One-year Results in 2 Given Gene Therapy at Low Dose Showing Promise, Axovant Reports

early trial results

Two Parkinson’s patients treated with AXO-Lenti-PD, an investigative gene therapy, in an ongoing clinical trial continue to show improvement 12 months later, Axovant, the therapy’s developer, said in a release.

These findings at one year after treatment are important because this timepoint allows for a better assessment of therapy durability, and a more assured differentiation between placebo effects and therapeutic response, the company added.

AXO-Lenti-PD has shown encouraging results in these two people given a first low dose in the SUNRISE-PD (NCT03720418) Phase 1/2 clinical trial, which is now enrolling up to 30 patients at sites in France and England.

The treatment works by delivering three genes involved in dopamine production directly to the brain via a surgical procedure.

Dopamine is a neurotransmitter — a molecule involved in transmitting information between neurons — that is critical to coordinating movement. Dopamine-producing (dopaminergic) neurons are lost in Parkinson’s, and the resulting drop in dopamine levels is the cause of many disease symptoms.

By ‘infecting’ brain cells with the genetic instructions to increase dopamine production, AXO-Lenti-PD aims to turn other cells into dopaminergic neurons.

Current dopamine replacement therapies require continual oral doses of dopamine, whose effectiveness fades over time. The period between when one dose’s effectiveness wanes and the taking of a next dose can result in “off periods,” wherein patients report a return of symptoms such as poor motor control, stiffness, fatigue and mood changes.

Helping the brain to again produce adequate levels of dopamine would, in theory, eliminate the need for periodic oral doses, which could significantly limit off periods.

Previous studies in primate models of Parkinson’s found AXO-Lenti-PD to be safe and effective, and SUNRISE-PD results at three months’ post-treatment found that a one-time delivery of the therapy significantly improved patient scores on the Unified Parkinson’s Disease Rating Scale (UPDRS), a standard assessment of motor and non-motor symptoms associated with Parkinson’s.

The trial consists of two parts. Part A is an open-label, dose-escalation phase in which patients receive one of potentially three escalating doses of the gene therapy. In part B, a new group of patients will be randomized to either the ideal part A dose or to a sham procedure as an untreated control group. SUNRISE-PD’s goal is to test the safety, tolerability, and effectiveness of the potential treatment.

Both patients here, the first two enrolled, received the lowest dose (4.2×106 transducing units) of AXO-Lenti-PD.

One-year results show positive changes of 24 points and 20 points (respectively for the two patients) on the UPDRS Part III “Off” score, representing a 37% improvement in off-period motor symptoms, Axovant reported. Improvement at six months was 29%, as measured on the same scale.

These patients also showed an average 13-point positive change from baseline (study start) — representing a 44% improvement — on the UPDRS Part II “Off” score, which assesses daily life activities. On the PDQ-39 score index, another quality-of-life measure in Parkinson’s disease, these two showed an average 15-point positive change, or a 30% improvement from baseline to 12 months.

Both patients tolerated AXO-Lenti-PD well, and neither reported any serious side effects. One maintained a diary of on/off periods, which is useful in evaluating changes that might be due to therapy across time.

People being enrolled in SUNRISE-PD have had Parkinson’s for at least five years, have motor fluctuations and dyskinesia (jerky, involuntary movements), and are between the ages of 48 and 70.  More information can be found here.

The company expects to soon release six-month results on the first two patients given a second and higher dose of AXO-Lenti-PD. This dose is three times higher than that given the first cohort.

If dose-escalation results allow, Axovant expects to begin the randomized and placebo-controlled part B of the SUNRISE-PD as a Phase 2 study by the close of 2020.

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Acorda Launches Online Tool to Help Parkinson’s Patients Discuss ‘Off Episodes’

sex-related differences

To help Parkinson’s patients identify and discuss their “off episodes” — the return of disease symptoms — with healthcare providers, Acorda Therapeutics is offering a new online tool.

