Horizon Pharma (officially Horizon Pharma plc, Nasdaq: HZNP) is one of the biggest bio-pharmaceutical companies in the US. It’s a pharma company that’s well known for acquiring, rebranding, repackaging, and repricing specialist biopharmaceutical products for sale, especially to the United States healthcare system (which accounts for over 97 percent of their global sales).
As we plunge into 2019, we take a look at some of the eye-catching news headlines surrounding Horizon Pharma brand last year (2018).
Ravicti (glycerol phenylbutyrate) is a supplemental oral drug, popularly used as a nitrogen-binding agent for the treatment of several forms of chronic urea cycle disorders (UCDs). The drug works by counteracting or reducing the harmful accumulation of ammonia in the body as a result of the urea disorder.
Since it was first given the greenlight as a prescription drug by the Food and Drug Administration (FDA) back in 2013, Ravicti has been used particularly in the US to manage elevated ammonia levels in adults and children older than 2 months.
That is why it came as a huge relief when the FDA extended the age range of the drug to include infants younger than two months. This means Ravicti is now FDA-approved for everyone, including newborns.
In early 2018, Horizon Pharma USA, Inc., the American subsidiary of Horizon Pharma plc announced a plan to establish a first clinical team endowment at the CNRDI. The biopharma company made a $3 million commitment to the endowment for the next 6 years. The so-called Horizon Pharma Clinical Care Endowment was designed to give the Children’s National Rare Disease Institute a foundation from which to speed up the diagnosis and treatment of rare diseases in both adults and children. Given that one in every 10 Americans has a rare disease, the endowment will help a clinical team comprised of social workers, nutritionists, researchers, and genetic counselors to provide better patient care and facilitate early discovery.
Horizon Pharma announced in late September that they would collaborate with NAPRTCS to launch a natural history registry to cater to long-term data evaluating Americans living with cystinosis, a rare, life-threatening disorder that causes irreparable damage to vital organs, cells, and tissue. According to the joint press release by the two organizations, the cystinosis registry will gather and sort patient data reported by their doctors. This data, in turn, will help healthcare practitioners get a better understanding of the natural history of nephropathic cystinosis, which currently affects only around 2,000 people across the globe.
This was big news because the data will also be made available to researchers and other stakeholders seeking to discover and develop potential drugs and treatments for the disorder. The company also contacted medical leaders, leading physicians in the cystinosis community, and other healthcare professionals to ensure that the registry will make the desired impact.
On October 4th, 2018, at the American Thyroid Association Annual Meeting, Horizon Pharma presented findings about the follow-up study that was held on the 48th week following the study period for the Phase 2 clinical trial studying teprotumumab, their later-stage drug for the treatment of active TED (thyroid eye disease).
According to the new data, the proptosis response of patient using teprotumumab since the initiation of the 24-week treatment period was nearly 74 percent compared to 13.3 percent for those who did not receive the treatment. The data further shows that 53 percent of on-teprotumumab patients maintained consistent improvement by the 48th week.
Teprotumumab is a potentially effective medicine developed by Horizon Pharma that’s currently on clinical trial for the treatment of this rare thyroid/eye condition. Although both the efficacy and safety of the drug have not been established by the FDA, the new data indicates that the end of the tunnel on TED is closer than ever.
In late September 2018, Horizon Pharma announced plans to enroll 30 patients in their new clinical trials examining the use of KRYSTEXXA® with Methotrexate to facilitate treatment for people living with chronic gout refractory (popularly known as uncontrolled gout). This was important news because most people suffering from the condition have been unresponsive to a diversity of oral therapies traditionally recommended by physicians.
The use of KRYSTEXXA, a crucial therapy for patients showing progressive symptoms of uncontrolled gout, with methotrexate (a known regulator of the immune system) will help reduce the levels of antibodies that fight against conventional therapeutic drugs.
Earlier clinical trials with KRYSTEXXA lasting over 6 months showed that 42 percent of patients responded fully to oral therapies, maintaining low levels of serum uric acid. In the newly announced trial, Horizon Pharma intends to emulate these results and eventually address the burden of chronic gout refractory once and for all.
Early in May, Horizon Pharma plc was pleased to announce that Sangita Iyer had been named a 2018 Healthcare Businesswomen’s Association (HBA) Rising Star, a prestigious award that honors women professionals who are successful in their early to mid-stage of their careers in healthcare.
Sangita, who has been with Horizon Pharma since joining from Fenwal, Inc in 2015, was recognized at the 29th annual Woman of the Year event, which was held in New York on May 03, 2018. The HBA is a world renowned nonprofit that champions the advancement of women in healthcare and recognizes their impact in the global healthcare sector.
According to HBA, Sangita Iyer was honored for her commitment to the healthcare sector, exceptional leadership, and myriad other contributions she has made to Horizon Pharma. Sangita is currently the director of Financial Planning and Analysis at the biopharmaceutical company.
In July 2018, the UK-based specialty pharmaceutical company Clinigen Group announced that they had purchased the global rights to Horizon Pharma’s Imukin for an undisclosed sum. This is a recombinant therapy that is designed to treat rare diseases such as Severe Malignant Osteopetrosis (SMO) and reduce the frequency of infections in people living with Chronic Granulomatous Disease (CGD).
At the time, Horizon Pharma plc was licensing Imukin in a total of 19 nations worldwide to facilitate SMO and GCD therapies. According to people privy to the purchase, Clinigen plans to revive Imukin by working closely with healthcare professionals to make sure that they fully understand in what ways the therapy is beneficial to patients.
All up, 2018 was a fairly busy and upbeat year for Horizon Pharma plc. For starters, its stock rose from a low of $13.76 at the start of the year to hit a high of a whopping $22.29 on November 5, 2018. If you crunch the numbers, that’s a 62 percent jump in Horizon’s stock value.
In the second month of 2018, Horizon Pharma made a rather bold move to increase the price on their flagship specialty drug, Vimovo, to nearly $3,000 for the 60-pill bottle. Interestingly, this wasn’t the first time Horizon revised the price upwards on the drug – in fact, it was the 11th time since it bought its US rights for $35 million in 2014.
Vimovo is a delayed-release pain reliever used to treat symptoms and signs of several forms of arthritis, including osteoarthritis, rheumatoid arthritis, and ankylosing spondylitis. Approved by FDA in 2010, Vimovo is actually a combination of two arthritis-focused therapies: Nexium by AstraZeneca and Aleve by Bayer. Back in 2014, it cost only $138 per 60-pill bottle.
Tricia Mullins, an ex-employee, brought a lawsuit against Horizon Pharma plc for allegedly firing her for being a whistleblower against their rival pharmaceutical company, Aegerion. According to Mullins, Horizon Pharma fired her in retaliation soon after Aegerion agreed on a $40 million settlement with the US Justice Department for misleading investors, not following regulatory safety procedures, and illegally marketing a cholesterol drug.
Horizon Pharma was named by Fortune Magazine and Great Place to Work as one of 2018’s best places to work for in Chicago. It is an honor that the pharma company has received for a number of years in a row. In fact, earlier in the year, Fortune named Horizon the #1 biopharma company to work for in 2018. In Great Places to Work’s 2018 list, Horizon Pharma was ranked 4th out of 25 other small and medium-sized firms in the greater Chicago area.
Interested in learning more about the best startups in pharma and healthtech? Check our guide to 31 best startups in 2018.