Immusoft’s platform, Immune System Programming (ISP™) is a new medical paradigm, which can program cells to continually produce and secrete therapeutic proteins and rare antibodies that have been impossible to elicit with a vaccine. ISP™ offers many benefits of traditional approaches and modern gene therapy with less risk and greater control; replacing lifelong infusions with drug-producing cells
“The future of pharma is to learn to address the human body as an information platform. A company in Seattle named Immusoft, partly financed by venture investor Peter Thiel, has been successfully pursuing this strategy and proven its feasibility [for HIV and other diseases] in mice, using viruses and other vectors to program immune cells to emit needed molecules.” -- George Gilder, columnist and futurist
"If they can achieve therapeutic levels of antibody, this could be a real advance in cellular therapy" - Dr. David Baltimore, Nobel Laureate for Immunology, Professor at Caltech "It's a very attractive idea to supply stable levels of antibodies from a cellular source in the body. The challenge is finding the right cell that would persist for the desired amount of time – days, or years in some cases – and that's a big challenge." - Dr. Michael Sadelain, Memorial Sloan-Kettering Cancer Center "It might be possible to recreate the biochemical environment of youth." - Matt Scholz, CEO Immusoft
"Instead of letting the body try to randomly patch up the right combination to grab onto whatever it's attacking, we could leverage computational power to develop that novel protein or antibody in a computer and then simply tell the body how to make it." - Matt Scholz, CEO
It has no venture capital backing and just four employees, but in April, the company was one of six startups to win a grant from the Thiel Foundation, which aims to support what it calls “radical scientific innovations.” - Luke Timmerman - National Biotech Editor of Xconomy
"We want to take advantage of the revolution in healthcare and science that is happening and reward companies who are operating in a nimble way," circumventing the slow moving institutions of academia and traditional grants." - Lindy Fishburne, Executive Director Breakout Labs a project of the Thiel Foundation
“The goal of this study is to use Immusoft’s ISP™ platform to make human cells produce VRC01, an antibody that neutralizes many strains of HIV, in a mouse. Once we can demonstrate that the mouse cells can produce VRC01, we'll move on to primates, and then human trials.” - Dr. Eric Herbig, CSO
Opening a new frontier in biomedicine
At this point, Immusoft anticipates several key revenue streams.
The ISP™ platform will be out-licensed to biopharmaceutical companies seeking a robust, FDA-approved drug delivery technology to deliver life-saving biologic-based medicines to patients. We have already started discussions with biopharmaceutical companies interested in using the technology for the delivery of broadly neutralizing antibodies to HIV as well as those who are interested in using ISP™ for enzyme replacement therapy.
After ISP™ has been FDA-approved for a single indication (i.e. HIV or MPS I), biopharmaceutical companies will have greater incentive to use it in place of batch-to-batch scale-up of current Good Manufacturing Practice (cGMP) produced biologic medications — a precise and costly method to produce these types of medications. ISP™ largely eliminates the need for industrial-sized commercial bioreactors to produce protein therapeutic drugs. A partnership with a biopharmaceutical company to take our first indication into human clinical trials to receive FDA approval will provide this benchmark.
Separate from licensing ISP™ to biopharmaceutical companies to use, the treatment of rare genetic disorders also offers a unique business opportunity for Immusoft.
With fewer than 5,000 patients worldwide, MPS I, for example, has a small patient population. Immusoft would offer to treat MPS I patients directly using cGMP disposable bioreactor technology commercially available from GE Healthcare (i.e. Wave Bioreactor™) or an advanced closed system bioreactor created specifically for cellular therapy from Miltenyi Biotec (i.e. CliniMACS Prodigy™).
Currently, patients suffering from lysosomal storage disorders are charged between $250,000 and $400,000 yearly for their medication, and endure multiple-hour drug infusions every week. The annual market for MPS I is $180M. There are seven different types of MPS lysosomal storage disorders and several other lysosomal storage disorders with FDA-approved medications for which Immusoft’s ISP™ technology could be effective, including Gaucher’s disease, Fabry’s disease, and Pompe disease. Treatment of rare genetic diseases with ISP™ technology would likely require several doses of ISP™ cell therapy product. However, once enough cells have engrafted and blood serum levels of the medication are present, the patient would not require infusions of the drug intravenously.
ISP™-modified cells can be frozen and banked for administration at some point in the future if necessary. In this capacity, our ISP™ cell therapy product represents a disruptive technology to companies like BioMarin and Genzyme, which receive huge profits from the manufacture and distribution of these medications for intravenous infusions worldwide. The convenience of ISP™ technology as well as the sustained production of the medication by the patient’s own cells is thought to provide significant benefit over intravenous drug infusions. Moreover, Immusoft can treat patient populations of fewer than 100 patients, something that would be economically infeasible for biopharmaceutical companies to scale using the conventional bioreactor approach.
Several private foundations exist for these neglected lysosomal storage diseases. ISP™ technology offers the hope that some of these could be treatable with an approach like ours.