Called the “Do Tell” Your Doctor Tool, the questionnaire is used to record such episodes, which can be hard to identify because these episodic symptoms and their frequency vary for each patient. Lack of awareness makes it difficult for patients to talk about off periods with medical professionals, and family and friends.

The questionnaire provides a visual guide and glossary to help patients recognize and document symptoms. It’s hosted by Live Well. Do Tell., an Acorda initiative that aims to address barriers that Parkinson’s patients and their caregivers face in communicating motor and non-motor symptoms.

Patients generally have a hard time explaining their off periods during doctor’s visits, resulting in missed opportunities for discussion, said Ron Cohen, Acorda’s president and CEO.

“We expect the ‘Do Tell’ Your Doctor Tool to increase effective communication between [people with Parkinson’s] and their healthcare providers, thereby enhancing their ability to optimize therapeutic outcomes,” said Cohen, MD, in a news release.

Parkinson’s is characterized by the lack of a brain signaling molecule called dopamine, which leads to tremors, stiffness, and slowness. Theoretically, replacing the missing dopamine would be an ideal way to treat the disease. But the molecule is too big to cross the blood-brain barrier, so a dopamine precursor called levodopa is used instead. When levodopa becomes dopamine inside the brain, Parkinson’s motor problems subside.

However, long-term use causes these beneficial effects to wear off before a new levodopa dose can be taken. That’s when symptoms re-emerge. Such off episodes can be offset to a certain degree by increasing either the levodopa dosage or the dosing frequency but doing so may cause dyskinesia — uncontrolled and abnormal movements. Other agents, including carbidopa, in combination with levodopa can reduce or help manage off episodes.

As for the online tool, it was adapted from a clinically validated questionnaire developed by Duke University with help from a multidisciplinary steering committee of Parkinson’s disease community leaders. It also underwent testing at InMotion, an Ohio wellness center that offers free evidence-based exercise and education programs and other support to Parkinson’s patients and their caregivers.

Using the tool, patients identify and rank symptoms. Their answers are then used to produce a tailored “word cloud” that delineates the symptoms and how troublesome they are at a glance.

“This tool will be a valuable resource to help improve conversations between people with Parkinson’s, their care partners and healthcare providers,” said Karen Jaffe, who has Parkinson’s and is a member of the Live Well. Do Tell. steering committee and the InMotion board. “We were excited to offer our input on this important tool to ensure it is user-friendly and serves the needs of our community.”

About one million U.S. residents, and 10 million people globally live with Parkinson’s disease. Some 40% of U.S. patients experience off periods within five years of starting treatment.

Acorda Therapeutics is the developer of Inbrija (CVT-301), an inhaled formulation of levodopa.

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Nourianz Now Available in US as Add-on to Carbidopa/Levodopa to Treat Parkinson’s Off Periods

Nourianz tablets

Kyowa Kirin’s Nourianz (istradefylline) tablets are now available in the United States as an add-on treatment for off periods in Parkinson’s disease patients on a carbidopa/levodopa regimen.

Off periods — when the effects of a medication wear off before a new dose can be taken — are characterized by the re-emergence of Parkinson’s motor symptoms and are typically more common as the disease progresses. Within five years of starting levodopa/carbidopa therapy, approximately 50% of patients may experience off periods.

“We are pleased to offer patients Nourianz, the first and only FDA-approved adenosine A2Areceptor antagonist treatment for ‘off’ time associated with [Parkinson’s],” Tom Stratford, president of Kyowa Kirin USA Holdings, said in a press release. “Nourianz administered with levodopa/carbidopa therapy can help reduce ‘off’ time and increase ‘on’ time without troublesome dyskinesia.”

Nourianz blocks a receptor, known as the adenosine A2A receptor, found at high levels in the basal ganglia, a region of the brain that controls movement. By blocking this receptor, Nourianz can alter the release of neurotransmitters — chemical substances produced in response to nerve signals that allow nerve cells to communicate — in the basal ganglia, regulating motor activity.

