Regenerative medicine sector on pace for exceptional year | 2020-08-18

A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine (ARM), finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising despite the challenges posed by the ongoing COVID-19 pandemic.

ARM found that about $10.7 billion has been generated globally by companies involved in the sector, a total that exceeds the amount raised in all of 2019 by a significant 120% margin.

In addition, for the first time since ARM began tracking the sector, the alliance determined that there are now more than 1,000 companies worldwide actively involved in cell and gene therapy as well as tissue engineering, a total that has grown about 2% since the beginning of the year.

Regionally, there has been a 2% growth in companies located in North America to 543, or approximately 54% of the global total, with 238 companies operating in Europe and Israel and 184 companies located in Asia, where the fastest growth occurred among the community of global companies last year.

No shortage of cash resources

The companies have been active in business development, with $10.7 billion collectively generated from financing transactions as well as corporate partnerships, an amount that is well on track to break the existing annual record of $13.4 billion that was set in 2018.

Among the notable financing transactions, Iovance Biotherapeutics Inc., of San Carlos, Calif., which is developing T cell-based cancer immunotherapies (tumor-infiltrating lymphocyte, TIL and peripheral-blood lymphocyte, PBL), closed a $603.7 million public offering to fund the commercial launch of lifileucel for advanced melanoma and LN-145 for advanced cervical cancer, and to start a program directed at registration of Iovance’s TIL therapies in non-small-cell lung cancer. The funding will also support ongoing commercial manufacturing activities, for the development of its IL-2 analogue, IOV-3001, as well as for other general corporate purposes.

Legend Biotech Corp. closed an IPO of about 21 million American depositary shares (ADSs), each representing two ordinary shares, at a public offering price of $23 per ADS, for total gross proceeds of about $487.3 million that will fund its Janssen Biotech Inc.-partnered BCMA-targeting CAR T candidate, LCAR-B38M.

On the venture capital front, cell and gene engineering company Sana Biotechnology Inc. raised $700 million in a record-setting first-round financing. The transaction helped boost the amount of venture capital invested into companies in the space in 1H 2020 to more than $3 billion.


The report also noted that business development continues to be brisk, with up-front payments from 2020 partnerships amounting to $1.6 billion. The leading deal was signed in June with CSL Behring licensing the global rights to Uniqure NV’s late-stage hemophilia B gene therapy candidate, AMT-061, in return for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. In the same therapeutic area, Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., agreed to pay Cambridge, Mass.-based Intellia Therapeutics Inc. $70 million up front, alongside making a $30 million equity investment in the company, in an expansion of an ongoing partnership. The revised collaboration provides Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. Regeneron also receives nonexclusive rights to independently develop and commercialize ex vivo gene edited products

Productive pipeline

The growth of the sector is reflected in increased clinical development. The pipeline of regenerative medicine products is robust and continues to grow, with ARM identifying 1,078 ongoing clinical trials around the world. Among those ongoing studies, 63.4% are testing therapies in the mid- and late stages of their development, including 97 products currently in phase III testing, comprising 35 gene therapies, 15 gene-modified cell therapies, 30 cell therapies and 16 in tissue engineering, the ARM report found.

In terms of therapeutic focus, approximately 62% of the products being tested are targeting oncology indications, including rare forms of cancer, well ahead of the 5% of experimental therapies targeting CNS disorders and about 4% that are focusing on cardiovascular diseases.

The report also identified 11 ongoing clinical trials that are utilizing regenerative medicines and advanced therapies to treat COVID-19. In an ARM webinar on the topic, Michael Lehmicke, director, science and industry affairs at the alliance, noted that developers are utilizing mesenchymal stem cells and other stromal cells to treat acute respiratory distress syndrome caused by COVID-19.

High profile

In terms of FDA approvals for gene therapies, the industry is anxiously awaiting the agency’s decision on San Rafael, Calif.-based Biomarin Pharmaceutical Inc.’s high-profile marketing application for Valrox (valoctocogene roxaparvovec) its gene therapy to treat severe hemophilia, which is under priority review with a PDUFA date of Aug. 21. The application is based on a phase III interim analysis of study participants treated with investigational product and three-year phase I/II data.

If approved, the therapy would be the first potentially curative (one and done) approach to hemophilia A, eliminating the need for blood transfusions and factor VIII replacement therapy after a single infusion. People suffering from the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints and that group makes up approximately 43% of the hemophilia A population.

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