The combination of royalties from licensing relationships with biopharmaceutical companies and revenues from direct sales of ISP™ cell therapy products will account for a significant portion of our revenues. So too, we anticipate, would be FDA-approval of ISP™ technology indications with larger patient populations and consequently larger market sizes, such as heart disease, which is worth more than $100M annually.
All three of those revenue streams combined put us within reach of privately funding the company through phase III clinical trials. We suspect that would make Immusoft an attractive acquisition for a large pharmaceutical company such as Pfizer or Merck.
If Immusoft ever decided to provide an initial public offering it would likely come after FDA-approval of our first or second ISP™ cell therapy product.
Immusoft effectively is a part of four distinct markets: HIV treatments, treatments for rare genetic disorders (MPS I, Pompe, PKU, Hemophilia), heart disease, and out-licensing relationships.
HIV, which is the fastest path to clinical trials, has a U.S. market value of more than $9B. The NIH paid for our HIV proof of concept, and our ISP™ cell therapy product can be used to produce two broadly neutralizing antibodies to HIV.
The market for rare genetic disorders (MPS I, Pompe, PKU, Hemophilia) for which we could use ISP™ to treat by directing a cell therapy product to produce missing enzymes is $180M annually. Each of those four disorders — MPS I, Pompe, PKU, Hemophilia — has a market value of $6B. And with each of these, it is anticipated that the regulatory approval process would be faster and less expensive than either HIV or other indications such as heart disease.
Heart disease has a market value of more than $100B. There is a well-documented, naturally occurring HDL protein that has proven effective in human trials. We would use a ISP™ cell therapy product to produce a better version of this naturally occurring HDL to remove arterial plaque.
Finally, our out-licensing deals can be expected to be worth an estimated $10M to $250M, our direct sales of ISP™ cell therapy products worth an estimated $100M to more than $500M, and acquisition of the company by a leading biopharmaceutical company worth more than $1B.
We understand that progress has been hindered in the advancement of delivering biologic-based medicines because routine and frequent needle injections can incur more cost to the end user(s). Many of these life-saving medications cannot be delivered orally in a pill, and can cost between $250-$1000 per month — a prohibitive cost to most patients.
Enhanced delivery of protein therapeutic medicines like ISP™ enables drug discovery companies to do what they do best, design more effective molecular compounds for the treatment of disease. Immusoft has developed a platform technology that can deliver these drugs by simply replacing the source code that produces this sequence.
ISP™ technology enables the production of antibodies, which would be impossible to elicit with a vaccine using the conventional vaccine approach, can produce structurally engineered proteins which bind with higher affinity, and can replace a lifetime of infusions with a patient’s drug-producing cells.
Ten years ago a platform technology would have never made it to market simply because it would be too disruptive. Either the FDA would have never allowed it because it was too radical or biopharmaceutical companies would have lobbied against it because it would significantly interfere with profit margins.
What a difference a decade makes. The gene/cell therapy regulatory environment within the FDA (and abroad) is changing, and biopharmaceutical companies suffering from the extremely high cost of bringing a biologic to market (more than $1B on average) are seeking platform technologies that enable them to scale-up their medications to more users. Furthermore, crowd-investing platforms provide a mechanism where companies with disruptive technologies can thrive without the buy-in from venture capital investment funds that have typically acted as gatekeepers to innovative ideas.
The world of medicine and disease management changes daily. But even the best of medicines and treatments are nothing more than sugar water if they can’t be delivered effectively and efficiently. Immusoft’s ISP™ technology steps in to fill the void between therapeutic advances and the ability to deliver biologic-based medicines. We believe it’s the future of medicine. And we’re happy to shake up the system — to change the medical paradigm.
We have mitigated many risks since founding the company in 2009. What follows are just a few of the challenges we’ve faced and solved:
·Can you insert DNA into an immune cell and secrete a desired protein? Yes.
·Will the secreted protein be produced in therapeutic quantities? Yes.
·Will the ISP™ cell therapy product engraft into the host? Yes.
·Can you eliminate the ISP™ cell therapy product from the host? Yes.
·Can you produce lysosomal storage enzymes? Yes.
Risks we’re still working on solving:
·Will the lysosomal storage enzymes be taken up into the enzyme-deficient cells?
·Will the sustained delivery of broadly neutralizing antibodies eradicate HIV permanently in a patient?
·Will biopharmaceutical companies adopt the platform technology?
·Will we raise enough capital to enter a human clinical trial with a ISP™ cell therapy product to test the safety and efficacy of our treatment approach?
·Can the technology be scaled in order to be made available in hospitals and out-patient care facilities?
We anticipate our customers will come through the following: partnership and distribution agreements with biopharmaceutical companies; private foundations (such as rare disease foundations like MPS I Society and Muscular Dystrophy Association); community outreach (HIV+ support groups); and, a comprehensive integrated marketing and communications as well as sales strategy.
At this point, we’re primarily focused on the technology and clinical trials. If we can demonstrate the technology in a human clinical trial and receive FDA approval for an ISP™ cell therapy product, we will have cleared a major hurdle to successful commercialization of the ISP™ platform. Subsequent to that technical advance, we would focus increased efforts on more traditional customer development and relationship management functions.
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