The U.S Food and Drug Administration (FDA) approved Nourianz in August based on the results of four randomized, placebo-controlled Phase 2 and 3 clinical trials (NCT00955526, NCT00455507, NCT01968031, and NCT00250393).

The trials assessed the safety and efficacy of two doses (20 mg and 40 mg) of Nourianz to reduce the mean total hours of awake time per day spent in the off state and also lessen motor symptoms.

A total of 1,143 Parkinson’s patients taking levodopa/carbidopa, levodopa/benserazide, or levodopa and any other dopa-decarboxylase inhibitor were recruited. Treatment with Nourianz significantly decreased daily off time, compared with patients on a placebo, and improved motor function.

The most common side effects of Nourianz included involuntary muscle movement (dyskinesia), dizziness, constipation, nausea, hallucinations, and insomnia.

“In my clinical practice, I see patients who experience the troublesome effects of Parkinson’s disease and ‘off’ episodes that interfere with activities of daily living,” said Peter A. LeWitt, MD, a professor of neurology at Wayne State University School of Medicine and director of the Parkinson’s Disease and Movement Disorders Program, Henry Ford Hospital.

“Nourianz represents an important milestone and provides U.S. patients and their caregivers with a nondopaminergic, once-a-day oral treatment option to significantly decrease the amount of ‘off’ time,” LeWitt added.

Nourianz has been marketed in Japan under the brand name Nouriast since May 2013.

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Accordion Pill Enables Higher Optimal Doses of Levodopa Than Sinemet, Data Show

Accordion Pill

Patients with Parkinson’s disease who were treated with Accordion Pill Carbidopa/Levodopa (AP-CD/LD) tolerated a higher dose of levodopa and experienced less variability in plasma levels of this standard therapy than those on Sinemet.

Those are the top-line results from a Phase 3 clinical trial and data from a pharmacological study.

The effectiveness of levodopa may wear off with chronic treatment, resulting in the reappearance of motor complications, known as “off” periods. As this is associated with levodopa’s limited absorption in the upper gastrointestinal tract, Intec Pharma developed AP-CD/LD, which includes a gastric retention and release system to enable both immediate and controlled release. This controlled release mode prolongs the discharge of the medication into the stomach to eight to 12 hours, which may improve absorption.

The double-blind ACCORDANCE study (NCT02605434) is comparing AP-CD/LD with Merck’s Sinemet, an immediate release formulation of CD/LD. A total of 320 patients with advanced Parkinson’s were included in the double-blind part of the study (65.6% men, average disease duration 8.7 years), with the final visit occurring last May.

All eligible participants were on a daily levodopa dose within 400 and 1,300 mg and experienced at least 2.5 hours of off periods. After study completion, the patients could join a 12-month open-label safety extension study of AP‑CD/LD.

After two periods of six weeks each to stabilize and optimize patients on Sinemet and then on AP-CD/LD, the patients were assigned randomly to either approach over 13 weeks, with a two-week follow-up.

Two AP-CD/LD doses were tested: 50 mg of carbidopa, with 400 or 500 mg of levodopa, two or three times daily. Similar to the baseline percentage of daily off time, the patients’ mean age did not differ significantly between the two groups: 62.8 years in the AP-CD/LD group and 64.9 years in patients receiving IR-CD/LD.

At the recent  International Congress of Parkinson’s and Movement Disorder Society (MDS 2019),  R. Michael Gendreau, MD, PhD, Intec’s chief medical officer, presented the scientific poster “Patients Experiencing Motor Fluctuations with Parkinson’s Disease: Participant Characteristics in the ACCORDANCE Phase 3 Efficacy and Safety Trial of Accordion Pill-Carbidopa/Levodopa.” The presentation showed that patients on AP-CD/LD tolerated higher daily doses of levodopa than those taking Sinemet.

Specifically, 86.2% of patients taking AP-CD/LD achieved an optimal levodopa dose of 1,200 mg or greater, compared to only 19.7% among those on Sinemet.

