SarcoMedUSA INC. was formed in 2017 to determine if Pulmozyme could improve Pulmonary Sarcoidosis. The company is nearing a final license agreement for Tigerase. Tigerase is approved and SarcoMedUSA will conduct a sponsored Pulmonary Sarcoidosis study in a tertiary referral University that specializes in Sarcoidosis treatment.

Additionally, the company has an option on a no-alpha IL-2 mutein for cancer. SarcoMedUSA is working with Roswell Park Cancer Center in New York. There are final toxicology studies being completed and humanized mice studies. The drug has been used in the clinic whereby two (2) of twelve (12) patients with advanced cancer had partial remission.


SarcoMedUSA was formed in Texas in 2017 to determine if Pulmozyme could improve Pulmonary Sarcoidosis. The company is nearing a final license agreement for Tigerase. Tigerase is approved and SarcoMedUSA will conduct a sponsored Pulmonary Sarcoidosis study in a tertiary referral University that specializes in Sarcoidosis treatment.

Additionally, the company has an option on a no-alpha IL-2 mutein for cancer. SarcoMedUSA is working with Roswell Park Cancer Center in New York. There are final toxicology studies being completed in humanized mice studies which are ongoing and in our lab. The drug has been used in the Cuban clinic whereby two (2) of twelve (12) patients with advanced cancer had partial remission.

SarcoMed has assembled a nationally recognized team of drug development experts to move SM001 (alidornase) into the clinic. In 2021, our team was able to successfully complete a Pre-IND meeting with the Pulmonary Division of the FDA, which granted us orphan status.

Pulmonary Sarcoidosis is a significant market opportunity, with potential to generate over a billion dollars in annual revenue and help address a significant unmet need for the world wide patient population. SarcoMed is also exploring additional indications for SM001 such as Adult Respiratory Distress Syndrome, Idiopathic Pulmonary Fibrosis and Cystic Fibrosis.


Brian Davies, M.D., CEO


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Sarcoidosis is a heterogeneous, systemic disease characterized by noncaseating granulomatous inflammation affecting many organs. Almost 90% of the patients develop pulmonary sarcoidosis with the lung and mediastinal lymph nodes representing the most common sites affected by a sustained and progressive inflammation. The prevalence of sarcoidosis varies between different ethnic groups with the highest prevalence among Scandinavians and African Americans, while only rarely reported among Arabians and Asians. Sarcoidosis occurs in younger adults, typically peaking between 20 and 39 years of age.


The disease has a bimodal distribution pattern, with the second disease incidence peaking between 60 and 69 years of age, especially among women. Although the exact cause of sarcoidosis remains obscure, several putative etiopathogenic factors have been postulated. Among them, the presence of mycobacteria or propionibacteria DNA has very recently been identified in a considerable proportion of clinical isolates suggesting bacterial infections as either the cause or an essential cofactor for sarcoidosis pathogenesis. Once manifested, the hallmarks of sarcoidosis are aberrant immune responses to so far ill-defined antigenic triggers as well as a distinct granuloma formation. A clinical variant of sarcoidosis, called Lofgren syndrome, usually exhibits acute and distinct clinical entity that is characterized by the triad of erythema nodosum, hilar lymphadenopathy, and arthritis.

This trait of sarcoidosis has an excellent prognosis with a high remittance rate within the first 2 years following disease onset. However, a significant proportion of sarcoidosis patients who do not develop Lofgren syndrome (non-Lofgren syndrome) exhibits an insidious onset of the disease which develops into a chronic phase with granuloma persistence and a high risk of developing pulmonary fibrosis.


Microbial DNA along with Amyloid-like protein aggregates are found in sarcoidosis tissues Microbial DNA (mDNA) and Serum Amyloid A (SAA) aggregates cause inflammation mediated by TLR2 and TLR9. DNA stimulate Th1, Th17, TNF, IL6 cytokines which are critical to sarcoidosis inflammation. DNase I minimizes or prevents granuloma formation and inhibits the inflammatory effects of mDNA, AmyLP, SAA, hDNA through TLR2 and LR9.

Articles below that support the Mechanism of Action.

Chen et al. 2010. ‘Serum amyloid A regulates granulomatous inflammation in sarcoidosis through Toll-like receptor-2’, Am J Respir Crit Care Med, 181: 360-73.

• Drake et al. 2013. ‘Effects of broad-spectrum antimycobacterial therapy on chronic pulmonary sarcoidosis’, Sarcoidosis Vasc Diffuse Lung Dis, 30: 201-11.
• Eishi et al. 2002. ‘Quantitative analysis of mycobacterial and propionibacterial DNA in lymph nodes …’, J Clin Microbiol, 40: 198-204.
• Gupta et al. 2007. ‘Molecular evidence for the role of mycobacteria in sarcoidosis: a meta-analysis’, Eur Respir J, 30: 508-16.
• Tursi S, et al. (2017) Bacterial amyloid curli acts as a carrier for DNA to elicit an autoimmune response via TLR2 and TLR9. PLoS Pathog 13(4): e100631.


