A History of Success

Patrick Lu, Founder, President, and CEO of Sirnaomics, is discovering and developing innovative RNA therapeutics with small interfering RNA (siRNA) and message RNA (mRNA) as novel modalities. Armed with more than 25 years of biopharmaceutical industry experience with big pharma and start-up environments for nucleic acid therapeutics, Patrick has been able to mitigate challenges and difficulties throughout Sirnaomics’ growth and development, and transform the company from a humble start-up to an internationally recognized public entity. He has led raising $340M for the company and developing a number of novel siRNA therapeutics, STP705, STP707 and STP122G for treating cancer, fibrosis and liver metabolic diseases. It is worth pointing out that Sirnaomics is an international corporation with the headquarters in Germantown, Maryland (recently relocated) and two subsidiaries in Asia. The company’s core R&D and clinical studies are based in the U.S.

As a serial entrepreneur, Patrick understood well how to define his scientific knowledge and organizational skills and advance those capabilities for building a biopharmaceutical venture with clear goals to address unmet market and patient needs. The same approaches and understanding are the key for budding entrepreneurs to start and establish their ventures. As the organization’s leader, Patrick believes that his scientific vision and cohesive execution are the keys to the company reaching its current heights.

Sirnaomics is an RNA therapeutics biopharmaceutical company with product candidates in preclinical and clinical stages that focus on discovering and developing innovative drugs for indications with medical needs and significant market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in China and the United States. It is also the first company to achieve positive Phase II clinical outcomes in oncology for an RNAi therapeutic with its polypeptide nanoparticle siRNA drug formulation.

Accomplishments Towards an Enhanced Tomorrow

In the summer of this year, Sirnaomics obtained results from the clinical trial of STP705 for the treatment of cutaneous basal cell carcinoma (BCC), which achieved a 100% complete response (CR), indicating the promising viability of the treatment. STP705 is a siRNA  therapeutic that is composed of two siRNA oligonucleotides, targeting the expression of TGF-β1 and Cox-2 mRNA, respectively. Apart from achieving a 100% CR , the data also showed improved or stable cosmetic results with an excellent safety profile (no adverse events) and no significant cutaneous skin reactions.  As Patrick commented about this important progress: “The latest results from the Phase II clinical study of STP705 for BCC treatment, showed an incredible efficacy without any drug related AEs and SAEs, further validating the broad potential of this drug candidate for treatment of non-melanoma skin cancers and beyond. Based on the successes of both BCC and isSCC (cutaneous Squamous cell carcinoma in situ) clinical studies, Sirnaomics is spearheading the development of novel polypeptide-based siRNA therapeutics for various types of cancers.”

The company recently dosed the first patient in a Phase I clinical study of STP705to study fat reduction in adults undergoing abdominoplasty. In this clinical trial, Sirnaomics has the opportunity to study a leading siRNA therapeutic candidate for medical aesthetics treatment. The dose-ranging, randomized, double-blind, vehicle-controlled study has 10 patients currently enrolled to evaluate the safety and tolerability of STP705, which is administered via subcutaneous injection. The primary endpoints are to assess injection comfort, characterize local and systemic safety, and evaluate histological changes. The safety and tolerability will be compared among three different concentrations of STP705 (120ug/mL, 240ug/mL, 320ug/mL) to select dosages for future studies.

Non-invasive fat reduction is a procedure that decreases or eliminates stubborn fat pockets in specific body areas using methods like cryolipolysis, radio frequency, and laser lipolysis. The global market size of non-invasive fat reduction was valued at $1.1 billion in 2021, with the U.S. having the largest market share and is anticipated to have an annual growth rate of 16.1% from 2022 to 2031. The Asia Pacific region is expected to outgrow the rest of the world at a pace of 17% over the same period due to the rising obese population, increasing healthcare expenditure, increasing adoption of advanced technology, and rising awareness by social media platforms.

“A non-invasive fat-reduction option will potentially offer millions of people with submental fat, which is resistant to diet and exercise and influenced by aging and genetics, a unique way to address an all-too-common condition,” explains Patrick. “After receiving the green light on an IND in the U.S., we look forward to better understanding how efficacious STP705 can be as a medical aesthetics treatment.”

As a believer in RNA therapeutics, Patrick insists on taking a unique approach to develop novel RNA therapeutics with advanced technology platforms. This “to be different, to be novel and to be successful” pathway has gained trust and strong support from the leadership team, allowing the company to become one of the leaders in the field of RNA therapeutics on the global stage. The clinical success of RNA therapeutics is facing many challenges. It requires at least three fundamental cornerstones:

• Selecting the right target(s)
• Equipping with an efficient and safe delivery system
• Taking on the proper indication for clinical study

A step-wise approach can provide a further foundation to achieve successful treatments for local and systemic diseases.

Towards the Future

Building a leadership team with unique expertise and willing to work cohesively is the best solution to understand the company’s technology and product better. The company has two technology platforms: for Polypeptide Nanoparticle Delivery and GalNAc-based Conjugation Delivery. “We have two teams working specifically on the development of siRNA drug products with our specialized technology platforms at a R&D stage, while the drug candidates are reaching the “Early Selected Compound” stage, the CMC, Pharm/Tox, Regulatory and Clinical teams are working in concert to send the right treatment for clinical testing,” elucidates Patrick.

As a biopharmaceutical company specializing in RNA therapeutics, Sirnaomics has embraced two significant achievements:

1. having the first clinical successes of its two-Phase II and one-Phase I studies for the treatment of various types of cancer, which not only validates its technology platforms but also demonstrates an enriched product pipeline.
2. having raised more than $340 million with a successful initial public offering.

Sirnaomics has two well-established delivery platforms for RNA drug administration. The Polypeptide Nanoparticle (PNP) formulation is well-suited for tumor and ex-hepatic tissue delivery, while the GalNAc-based (GalAhead™ and PDoV™) formulation is for liver hepatocyte-specific delivery. Two PNP-based siRNA drug candidates, STP705 and STP707, have already been tested in multiple clinical trials at Phase I and II for treating BCC, isSCC, Liver Cancer, Melanoma, Pancreatic Cancer, and Colorectal Cancer. The GalNAc-based STP122G is going to be in the clinical study soon.