Previous results from ACCORDANCE showed that, compared with Sinemet, AP-CD/LD did not provide greater reduction in daily off periods, benefits in “on” time without troublesome dyskinesia (involuntary body movements), or improved motor function scores, as assessed with the  Unified Parkinson’s Disease Rating Scale.

In a press release, Gendreau said that this lack of significant benefits of AP-CD/LD may have been due to confounding effects from patients whose dose was increased to the maximum (50/500 mg).

An analysis of lower doses showed a greater difference in mean daily off time between AP-CD/LD and IR-CD/LD in participants who were not dose-limited during the Accordion Pill titration process.

“This suggests that for many participants, AP doses higher than those available in this study may have been necessary to achieve optimal efficacy,” said Gendreau.

Also at MDS 2019, Jeffrey A. Meckler, Intec’s vice chairman and CEO, presented the study, “Pharmacokinetics of Accordion Pill-Carbidopa/Levodopa Following Multiple Doses in Patients with Parkinson’s Disease.” That presentation showed a pharmacokinetic (PK) comparison of AP-CD/LD 50/500 mg three times per day and the immediate release form (37.5/150 mg) five times daily in 12 patients. (Of note, PK refers to how a compound is absorbed, distributed, metabolized and eliminated by the body.)

After treatment with the immediate release form on day 1, the patients were instructed to take AP-CD/LD until day 8, when they returned to the clinic in the off state and received AP-CD/LD three times over 10 hours.

In line with Phase 2 findings — which also showed reduced motor fluctuations — preliminary results showed that AP-CD/LD provided less variability in levodopa’s plasma levels. Overall, as variable plasma levodopa levels have been associated with motor complications, the findings “suggest that treatment with AP-CD/LD may reduce motor complications,” the team wrote. AP-CD/LD was well-tolerated and no serious adverse side effects were reported.

“We believe the data underscored the potential of AP-CD/LD in PD while highlighting its long-term safety data,” said Meckler. “We have initiated a formal process for partnering AP-CD/LD in PD and this enhanced exposure will be important as we seek to partner AP-CD/LD for continued late-stage clinical development and commercialization in [Parkinson’s] patients.”

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h-Patch Device for Slow Apomorphine Infusion May Ease Off Periods in Advanced Parkinson’s, Valeritas Says

apomorphine treatment

Under-the-skin infusions of low-dose apomorphine using a wearable device called h-Patch may help in avoiding off periods and improving motor function in Parkinson’s patients, Valeritas, the technology’s developer, reports.

A preclinical study found that apomorphine delivered using h-Patch could be detected in the blood within two hours, with a gradual decline upon completing the 24-hour subcutaneous infusion. Valeritas is planning to present these results at an upcoming medical conference.

Apomorphine is a derivative of morphine with effects similar to dopamine, the neurotransmitter released by specialized neurons that are progressively lost in Parkinson patients. In patients with advanced disease, apomorphine is the only approved option for the acute treatment of off episodes, or periods when the medication wears off and before a new dose can be taken. Off periods are characterized by the resurgence of motor and non-motor symptoms.

It is available in the U.S. as an under-the-skin injection (Apokyn, by US WorldMeds), but has been associated with pain and injection-site reactions.

In prior clinical trials, single-dose, subcutaneous infusions of apomorphine have shown efficacy in suppressing off periods, reducing dyskinesia — involuntary, jerky movements — and improving motor scores of Parkinson’s patients. They also enabled significantly lesser use of levodopa, which has been associated with reduced effectiveness and with impulsive and compulsive behaviors when used for a number of years.

Infusion pumps in current use can be bulky, “requiring delivery of relatively large volumes of therapeutics [to] remain a barrier” to both patients and caregivers, Valeritas states a press release.

“This study highlights a new subcutaneous delivery of an old drug (apomorphine) which is commonly used in Parkinson’s,” said Santosh Kesari, chair and professor of Translational Neurosciences and Neurotherapeutics at the John Wayne Cancer Institute and Pacific Neuroscience Institute, both in California.

