Author: inspirnastg

Inspirna to Present at the Jefferies Healthcare Conference

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class cancer therapeutics, announced today that Chief Executive Officer Dr. Usman “Oz” Azam, M.D., will participate in a corporate presentation at the Jefferies Healthcare Conference on June 8, 2023 at 1:30pm ET.

Links to the live and archived versions of the presentation will be available on Inspirna’s website under the News section.

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates. Inspirna’s lead drug candidate, ompenaclid (RGX-202), is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. Ompenaclid is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced or metastatic RAS mutant colorectal cancer.

Inspirna’s second clinical stage drug candidate, abequolixron (RGX-104), is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. Abequolixron is currently being tested in a Phase 1b/2 clinical trial in patients with advanced or metastatic lung cancer in combination with docetaxel and endometrial cancer in combination with ipilimumab through a clinical collaboration with Bristol Myers Squibb.

Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, Silicon Valley Bank, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna Appoints Dr. Usman “Oz” Azam, M.D., as New Chief Executive Officer

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class cancer therapeutics, announced today the appointment of Dr. Usman “Oz” Azam, M.D., as its new Chief Executive Officer. Masoud Tavazoie, M.D., Ph.D., co-founder of Inspirna, recently stepped down as Chief Executive Officer and Director and will assume a new role as Advisor to provide ongoing support to the Company.

“Leading Inspirna will be an exciting opportunity to develop and commercialize novel drug candidates that target critical molecular drivers of cancer, such as ompenaclid and abequolixron,” said Dr. Azam. “2023 is a compelling year for Inspirna as the company continues to advance its clinical and preclinical pipeline. Inspirna’s proprietary, microRNA-based target discovery platform, RNA-DRIVEr™, has unmatched potential to deliver patient benefit in cancers of high unmet need, and I look forward to providing future updates as we enter our next chapter of growth.”

“We are delighted to welcome Dr. Azam as Inspirna’s new Chief Executive Officer. Oz brings over two decades of drug development experience, serving at the helm of innovative biotechnology companies in recent years. He has also helped bring new drugs to market, most notably during his time as Global Head of Cell and Gene Therapies at Novartis where he helped deliver the first ever FDA approval for a CAR-T cell therapy in hematologic cancers,” said Dieter Weinand, Chairman of the Board. “With his significant expertise in the life sciences industry and proven leadership experience, Oz is uniquely positioned to lead Inspirna as we advance our pipeline candidates. As we welcome Oz, we also would like to thank Masoud for his many years of successful leadership. We wish him well in his next endeavors and look forward to continuing to benefit from his counsel as an advisor to the company.”

Dr. Azam most recently served as Chief Executive Officer of Empyrean Neuroscience, a genetic engineering company advancing a pipeline of neuroactive compounds targeting disorders of the central nervous system, and before that was President and Chief Executive Officer of Tmunity Therapeutics, a biotechnology company developing genetically engineered CAR-T cell therapies for solid tumor applications in cancer. Prior to Tmunity, he served as Global Head of Cell and Gene Therapies at Novartis, where he was responsible for commercial operations, business development licensing, new product commercialization, clinical development, regulatory affairs, and other aspects of the global cell and gene therapies business. Dr. Azam has also held roles as Chief Executive Officer of Novaccel Therapeutics, Chief Medical Officer of Aspreva Pharmaceuticals, and Chief Medical Officer of Ethicon Johnson & Johnson. Earlier in his career, he held positions at Johnson & Johnson, GSK, and Pfizer. Dr. Azam received his M.D. from the University of Liverpool School of Medicine and is board certified in obstetrics and gynecology in the United Kingdom.

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates. Inspirna’s lead drug candidate, ompenaclid (RGX-202), is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. Ompenaclid is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced or metastatic RAS mutant colorectal cancer.

