Pipeline in a Product: William Golden, Noveome Biotherapeutics

Noveome Biotherapeutics is a clinical-stage biopharmaceutical company focused on developing a unique “platform” biologic with powerful healing properties. The company’s lead product (ST266) is a new class of pharmaceutical product and consists of the secreted biological factors (the “secretome”) derived from a novel population of cells generated by a proprietary method of culturing amnion-derived epithelial cells. ST266 contains physiologic levels of biological factors, including those which stimulate anti-inflammatory and neuroprotective pathways essential to healing. ST266 is currently being evaluated in multiple indications including ophthalmological conditions such as optic neuritis, glaucoma and persistent corneal defects.

William Golden, founder, chairman and CEO of Noveome, spoke to Pharm Exec about the unique therapeutic potential of a secretome as a drug and the strategic approach to leveraging a platform biologic. 

Pharm Exec: As a secretome, ST266 is quite different from conventional biologics such as antibodies and even cell therapies. What is it, exactly?  

William Golden

William Golden: This therapeutic modality is quite different from conventional biologics. We takeC-section placentas which are normally discarded and isolate amnion epithelial cells. Those cells are put into our proprietary process and we harvest the secretome which contains hundreds of growth factors and cytokines involved in tissue healing. 

Tissue healing requires many biological factors acting at different times over the course of healing. During that time the levels of some biological factors go up and others decrease to create the perfect environment for healing. While a conventional drug that interacts with a single target might influence one part of the healing process, it can’t recreate the natural process – which is our goal with ST266. The biological factors contained within ST266 are at physiologic levels – picograms or nanograms per milliliter – and there are many of them, so a single dose can interact with many targets. In addition, the development goal with conventional therapeutics is to give the highest tolerable dose – typically milligrams – while avoiding too many side effects. Throughout all of our preclinical studies and clinical trials – in which we’ve now dosed more than 235 patients in nine clinical trials – we’ve never seen a serious adverse event. 

Another important aspect of ST266 is its lack of immunogenicity which results from the fact it is composed entirely of human biological factors. Immunogenicity can shutdown a clinic trial and for an approved drug, can limit its use and efficacy.    

You describe ST266 as a “platform biologic” and a “pipeline in a product.” What does that mean?

Unlike platform companies and platform technologies in which a foundational approach is used to discover and develop individual pipeline assets, ST266 is itself the platform; it has the potential to be used against a diverse range of diseases, conditions and injuries. We have a scalable manufacturing process to produce ST266 for all indications and a drug master file has been submitted to the FDA, supporting all IND applications. 

Not only does ST266 have a range of possible indications, it can be delivered via several routes of administration which also creates may options for treatment. These routes include topical application as well as local and systemic injection. Using a proprietary intranasal delivery device, we can bypass the blood-brain barrier and reach the optic nerve. 

With so many possible tissue healing indications, what is your strategy for selecting which to pursue? 

One of our initial studies in tissue healing was radiation burns with breast cancer patients, and in a recently published study, ultraviolet light burns, ST266 was applied as a spray and we showed that not only did ST266 prevent erythema – skin redness and rash – but biopsies of the tissue showed significantly reduced DNA crosslinking that characteristically occurs a result of ultraviolet radiation.  

We recently dosed our first patient in our clinical trial evaluating topical ocular delivery of ST266 for treatment of persistent corneal epithelial defects or PEDs. PEDs are non-healing wounds of the cornea caused by trauma, surgery and infection. They result from failure of the epithelial cells in the cornea to close the wound; this can lead to scarring, perforation and blindness. Currently, there are limited treatment options.  Interestingly, one current treatment option is to place a dried piece of amnion membrane over the eye, like a contact lens.  So it’s obvious that an added benefit of ST266 is that it is administered as eye drops – a much more patient-friendly and convenient route of administration. 

What other ophthalmologic conditions are you evaluating ST266 in clinical trials? 

A: We knew from preclinical studies that we could deposit ST266 intranasally to the back of the nose where the olfactory nerves are located using a novel intranasal delivery device. This device is different from conventional nasal sprays which deliver treatments such as antihistamines much less deeply. ST266 diffuses along those nerves and into the brain, getting past the blood-brain barrier. Interestingly, preclinical studies showed the highest concentration of radio-labeled ST266 was in the optic nerve. These results led us to explore inflammatory diseases or conditions of the optic nerve and that’s how optic neuritis and glaucoma emerged.  

Optic neuritis is the presenting symptom in about 50-60% of the cases of multiple sclerosis; essentially, it is the immune system attacking the optic nerve. It’s characterized by pain with eye movement and then one day, an individual will find that they’re essentially blind in one eye. The visual acuity eventually returns but it takes several months and afterwards, these individuals still have difficulty seeing at night.  In animal models of optic neuritis, their visual acuity returned the same levels as the non-diseased control animals when treated with ST266.  ST266 restored the number of retinal ganglion cells which are responsible for transmitting the images to the optic nerve and blocks demyelination of the nerves. 

What other indications are you exploring?

A: The ability to get ST266 past the blood-brain barrier certainly opens up many possibilities related to neurodegeneration. And there are certainly many instances of impaired tissue healing throughout the body. Our goal is to identify those with the greatest unmet need and ones that significantly impact quality of life.  One example is necrotizing enterocolitis, a life-threatening condition found in premature infants in which there is inflammation of the gastrointestinal system. We are working with a leading expert in the field and have gotten some very interesting and positive results in his animal models. Overall, we are very excited about the potential of ST266.