The h-Patch approach, Kesari added, “may offer a more consistent dose throughout the day” relative to oral dosing of apomorphine, and help improve functionality and quality of life. “Drug delivery for CNS disorders is still a significant barrier for optimizing new and old drugs,” he said.

Valeritas’ V-Go Wearable Insulin Delivery device for people with type 2 diabetes, which also uses the h-Patch technology, is marketed in the U.S. with the approval of the U.S. Food and Drug Administration.

“Valeritas continues to demonstrate the versatility of, and opportunities for its h-Patch technology beyond insulin delivery,” said John Timberlake, the company’s president and CEO.

“The h-Patch is a demonstrated patient-friendly, cost-effective, and powerful delivery method for a variety of therapeutics which we believe is ideal for subcutaneous delivery of apomorphine,” he added.

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CHMP Favors EU Approval of Inbrija to Treat Parkinson’s Off Periods

CHMP recommendation

The Committee for Medicinal Products for Human Use (CHMP) — an arm of the European Medicines Agency (EMA) — is recommending that Inbrija, by Acorda Therapeutics, be approved to treat off periods in Parkinson’s patients on a carbidopa/levodopa regimen.

The European Commission will review CHMP’s positive opinion, with a decision expected before year’s end.

“We are delighted that Inbrija has achieved this important milestone, and look forward to the EC’s final decision later this year. There are approximately 1.2 million people in the EU living with Parkinson’s. We estimate that 40% of these individuals experience off periods, which are considered extremely disruptive,” Ron Cohen, MD, president and CEO of Acorda, said in a press release.

Inbrija is a self-administered, inhaled formulation of levodopa developed to treat off episodes. These periods, when levopoda wears off before a new dose can be taken, are characterized by the re-emergence of Parkinson’s motor symptoms, and are typically more common as the disease progresses.

Inbrija delivers a precise dose of levodopa to patients’ brains that, because it is inhaled, bypasses the need to be absorbed in the digestive system. As such, it can deliver a higher and more consistent levodopa dose to the brain.

Inbrija is approved by the U.S. Food and Drug Administration (FDA) to treat Parkinson’s symptoms during off episodes. 

CHMP’s decision was based on clinical data from the Phase 3 SPAN-PD clinical trial (NCT02240030). The study evaluated the safety and effectiveness of two doses of Inbrija — 84 mg and 60 mg — or a placebo taken up to five times a day in 351 Parkinson’s patients experiencing off periods. Treatment was maintained for 12 weeks.

Results showed that Inbrija significantly improved patients’ motor function compared to placebo. Researchers found no safety concerns. The most common adverse events were cough, upper respiratory tract infection, and throat irritation.

Trial outcomes were published in The Lancet Neurology, in the study “Safety and efficacy of CVT-301 (levodopa inhalation powder) on motor function during off periods in patients with Parkinson’s disease: a randomised, double-blind, placebo-controlled phase 3 trial.”

Inbrija was also studied in two long-term Phase 3 trials — the CVT-301-005 trial (NCT02352363) and the CVT-301-004E study (NCT02242487) – that evaluated Inbrija used up to five times daily for 12 months.

Researchers found no changes in treated patients’ lung function compared with standard levodopa treatment, supporting Inbrija’s safety as an off-period medication.

Efficacy data from the CVT-301-005 trial also confirmed that Inbrija eases problems with motor function that emerge in off periods. Treatment was found to enable symptom control within 60 minutes of the dose and lowered the length of patients’ off times.

Acorda’s European application covers all European Union countries, as well as Norway, Liechtenstein, and Iceland.

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‘Framing OFF Through Art’ Exhibit on View at World Parkinson Congress

Artworks focus on off periods

More than 1 in every 3 — some 40 percent — of Parkinson’s patients have “off periods,” the return of motor and non-motor symptoms as the effectiveness of a  treatment’s dose wears off. Artworks created in collaboration with patients, part of an Acorda Therapeutics program, are helping to express what these periods are like and focus attention on them.