Our preclinical animal studies allowed for FDA Orphan drug approval. This FDA status grants faster approvals, provides tax advantages, FDA fee reductions and carries seven (7) years exclusivity.

The Murine sarcoidosis model study below was used by SarcoMedUSA for Orphan Drug Status approval.

Propionibacterium acnes (P. acnes) is the one of the microorganisms isolated from sarcoid lesions. P. acnes [protein & adjuvant] triggers a cellular immune response in sarcoid patients and induces pulmonary granulomas in sensitized mice. The heat-killed P. acnes is employed 28 days before therapy to ensure that the inflammation in the lung of a sarcoidosis-like condition is fully established prior to the initiation of therapy. In our study there were 8 animals in the control group (PBS) and 8 animals in the test group (rDNAse1). Inflammatory markers are measured in the control and test mice then compared after four weeks of therapy [day 56]:

• Significant decrease in all treated BALF cell cytokines/chemokines: g-CSF, INF-γ, IL-1β, IL-4, IL-6, IL-17, IP-10, MIP-1β, and MIP2 (p value range 0.003 to 0.05).

• Significant decrease in BALF leucocyte content in the lungs (p=0.007); total neutrophil content (p≤0.0001) neutrophil-leukocyte ratio (p=0.003).

• See results below:


SarcoMedUSA is working with Generium who received agency approval in Russia for Tigerase, a DNase 1. Below is a link to the paper that covers their Phase 3 study. approval study compared with Pulmozyme.

SarcoMed has arranged a Pilot [proof of concept study] in Kazakhstan [hospital has already approved the study]. Generium will pay for the cost of the drug. The CRO in Russia has quoted SarcoMedUSA $1 million for the study. The drug is approved in Kazakhstan. The doctor has ample patients for a 20-30 patient study.

We have three Pulmonary Specialist advisors who are experts in the Sarcoidosis field. If this sarcoid study shows improvement, then we would do a tech transfer to a US drug manufacturer, The results of this study will mitigate risk with the results of the study. If there is no positive signal, then SarcoMedUSA may stop development. Our advisors are helping design the protocol. Our advisors are experts in Sarcoidosis and have participated in designing numerous studies in Sarcoidosis.


The molecule IL-2 is a pleiotropic interleukin that is produced after the activation of the T cells (mostly CD4+) induced by antigens (Ag). This plays an essential role in the immune system, in its function as an autocrine as well as a paracrine growth factor.

The molecule induces the proliferation of the natural killer cells or NK lymphocytes, increasing their cytolytic or cytotoxic activity, as well as it has an essential role in the initialization and generation of memory CD8+ T cells (Gillis and Smith, 1977; Morgan et al., 1976). The combined actions of the IL-2 on the NK and the CD8+ T cells are postulated as its main mechanism to stimulate the antitumor activity. In a very outstanding way, the IL-2 particularly determines the development and the homeostasis of the T lymphocytes with CD4+FOXP3+ phenotype or regulatory T cells (Tregs).

These cells have an immune suppressive function and primarily they mediate the natural tolerance, being they are vital to prevent the occurrence of autoimmune illnesses, to limit excessive inflammatory responses and to maintain the general homeostasis of the immune system (Malek and Bayer, 2004; Setoguchi et al., 2005).

Nevertheless, preclinical and clinical studies have documented the capacity of some tumors attracting/expanding the Tregs, contributing to limit the antitumor response against the malignant neoplasia. The IL-2/IL-2R signaling mediated by the STAT5 phosphorylation also contributes to stabilize the expression of FOXP3 in the Tregs, increasing with its suppressive capacity. The over expression (constitutive) of the IL-2R alpha chain (CD25) and its contribution to form the high affinity trimeric receptor is decisive for the survival and suppressor function of the Tregs. For example, a recent study demonstrated that the capacity of the Tregs to capture IL-2 and to deprive it from the CD8+ T cells in vivo, suppresses the expansion and uncontrolled activation thereof. High doses of IL-2 has been used for many years in the therapy of cancer, by itself and in combination with different vaccines.

Several studies showed a significant antitumor effect in metastatic Melanoma and metastatic Renal Carcinoma patients, something which made possible the registration of this drug by the FDA in 1992. The use of high doses of IL-2 in the medical practice had consistently shown that a small percent (15 – 16%) of the treated patients has a significant antitumor response of relatively long duration. In particular 5-6% of the treated patients have complete response and they were virtually cured after 10 years of following up.

Recent preclinical and clinical studies have revived the interest in an old drug as it is the IL-2. A clinical study in melanoma where a combination of this interleukin was used with an anti-CTLA4 antibody showed strengthening evidence in the reached anti-tumor response. Similar results have been documented in preclinical models with different anti-checkpoints antibodies: i.e. anti-CTLA4; anti-PD1/PD-L1. Other authors document as advantageous the use of the IL-2 to expand tumor infiltrating lymphocytes (TILs) or engineered T cells (CART) so much in vitro as well as in vivo in the context of the modern therapies of adoptive transference.

