The last time Matinas BioPharma Holdings, Inc. (OTCMKTS:MTNB) made the news was for successfully completing a phase 1 study on its dyslipidemia candidate MAT9001. MAT9001 is an omega 3 fatty acid that showed superiority over its approved competitor Vascepa for lowering blood lipids in people with high and very high triglycerides.
While MAT9001 continues in development, now Matinas is attracting attention for a completely different product line of encochleated antifungals and antibiotics. Encochleated simply means that it is contained within a lipid envelope shaped like a microscopic croissant or cochlea. The basic idea of this line of candidates is to administer strong antibiotics and antifungals to patients without the active drug causing toxic side effects in the blood stream. Immune cells swallow the cochleates, which then unfurl inside the cells releasing the active drug within. The theoretical result is that the body is shielded from the systemic adverse effects of the active drug while the antibiotic or antifungal molecules are being ferried over directly to the sites of infection by the immune cells that swallow their containers.
Two of Mantinas’s cochleates have now received Qualified Infectious Disease Product status, or QIDP under the terms of the Generating Antibiotic Incentives Now Act (GAIN) passed in 2012. This status is similar to Orphan status for rare diseases, but directed at anti-infectives. QIDP offers eligibility for Fast Track, priority review and eligibility for five additional years of marketing exclusivity. Matinas’s first candidate to receive QIDP status was MAT2203 back in August. MAT2203 is an encochleated form of amphotericin B antifungal used to treat life-threatening infections in the immunocompromised. Today, December 17, Matinas announced another QIDP status agreement for another of its cochleates MAT2501, or encochleated amikacin for the treatment of non-tuberculous mycobacterium NTM infections.
The cochleate product line is completely different from its dyslipidemia trials, having been acquired from Aquarius Biotechnologies back in January. What’s advantageous about it is that it is essentially turnkey. If cochleates can work with one type of antibiotic or antifungal, they can theoretically work with any of them. And if one candidate (now two) can be granted QIDP status, then so can any other drug molecule that Matinas decides is worth “encochleating”. This gives Matinas an almost unlimited pipeline of potential candidates with special status with exclusive rights to the technology to boot.
Why these candidates are so important in the antibacterial and antifungal space is that there is currently no way of treating people with these serious infections without risking their lives from devastating side effects. Plus, the patient populations these infections occur in are typically post organ or bone marrow transplantation, which is why their immune systems cannot fight off the infection on their own. Having just survived organ failure or blood cancer only to have to encounter yet another life threatening situation is certainly no picnic.
According to Matinas CEO Roelof Rongen, the mortality rates for aspergillus fungal infections in transplant patients is between 39% and 75% even with treatment, partly because of the effects of the treatment itself. Untreated, the mortality rate is 100%, leaving a very grim choice indeed.
The second candidate to get QIDP status, encochleated amikacin, comprises a market of between 50,000 and 90,000 people in the US with NTM pulmonary disease. Intravenous administration has a high risk of toxicity to the kidneys and the ear, and can damage hearing or even cause deafness. Ironically, amikacin is specifically toxic to the cochlea in the ear, the actual organ of hearing. It would be poetic justice of the biotechnological variety indeed if encochleating amikacin can save patients from damage to their cochleas.
Matinas has already completed a phase 1 study for encochleated amphotericin B (CAMB) on healthy patients, showing that the encapsulated form of the antifungal shows no significant adverse effects. That means the cochleate envelopes are effectively shielding patients from any side effects of the drug. Preclinical studies have shown efficacy in mouse models with fungal infections, so there is reason to believe it can work in humans as well. That is what the phase 2a, sponsored by the National Institutes of Health, will try to prove. The trial is enrolling, with results expected in 2016.
A third encochleate using the antibiotic atovaquote for the opportunistic infection pneumocystic pneumonia is also in development. It has also shown efficacy in mouse models. Though farther back in development than CAMB, it points towards more evidence that the cochleates do work, not only in terms of blocking side effects, but also in terms of increased drug efficacy.
Positive results for the CAMB phase 2a trial could put Matinas into a new investment stratus by proving that strong anti-infectives and adverse side effects need not be linked together. Together with its so-far-successful dyslipidemia candidate, this little company is packed with potential and could see big gains in 2016.