Called Framing OFF Through Art, the six-piece exhibition was recently featured at the 5th World Parkinson Congress in Kyoto, Japan, attended by more than 3,000 people from at least 60 countries. The initiative is the centerpiece of Acorda’s Live Well. Do Tell program, launched last year.

Off periods are characterized by the re-emergence of Parkinson’s symptoms, such as sadness and anxiety, when the effects of levodopa/carbidopa wear off. Such episodes are typically more common as the disease progresses, and vary from person to person.

Because off periods can be difficult to discuss or even identify, the artwork is intended to visualize feelings associated with them. The hope is that the art will spur other patients to recognize their “off” symptoms, and discuss them with their care team. The artists who created the pieces have been touched in some way by Parkinson’s.

”Research has consistently shown that off periods are among the most common issues for people living with Parkinson’s disease,” Ron Cohen, MD, Acorda president and chief executive officer, said in a news release.

“We believe that people with Parkinson’s will be able to see aspects of their own experiences with Parkinson’s and off periods in these works of art.”

The artwork ranges from acrylic sculptures to oil paintings. Each artist was paired with two patients and their caregivers. The exhibition debuted in October at a New York event where the artists, patients and caregivers were guests. The need to better understand off periods, as well as how to discuss them, is highlighted in the Live Well. Do Tell statement of need.

As part of a rotating series, a piece called “Rooted Resilience” is featured on the Live Well. Do Tell website. Created by abstract impressionist and colorist Tim Kinney, who has several friends with Parkinson’s, the multi-color painting of a tree depicts the way Gustavo Pavon, diagnosed in 2006, described his off periods.

The painting’s transition from dark roots to the brightness of the leaves and sky is meant to illustrate emergence from an off time.

Marcela Del Bosque, Pavon’s wife and caregiver, is mentioned in a narrative accompanying the painting as describing the transition in this way:

“When Marcela saw the painting, she immediately picked up on this and noted that when Gustavo is in a dark place — in an off period — he’s quiet and doesn’t smile. Then, Gustavo’s bright smile that she loves reappears and he returns.”

The work’s branches and colors also symbolize Pavon’s growth — his “branching out” —  to embrace community support and speak more openly about his experience. For nearly a decade, Pavon had only told a few people about his condition and off symptoms that include shivering and stiffness.

Go here to learn more about off periods and for additional resources.

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Onstryv Now Approved for Parkinson’s Patients in Canada

Onstryv approval Canada

Onstryv (safinamide) has been approved for the treatment of Parkinson’s disease in Canada, where roughly 100,000 individuals live with the disorder.

The announcement was made by Quebec-based Valeo Pharma and Italian pharmaceutical conglomerate Zambon, the commercialization partner of Newron Pharmaceuticals, a biopharmaceutical company focused on the development of therapies for diseases of the central and peripheral nervous system.

The agreement with Zambon calls for Valeo Pharma, a specialty pharmaceutical company dedicated to commercializing innovative prescription products, to be responsible for all regulatory requirements, quality, sales and marketing, and drug distribution. Onstryv is expected to be available in Canada by June.

”The approval of safinamide in Canada is a step forward for patients who need new treatment options for Parkinson’s disease,” Roberto Tascione, Zambon’s CEO, said in the press release. “Our mission is to make this medication available to as many [Parkinson’s disease] patients worldwide as possible.”

Known as Onstryv in Canada and Xadago in the rest of the world, this compound is an oral, once-a-day, add-on therapy developed by Newron, and approved in the United States in March 2017 by the U.S. Food and Drug Administration to improve motor function in Parkinson’s patients who experience “off periods” while on treatment with levodopa and/or Lodosyn (carbidopa).

While partially effective, therapy using either or both levodopa and carbidopa results in debilitating fluctuations between a state of normal motor function (known as “on episodes”) and reduced motor function (“off periods”) as the treatment’s effectiveness wears off. What’s more, the increased doses necessary as the disease progresses frequently cause uncontrolled involuntary movements, a condition known as dyskinesia.

