Outstanding First Half for Paragon and its Portfolio Companies

Paragon and its portfolio companies continue to successfully advance biology-based innovation, creating impressive results.

Outstanding First Half for Paragon and its Portfolio Companies

First Half 2022 Newsletter

We are a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. Since 2017, Paragon and its partners have invested over $1.4 billion to build life science companies.

Paragon Update – Two Quarters of Stellar Growth

Paragon and its portfolio companies continue to successfully advance biology-based innovation, creating results like a multi-target research collaboration and license agreement between Evozyne and Takeda, a licensing agreement for Emalex Biosciences to expand its central nervous system platform, and Castle Creek Biosciences’ recent financing and acquisition of in vivo capabilities for the development of novel gene therapies. The team at Emalex has started presenting data from its positive pediatric Tourette syndrome study at medical conferences. We also kicked off a series of videos featuring Paragon and our portfolio companies. Read on for details on all this and more!

Harmony Announces Strong First Quarter Financials, New Clinical Trial

Harmony Biosciences (Nasdaq: HRMY) recently issued strong first quarter 2022 financial results, noting its cumulative net revenue has surpassed $500 million since WAKIX® (pitolisant) launch. Net revenue increased 43% year-over-year to $85.3 million for first quarter 2022. Last year, Harmony achieved its first full year of profitability and closed the fourth quarter 2021 with a net income of $22.7 million.

In April, Harmony initiated a Phase 3 registrational trial to evaluate the efficacy and safety of pitolisant in adult patients with Idiopathic hypersomnia (IH), known as the INTUNE study. IH is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness despite sufficient or even long sleep time.

In addition, topline data from Harmony’s Phase 2 preclinical proof-of-concept trial in Prader-Willi Syndrome is expected in the second half of 2022 and Myotonic Dystrophy topline data in 2023.

Evozyne Inks $400M Deal with Takeda

Takeda is once again tapping into Evozyne’s expertise in creating novel protein sequences. Recently Evozyne entered into a remarkable strategic research collaboration and license agreement with Takeda to research and develop proteins that could be incorporated into next-generation gene therapies for up to four rare disease targets.

According to the agreement, Evozyne will receive double-digit million dollars in upfront and research funding payments. The company is also eligible to receive future developmental, regulatory and commercial milestone payments of up to $400 million plus tiered royalties on net sales of any commercial product resulting from the collaboration.

This new agreement builds upon an earlier, separate agreement with Takeda and demonstrates the potential of Evozyne’s protein engineering platform.

Watch this new Evozyne video to learn more about its incredible, novel protein work.

Castle Creek Completes $112.8M Raise and Novavita Acquisition

Castle Creek Biosciences recently announced the completion of an oversubscribed and upsized preferred stock financing of $112.8 million. The financing is expected to provide sufficient capital for the company’s completion of a Phase 3 study and issuance of topline results of its lead ex vivo product candidate for recessive dystrophic epidermolysis bullosa (RDEB), a progressive, painful and debilitating rare genetic skin disorder. In addition, the funding positions Castle Creek with capital to advance its in vivo work to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration for hereditary tyrosinemia type 1 (HT1), its first indication using the in vivo gene therapy technology.

Castle Creek’s dual technology platform of ex vivo and in vivo technologies is the foundation for developing novel gene therapies for a broad range of genetic diseases with limited or no treatment options.

Castle Creek’s acquisition earlier this year of Novavita Thera, Inc., a preclinical gene therapy company focused on liver and metabolic diseases, expanded Castle Creek’s technology platform by adding in vivo capabilities to its existing ex vivo approach.

The company also announced two key appointments in connection with the acquisition: Joseph Lillegard, MD, PhD, as chief scientific officer and Robert A. Kaiser, PhD, DABT, vice president of preclinical development. Drs. Lillegard and Kaiser will be the company leads for Castle Creek’s research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

Emalex Shares Positive TS Study Data; Acquires License for RLSa

At the American Academy of Neurology’s (ANN) annual meeting, Emalex Biosciences presented an abstract on its phase 2B study positive topline results for a potential therapy to treat pediatric patients with Tourette syndrome. Study data received last November showed statistically significant and clinically meaningful results with a 30% reduction in tics. The most frequent treatment-related side effects were headache, somnolence, sedation, anxiety, and restlessness. A phase 3 study is being developed and is expected to begin mid-year 2022.