The preferential action of the IL-2 on the Tregs cells in vivo is undoubtedly an obstacle for its therapeutic exploitation in different contexts. In particular several studies in patients and in mice have shown that the capacity of the IL-2 to expand the Tregs limits significantly its anti-tumor effect. In patients a clear correlation between the expansion of Tregs ICOS+ and the absence of clinical effectiveness has been observed. Another bigger disadvantage for the native IL-2, concerning its therapeutic exploitation is its great toxicity. It is reported that the therapy with high doses of IL-2 induces serious adverse events and that many patients are forced to suspend the therapy due to the induced toxicity (Vial and Descotes, 1992).

In fact, only patients with a very good general state can be subjected to the therapy, limiting significantly the impact. The main toxic effect caused by the IL-2 is the Syndrome of Capillary Fragility or Vascular Leak Syndrome (VLS). Recent articles suggest that this syndrome is mediated by the direct signaling of the IL-2 on the endothelial cells that express the high affinity trimeric receptor. The mIL-2 is a variant of the human IL-2 designed to preferentially stimulate the cells responsible for the antitumor activity, mainly the cytotoxic CD8+ T cells and the NK natural killer cells, without expanding/activating the Tregs cells.

Contrary to the human native IL-2, the mIL-2 has affected the capacity to interact with the alpha chain of its receptor (Carmenate et al., 2013). Therefore it has a very low capacity to stimulate the Treg cells that suppress the antitumor activity. It is for this reason that the mIL-2 shows bigger antitumor effect that the native IL-2 in vivo since it is able to increase the proportion between the effector lymphocytes and the regulatory T cells, favoring the antitumor response (Carmenate et al., 2013). Additionally, the mIL-2 has demonstrated that it induces less toxic effects than the native IL[1]2 even when it is administered at doses substantially bigger than necessary to induce the antitumor effect in mice.

The explanation of this low toxicity is not totally elucidated, but it could derive from its smallest capacity to interact with the high affinity trimeric receptors expressed on the endothelial cells of the lung and other organs. As a whole, the increased capacity to expand the CD8+ T and NK cells, in relation to the Tregs, and its reduced toxicity makes the mIL-2 a better candidate that the native IL-2 for the therapy of cancer. Particularly attractive is the exploration of its effect in combination with other therapies. For example: 1) The denominated anti-Checkpoints (anti-CTLA4/PD1/PDL1) that fundamentally liberate the brakes to the cytotoxic activity of the CD8+ T cells against mutated antigens in the tumor. It could be particularly useful in combination with tumors where the Tregs are outstanding in the tumor microenvironment or in the context where the activation of NK cell complements the CD8+T cells’ cytotoxicity, limiting the tumor escape by MHC-I expression decreases. 2) Therapeutic vaccines of cancer that seek to expand a CD8+ T cell response. In this context the mutant of IL-2 can contribute to expand the CD8+ T cells, stimulating them directly and making them independent from the regulation mediated by the Tregs.

Therapies of adoptive transference of T cells, native or engineered. Of particular interest can be the use in vivo of the mutant, to expand the CD8+ T or NK cell effectors that have been infused in a patient, without expanding those potentially noxious Tregs.

Pre-clinical studies of IL-2 and No-Alpha IL-2 Mutein

The No Alpha IL-2 Mutein was developed at the Center for Immunologic Molecular (“CIM”) by Dr. Rojas Dorantes Gertrudis, Dr. Leon Monzon Kalet, and Dr. Carmenate Portilla Tania. The IL-2 structure was modified by changing four (4) amino acids that modify the alpha receptor. The alpha affinity is reduced 1000-fold and this reduces the noted vascular side effects of wet IL-2.

The team has shown mono therapy effects in several cancer and synergistic effects with check point drugs in animal models. The NOEL is (2mg/kg (3.2 x (10)4 IU/ kg/ day) and in a Phase 1 study the drug is being used at one hundred (100) x the dose of wet IL-2 with plans to advance to higher levels. No serious toxicities have been noticed at the higher dose. There have been two (2) partial remission responses in the first twelve (12) patients treated.

The half-life for No-Alpha IL-2 is the same as wet IL-2. There is increased capacity to expand the CD8+ T and NK cells in relation to the T Regs, and its reduced toxicity makes the No Alpha Mutein IL-2 a better candidate that native IL-2 for cancer. SarcoMedUSA is confirming tests done in Cuba, at Roswell Park Cancer Center and our own laboratory. We are finalizing toxicity studies done in Canada. We are using Humanized mice studies with tumor engraftment to determine the clinical benefit of reducing tumor growth. If validated then SarcoMedUSA along with Roswell Park researchers will file an IND with the FDA in the United States.