Onstryv raises the level and function of dopamine in the brain, both through the reversible blockage of the enzyme monoamine oxidase B that normally breaks down this chemical, and by inhibiting transporters responsible for its absorption and retention. In addition, the medicine inhibits the excessive release of the signaling molecule glutamate.

Following four randomized, double-blind, placebo-controlled Phase 3 trials, published results indicated that the addition of Onstryv increased the frequency of on episodes, decreased off periods, and improved motor function in levodopa-treated patients.

In 2017, Valeo and Zambon announced a partnership granting Valeo exclusive Canadian rights to commercialize Onstryv. In addition to Canada and the U.S., the medicine is approved in the European Union and Switzerland and was recently approved in Australia.

“There is a growing need for new treatments to manage Parkinson’s disease, and Onstryv provides an important option for patients that require better control of their symptoms,” said Steve Saviuk, Valeo’s CEO. “We look  forward to launching the first new oral treatment for Parkinson’s disease in over a decade to Canadian patients in need of a new therapeutic choice.”

In recent related news, a review study found that, along with cannabinoids and opioids, Onstryv may relieve Parkinson’s patients’ pain, a frequent non-motor symptom.

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Inbrija Approved in US to Treat Off Periods in Parkinson’s Patients on Carbidopa/Levodopa


The U.S. Food and Drug Administration has approved Inbrija (levodopa inhalation powder)‎ for the treatment of Parkinson’s off periods in patients on a carbidopa/levodopa regimen.

Acorda Therapeutics’ therapy is expected to be available by prescription in the first quarter of 2019. It will be distributed through a network of specialty pharmacies in the U.S.

“Today’s approval of INBRIJA marks a major milestone for both Acorda and the Parkinson’s community, for whom we are gratified to have developed this much needed therapy,” Ron Cohen, MD, Acorda’s president and CEO, said in a press release. Cohen noted the more than “two decades of research and development” needed for this approval, as well as the “enormous perseverance and ingenuity” by Acorda’s team.

Inbrija is a self-administered, orally inhaled levodopa treatment for off periods, which are characterized by the re-emergence of Parkinson’s motor symptoms due to low levels of dopamine between doses of standard treatment. These off episodes are typically more common as the disease progresses.

The Michael J. Fox Foundation helped to fund the early development of Acorda’s medication, a decision based on the impact off periods have on patients’ lives, according to Todd Sherer, PhD, the foundation’s CEO.

“We knew we had to help address this unmet need, and this approval is a significant step forward for the community as it provides a new option to manage these gaps in symptom control,” he said.

Inbrija uses Acorda’s ARCUS technology, a system designed to deliver medication to the lungs through inhalation. ARCUS transforms molecules into a light, porous, dry powder, enabling the delivery of much higher doses of medication.

The FDA’s decision was based on a clinical program that included approximately 900 Parkinson’s patients. The pivotal Phase 3 SPAN-PD trial (NCT02240030) evaluated the efficacy and safety of 84 mg and 60 mg of Inbrija in 351 participants with mild to moderate Parkinson’s who were experiencing off periods.

Results of the double-blind study showed a statistically significant improvement in motor function at the final 12-week visit, seen by a reduction in the Unified Parkinson’s Disease Rating Scale Part III (UPDRS-III) score with Inbrija’s higher dose, compared with placebo, at 30 minutes post-dose. Inbrija’s effects were seen as early as 10 minutes after treatment.

The most common adverse reactions with Inbrija were cough, upper respiratory tract infection, nausea, and discolored sputum, which may indicate a bacterial infection.

Treatment with Inbrija was also analyzed in a one-year, randomized, open-label Phase 3 trial with 398 participants (NCT02352363). Results showed a similar average reduction in forced expiratory volume in one second — a measure of pulmonary function — in the Inbrija (84 mg) and observational groups.

Inbrija also eased Parkinson’s symptoms at all time points, as measured with the UPDRS-III scale, enabled symptom control within 60 minutes of the dose and reduction of total daily off times, and led to improved Patient Global Impression of Change scores in 75% of patients, which reflects the patients’ assessment of treatment effectiveness.