Looking to expand its central nervous system platform, Emalex entered into a licensing agreement with East Carolina University for the patent rights to potentially advance clinical studies for the treatment of Restless Legs Syndrome with augmentation (RLSa).

RLS, also referred to as Willis-Ekbom Disease, is a neurological sensory disorder characterized by an irresistible urge to move one’s legs due to uncomfortable sensations. According to the International Parkinson and Movement Disorder Society, approximately 5%-10% of the general population is impacted by RLS, making it the most common movement disorder. RLSa is the worsening of RLS symptoms despite treatment with conventional therapies.

CiRC Team Expands; Moves to New Lab Space

Paragon’s latest company, CiRC Biosciences, continues to advance its pre-clinical work on potential vision restoration therapies for advanced retinitis pigmentosa and geographic atrophy age-related macular degeneration. The company has expanded its scientific team and recently completed its relocation to a larger lab space at 400 N. Aberdeen in Chicago.

News Briefs

Jeff Aronin, founder, chairman and CEO of Paragon Biosciences, recently received two awards: a platinum award for Entrepreneur of the Year by the TITAN Business Awards and a gold Stevie® Award recipient from the American Business Awards® as Entrepreneur of the Year. “These awards are a reflection of our critical work to accelerate biology-based scientific breakthroughs to solve some of society’s most challenging problem,” said Aronin. “These recognitions belong to the hard-working scientists and other employees that make up the Paragon family.”

Like what you’re reading? Subscribe to our newsletter and get the latest Paragon news delivered directly to your inbox.

Get Social

Keep up on the latest Paragon news by following our social media channels:

LinkedIn: https://www.linkedin.com/company/paragon-biosciences-llc
Twitter: https://twitter.com/ParagonBiosci
Facebook: https://www.facebook.com/ParagonBiosciences/
YouTube: https://www.youtube.com/channel/UChTOcSq9sjSVh6QDr9GEqjg

For company and industry insights, follow Jeff Aronin on
LinkedIn: https://www.linkedin.com/in/jeffrey-aronin/

First Half 2022 Newsletter

We are a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. Since 2017, Paragon and its partners have invested over $1.4 billion to build life science companies.

Paragon Update – Two Quarters of Stellar Growth

Paragon and its portfolio companies continue to successfully advance biology-based innovation, creating results like a multi-target research collaboration and license agreement between Evozyne and Takeda, a licensing agreement for Emalex Biosciences to expand its central nervous system platform, and Castle Creek Biosciences’ recent financing and acquisition of in vivo capabilities for the development of novel gene therapies. The team at Emalex has started presenting data from its positive pediatric Tourette syndrome study at medical conferences. We also kicked off a series of videos featuring Paragon and our portfolio companies. Read on for details on all this and more!

Harmony Announces Strong First Quarter Financials, New Clinical Trial

Harmony Biosciences (Nasdaq: HRMY) recently issued strong first quarter 2022 financial results, noting its cumulative net revenue has surpassed $500 million since WAKIX® (pitolisant) launch. Net revenue increased 43% year-over-year to $85.3 million for first quarter 2022. Last year, Harmony achieved its first full year of profitability and closed the fourth quarter 2021 with a net income of $22.7 million.

In April, Harmony initiated a Phase 3 registrational trial to evaluate the efficacy and safety of pitolisant in adult patients with Idiopathic hypersomnia (IH), known as the INTUNE study. IH is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness despite sufficient or even long sleep time.

In addition, topline data from Harmony’s Phase 2 preclinical proof-of-concept trial in Prader-Willi Syndrome is expected in the second half of 2022 and Myotonic Dystrophy topline data in 2023.

Evozyne Inks $400M Deal with Takeda

Takeda is once again tapping into Evozyne’s expertise in creating novel protein sequences. Recently Evozyne entered into a remarkable strategic research collaboration and license agreement with Takeda to research and develop proteins that could be incorporated into next-generation gene therapies for up to four rare disease targets.

