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Orgenesis leads the charge at an exciting time for cell and gene therapy

Published: 11:00 09 Mar 2021 EST

Snapshot

The company is transforming the delivery of cell and gene therapy through its point-of-care therapeutics, technologies and network, thereby lowering costs

Orgenesis - Orgenesis leads the charge at an exciting time for cell and gene therapy
  • Global biotech unlocking the potential of personalized therapies and closed processing systems helpful in manufacturing
  • Maryland-based firm has a Cell and Gene Therapy (CGT) Biotech platform
  • The platform consists of three core elements: POCare Therapeutics, POCare Technologies, and POCare Network

What Orgenesis does:

Orgenesis Inc (NASDAQ:ORGS) is focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform. Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues.

The Germantown, Maryland-based company’s aim is to provide life-changing treatments at the point-of-care to patients at low cost. It's Cell and Gene Therapy Biotech platform has three key elements. The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies.

Finally, the third component rests on a point-of care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which Orgenesis is able to in-license technologies or advanced therapy medicinal products (ATMPs) and co-develop them with its partners.

In February last year, Orgenesis completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million in a bid to maximize value for shareholders and accelerate the rollout of our other divisions, with a focus on expanding the point-of-care business..

The successful sale spotlighted Orgenesis boss Vered Caplan’s considerable leadership skills. She has since been named one of the Top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of the world’s top drugmakers.

Caplan acquired Masthercell in March 2015, and grew the CDMO segment revenue from a run-rate of just $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million. Caplan has indicated that she plans to use the Masthercell sale proceeds to grow Orgenesis's evolving point-of-care cell therapy business and develop advanced therapy medicinal products. 

How is it doing:

On March 9 this year, the company told investors it expects its revenue to more than double in 2021, based on its existing contracts alone.

In a statement reporting its 2020 full year results, the firm revealed it was seeing traction and growth across its therapeutic pipeline, technology and network activity and that for the year to end-December, 2020, revenue had nearly doubled to $7.7 million, compared to $3.9 million in 2019.

"Over the past year, we have been hard at work building the foundation for our POCare platform, which we believe has the potential to unlock value across the cell and gene therapy industry," Vered Caplan, the CEO had said.

Notably, Orgenesis said it had "invested heavily" in new point-of-care technologies that can be integrated into its new OrGenesis Mobile Processing Units and Labs (OMPULs) and now developed, purchased or in-licensed more than 30 therapies and continues to expand its pipeline through its partnership with POCare centers.

On the network front, the company has established POCare development and service centers in the US, Belgium, Israel and South Korea, and ten joint venture agreements with regional partners that are financially committed to validate its therapies.

"In partnership with them, we have set up a network of hospitals and other partners in a total of 14 countries to date and we expect to sign agreements with many more premier institutions in the coming weeks and months, said Caplan.

It also now has its first commercial product on the market in the US, namely KYSLECEL for pancreatitis, following the company's acquisition of Koligo Therapeutics last October.

Koligo Therapeutics is a regenerative medicine company and the purchase included all of the assets of Tissue Genesis LLC. The consideration terms included $15 million in shares of Orgenesis stock valued at $7 share, which will be issued to Koligo’s accredited investors (with certain non-accredited investors to be paid solely in cash) and an assumption of $1.3 million in Koligo’s liabilities, estimated to be nearly all of Koligo’s liabilities.

Koligo’s management team joined Orgenesis (NASDAQ:ORGS) to continue commercial and development activities, adding a highly experienced US team to help further bolster Orgenesis’ point-of-care network in the US.

The company is also focused on the coronavirus (COVID-19) space. It is planning patient recruitment for a phase 2 randomized clinical trial of Koligo’s KT-PC-301, subject to US Food and Drug Administration (FDA) review and clearance of an investigational new drug (IND) application. KT-PC-301 is an autologous clinical development-stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, which Orgenesis acquired as part of the Koligo acquisition.

In addition, Orgenesis is gearing up for a Phase 2 study of Ranpirnase for the treatment of conditions caused by human papillomavirus, pending a planned IND submission to the FDA.

Meanwhile, Orgenesis has advanced the development of a cell-based vaccine platform targeting COVID-19 and other viral diseases. It has launched BioShield to set up a first line of defense against COVID-19 through the discovery of neutralizing human antibodies to contain the spread of the viral pathogen.

In addition, Orgenesis has signed an agreement with Fortune 500 science giant Leidos to develop, and potentially obtain FDA approval of Ranpirnase for treating COVID-19 patients. Ranpirnase has shown preclinical antiviral activity in diseases such as influenza, Ebola, SARS, and cytomegalovirus. Orgenesis acquired the broad-spectrum Ranpirnase antiviral as a part of its $12 million cash-and-stock deal to acquire Tamir Biotechnology.

Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

Infection points:

  • Massive upside potential as the sale of Masthercell Global equips Orgenesis with a war chest for acquisitions
  • Acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform ranpirnase, provides Orgegenesis with a significant opportunity
  • Possible gains from leveraging its expertise in the field of cell and gene therapy, including anti-viral technologies relevant to the coronavirus
  • Likely to gain traction from its joint ventures with several leading universities and healthcare institutions in Asia, North America and Europe
  • Expanding cell therapy market pegged at more than $32 billion

What the boss says:

In a statement, accompanying the financial results for 2020, Orgenesis CEO Vered Caplan said: "I am pleased to report that our revenues nearly doubled in 2020 to $7.7 million, which reflects growth in revenues related to technology transfer, setup, and validation of both our therapies and systems for clinical use.  We are committed to expanding our capacity and enabling the supply of our therapies globally, which provides us visibility into our future revenue growth.  Based on just the existing contracts in hand, we anticipate for revenue to more than double in 2021."

Contact the author Uttara Choudhury at uttara@proactiveinvestors.com

Follow her on Twitter: @UttaraProactive 

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