“In the clinical study program, Inbrija established its safety profile and demonstrated clinically meaningful improvements in motor function,” said Robert A. Hauser, MD, a professor of neurology and director of the Parkinson’s Disease and Movement Disorders Center at the University of South Florida. “Inbrija helps address a significant unmet need for people with Parkinson’s, and we look forward to adding this new treatment option to our armamentarium.”

Inbrija is not to be used by patients currently on or treated with a nonselective monoamine oxidase inhibitor, such as the antidepressants and anxiolytics phenelzine or tranylcypromine, within the last two weeks.

The company is currently seeking EU approval for Inbrija.

Burkhard Blank, MD, Acorda’s chief medical officer, said he was “delighted” with Inbrija’s approval and its upcoming availability for on-demand use, based on each patient’s needs.

“We thank the FDA for a constructive dialogue throughout the development program and their partnership during the review cycle. We especially thank all those who volunteered for the Inbrija clinical trials, without whose commitment new medications could not be developed,” he said.

He further noted the important role played by “people living with Parkinson’s, their care partners, researchers, clinicians and advocacy groups,” to achieve FDA approval.

A webcast of a recent conference call hosted by Acorda is available here.

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New Art Initiative Aims to Improve Understanding and Discussion of Parkinson’s Off Periods

art initiative, off periods

A new initiative by Acorda Therapeutics uses art to help people with Parkinson’s disease recognize and communicate about their off-period experiences.

The initiative, called Framing OFF Through Art, builds on Acorda’s Live Well. Do Tell program, launched earlier this year.

Framing OFF Through Art includes artwork inspired by the experiences of patients with Parkinson’s and their caregivers. The artists who created the pieces also have personal connections to the disease. Each artist partnered with two patients and their caregivers. The art aims to educate and encourage others with Parkinson’s and their care partners to identify off-period symptoms and discuss them with their healthcare professionals.

“Art is a powerful way to engage a community, and in Framing OFF Through Art, our goal is to create an emotional connection that results in better dialogues about Parkinson’s symptoms,” Ron Cohen, MD, Acorda’s president and CEO, said in a press release.

The artwork debuted on Oct. 23 at an event in New York, where the artists, patients, and their caregivers were guests. The first piece was created by Julie B., whose mom is living with the disease, and is available on the Live Well. Do Tell website. More art and personal stories will be revealed each month.

“I know that some of these symptoms are really difficult to put into words. A visual representation can really communicate what you are going through,” Julie B. said in a video interview. “One of the big takeaways from this initiative is the idea of an open and honest conversation.”

Off periods are a common complication in Parkinson’s patients, characterized by the return of motor and non-motor symptoms — such as anxiety and sadness — when levodopa’s effects wear off. The need to better understand off periods, as well as how to talk with others about them, had been originally highlighted in the Live Well. Do Tell statement of need.

This report was the first output of the campaign, and includes six actions for the Parkinson’s community: tailor resources at diagnosis; broaden the Parkinson’s community’s ability to identify off periods; educate about motor fluctuations and how they do not necessarily mean disease progression; identify opportunities for caregivers to discuss their experiences with healthcare professionals; enhance the patient-clinician dialogue; and improve clinical practice via insights from movement disorder specialists and a new tool to measure off periods.

The report was based on insights from the campaign’s steering committee meeting that included Parkinson’s community leaders. Others may join the conversation by signing up for initiative updates and news.

“Off periods can be frustrating and challenging, as symptoms are varied and often difficult for patients to communicate effectively to their care providers,” said Matthew Stern, MD, director emeritus of the Parkinson’s Disease and Movement Disorders Center at Penn Medicine.

“By helping people with Parkinson’s understand how to verbalize what they are experiencing, we, as their healthcare providers, can better understand their needs and help them achieve a better sense of well-being,” added Stern, who is the chair of the Live Well. Do Tell steering committee.

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