slow uptake of payer coverage for new drugs that can hamper early access to treatment.“Anything we can do to try to get the drug to [patients] as soon as possible … while we work out all the kinks and bugs with the payer is what we’re going to do,” he said.IntraBio has developed a copay offset program to help with patient costs, Odquist noted, adding that, “in many instances, our patients can pay as little as zero out of pocket for their copay.” The company has also set up a “bridge program” to help temporarily provide NPC patients and their families with Aqneursa in the event they have to switch insurance, Odquist said.IntraBio has yet to reveal Aqneursa’s price tag, though the medicine’s cost is expected to become public this week and ought to be “very compelling,” according to Lubkeman, the COO. As for education, IntraBio has engaged a dedicated support staff that can help new patients get onboarded and better understand how Aqneursa fits into their disease. Meanwhile, the drugmaker is also “partnering actively” with the National Niemann Pick Disease Foundation and, as of Monday, has already hosted two webinars “at the patient level,” Lubkeman said.Further, IntraBio will “maintain an ongoing dialogue with the NPC community in partnership with patient advocacy groups on social media,” he said, with the assurance that the company will be cautious and compliant in how it engages with the rare disease community. Overall, IntraBio has been prepping its Aqneursa rollout for “many months,” building out its field medical capabilities, payer teams, clinical account manager forces and more, Odquist said.“Our intent was basically to hit the ground running on the day of approval and make the medicine available on approval—and we’ve been able to accomplish that,” he said, noting that IntraBio is already receiving prescriptions just days after Aqneursa’s FDA green light.As a private company, IntraBio is not able to divulge specific numbers around its field support teams. That said, according to Lubjeman, it has “fielded a very focused team of clinical account managers, business operations specialists and field medical directors appropriate to a rare disease market.”

IntraBio’s green light likely comes as a blow to Zevra Therapeutics, which snagged a historic approval for Miplyffa in Niemann-Pick disease type C on Friday. While it was just last week that Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for the lysosomal storage disorder Niemann-Pick disease type C (NPC), IntraBio has already one-upped its rare disease rival.Late Tuesday, the FDA approved IntraBio’s levacetylleucine under the brand name Aqneursa to treat neurological symptoms of NPC in adults and kids weighing at least 15 kg (about 33 pounds).Aqneursa has been approved as a standalone therapy and is available now, according to a release from IntraBio—which hails from the U.K. but recently shifted its headquarters to Austin, Texas, following a $40 million funding round.The green light likely comes as a blow to Zevra, which snagged a historic approval for Miplyffa in NPC on Friday. But, unlike IntraBio’s monotherapy, Miplyffa must be taken with the enzyme inhibitor miglustat. What’s more, Miplyffa isn’t expected to hit the market for another eight to 12 weeks, according to Zevra.In turn, IntraBio’s approval and launch plan have effectively erased Zevra’s head start. NPC, a rare lysosomal storage disorder, prevents the body from moving cholesterol and other lipids in cells. This leads to a buildup of cholesterol and other fats in the liver, spleen or lungs that can cause organ damage. On average, people with NPC only live for about 13 years, the FDA noted in its release.The disease can also impair patients’ speech, cognition, swallowing, movement and fine motor skills.The FDA based its approval of Aqneursa on results from IntraBio’s pivotal trial IB1001-301, which looked at the drug’s effect on neurological symptoms and functioning in adults and kids over the age of 4 with a confirmed NPC diagnosis.The trial met its primary efficacy endpoint and every secondary endpoint across all patient cohorts receiving Aqneursa, IntraBio said. The study found that Aqneursa “significantly” improved neurological signs and symptoms of the disease and led to functional benefits within 12 weeks.The main outcome the FDA looked at in the study was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA)—known as the functional SARA (fSARA)—which measures gait, stability, speech and upper and lower limb coordination across eight individual domains. The fSARA, meanwhile, only assesses gait, sitting, stance and speech disturbance domains with modifications to the scoring responses.Patients on Aqneursa showed greater improvement on fSARA scores versus those on placebo, and the results on the modified assessment were buoyed by consistent outcomes demonstrated in the original test, IntraBio explained.While the most common side effects associated with Aqneursa are abdominal pain, difficult swallowing, upper respiratory tract infection and vomiting, the drug’s prescribing information does carry a warning that Aqneursa may cause embryo-fetal harm if used during pregnancy.Patients should inform their doctors of a known or suspected pregnancy before taking IntraBio’s drug, the FDA noted.Aqneursa is taken orally up to three times a day, with or without food. The dosing varies depending upon the individual patient’s body weight.Aqneursa marks IntraBio’s first commercial product and, with an approval in hand, the company isn’t content to stop at NPC.“Based on our clinical research, we believe that Aqneursa may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Aqneursa for these additional indications,” Mallory Factor, CEO of IntraBio, said in a statement. In order to help patients access the treatment, IntraBio has launched a support program dubbed Aqneursa Cares. The program will include financial support to “reduce or eliminate” out-of-pocket costs for qualifying patients as well as connect them with third-party resources. The program will also feature educational resources and a team of disease specialists.