According to the agreement, Evozyne will receive double-digit million dollars in upfront and research funding payments. The company is also eligible to receive future developmental, regulatory and commercial milestone payments of up to $400 million plus tiered royalties on net sales of any commercial product resulting from the collaboration.

This new agreement builds upon an earlier, separate agreement with Takeda and demonstrates the potential of Evozyne’s protein engineering platform.

Watch this new Evozyne video to learn more about its incredible, novel protein work.

Castle Creek Completes $112.8M Raise and Novavita Acquisition

Castle Creek Biosciences recently announced the completion of an oversubscribed and upsized preferred stock financing of $112.8 million. The financing is expected to provide sufficient capital for the company’s completion of a Phase 3 study and issuance of topline results of its lead ex vivo product candidate for recessive dystrophic epidermolysis bullosa (RDEB), a progressive, painful and debilitating rare genetic skin disorder. In addition, the funding positions Castle Creek with capital to advance its in vivo work to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration for hereditary tyrosinemia type 1 (HT1), its first indication using the in vivo gene therapy technology.

Castle Creek’s dual technology platform of ex vivo and in vivo technologies is the foundation for developing novel gene therapies for a broad range of genetic diseases with limited or no treatment options.

Castle Creek’s acquisition earlier this year of Novavita Thera, Inc., a preclinical gene therapy company focused on liver and metabolic diseases, expanded Castle Creek’s technology platform by adding in vivo capabilities to its existing ex vivo approach.

The company also announced two key appointments in connection with the acquisition: Joseph Lillegard, MD, PhD, as chief scientific officer and Robert A. Kaiser, PhD, DABT, vice president of preclinical development. Drs. Lillegard and Kaiser will be the company leads for Castle Creek’s research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

Emalex Shares Positive TS Study Data; Acquires License for RLSa

At the American Academy of Neurology’s (ANN) annual meeting, Emalex Biosciences presented an abstract on its phase 2B study positive topline results for a potential therapy to treat pediatric patients with Tourette syndrome. Study data received last November showed statistically significant and clinically meaningful results with a 30% reduction in tics. The most frequent treatment-related side effects were headache, somnolence, sedation, anxiety, and restlessness. A phase 3 study is being developed and is expected to begin mid-year 2022.

Looking to expand its central nervous system platform, Emalex entered into a licensing agreement with East Carolina University for the patent rights to potentially advance clinical studies for the treatment of Restless Legs Syndrome with augmentation (RLSa).

RLS, also referred to as Willis-Ekbom Disease, is a neurological sensory disorder characterized by an irresistible urge to move one’s legs due to uncomfortable sensations. According to the International Parkinson and Movement Disorder Society, approximately 5%-10% of the general population is impacted by RLS, making it the most common movement disorder. RLSa is the worsening of RLS symptoms despite treatment with conventional therapies.

CiRC Team Expands; Moves to New Lab Space

Paragon’s latest company, CiRC Biosciences, continues to advance its pre-clinical work on potential vision restoration therapies for advanced retinitis pigmentosa and geographic atrophy age-related macular degeneration. The company has expanded its scientific team and recently completed its relocation to a larger lab space at 400 N. Aberdeen in Chicago.

News Briefs

Jeff Aronin, founder, chairman and CEO of Paragon Biosciences, recently received two awards: a platinum award for Entrepreneur of the Year by the TITAN Business Awards and a gold Stevie® Award recipient from the American Business Awards® as Entrepreneur of the Year. “These awards are a reflection of our critical work to accelerate biology-based scientific breakthroughs to solve some of society’s most challenging problem,” said Aronin. “These recognitions belong to the hard-working scientists and other employees that make up the Paragon family.”

Like what you’re reading? Subscribe to our newsletter and get the latest Paragon news delivered directly to your inbox.

Get Social

Keep up on the latest Paragon news by following our social media channels:

LinkedIn: https://www.linkedin.com/company/paragon-biosciences-llc
Twitter: https://twitter.com/ParagonBiosci
Facebook: https://www.facebook.com/ParagonBiosciences/
YouTube: https://www.youtube.com/channel/UChTOcSq9sjSVh6QDr9GEqjg

For company and industry insights, follow Jeff Aronin on
LinkedIn: https://www.linkedin.com/in/jeffrey-aronin/