Inspirna’s second clinical stage drug candidate, abequolixron (RGX-104), is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. Abequolixron is currently being tested in a Phase 1b/2 clinical trial in patients with advanced or metastatic lung cancer in combination with docetaxel and endometrial cancer in combination with ipilimumab through a clinical collaboration with Bristol Myers Squibb.

Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, Silicon Valley Bank, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna Announces Interim Data from Phase 1b/2 Study of Abequolixron (RGX-104) in Relapsed or Refractory Lung Cancer

Abequolixron in combination with docetaxel in heavily-pretreated NSCLC and SCLC is well tolerated and demonstrates encouraging preliminary clinical activity

January 05, 2023 08:00 AM Eastern Standard Time

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule cancer therapeutics, announced today interim data from its ongoing Phase 1b/2 clinical trial of abequolixron in patients with non-small cell lung cancer (NSCLC) and small-cell lung cancer (SCLC).

Abequolixron (RGX-104) is a first-in-class, small molecule liver X receptor (LXR) agonist designed to target APOE dysregulation and enhance anti-tumor immunity by inhibiting tumor angiogenesis, depleting myeloid derived suppressor cells (MDSC), and activating cytotoxic T lymphocytes (CTL). Abequolixron is currently being evaluated in a Phase 1b/2 clinical trial in combination with docetaxel in second- or third-line (2/3L) NSCLC as well as second-line (2L) SCLC. The primary objectives of the study include characterizing the safety profile and the antitumor activity of the abequolixron and docetaxel combination.

“The data thus far demonstrate that abequolixron is well tolerated in combination with full dose docetaxel, while showing encouraging clinical activity suggesting that this drug candidate could be a valuable addition to a current standard of care chemotherapy in heavily-pretreated patients with lung cancer,” said Hossein Borghaei, D.O., M.S., Chief of the Division of Thoracic Medical Oncology at Fox Chase Cancer Center and Principal Investigator on the study. “I am very encouraged by the overall response rate, disease control rate, and time to progression so far seen in the study and I look forward to further evaluation of this unique compound as the clinical trial progresses.”

“These data, while early, clearly show the potential that abequolixron may have in driving deep responses in patients with heavily-pretreated lung cancer,” said Masoud Tavazoie, M.D., Ph.D., Chief Executive Officer of Inspirna. “We are excited to begin the new year by announcing these promising data for abequolixron and look forward to sharing more updates as we continue advancing our novel pipeline in difficult-to-treat cancers.”

Key findings from the data

  • As of December 2, 2022, 17 patients were treated at the Recommended Phase 2 Dose (RP2D) as determined in the dose escalation stage, with 120mg abequolixron twice daily (BID), 5 days on/2 days off, plus docetaxel. Thirteen patients were evaluable for response per RECIST v1.1, including five patients with 2/3L NSCLC and eight patients with 2L SCLC.
  • The ORR across all evaluable patients was 38% (n=13), with a disease control rate (DCR) of 77%, including five patients who achieved partial response (PR).
  • In the 2/3L NSCLC cohort, of five evaluable patients, four patients achieved PRs with two patients achieving confirmed partial responses. Four of the five patients were on study treatment for > 19 weeks, including one patient for 25 weeks, one patient for 28 weeks and one ongoing patient at 37 weeks.
  • In the 2L SCLC cohort, of eight evaluable patients, one patient achieved a PR and five patients had a best overall response (BoR) of stable disease (SD). Four patients (50%) were on study drug progression free for > 24 weeks. One of the patients with a BoR of SD, although experiencing CNS progression at week 12, stayed on therapy for clinical benefit for 30 weeks without experiencing systemic PD.
  • Across the two cohorts (N=17), the most common TEAEs were fatigue in 12 patients, diarrhea and nausea in 9 patients, decreased appetite in 8 patients and neutropenia and weight loss in 7 patients. Of the most common TEAEs, Grade 4 events were neutropenia (2 patients) and Grade 3 events were fatigue (2 patients), diarrhea (1 patient), nausea (1 patient), decreased appetite (1 patient) and the others were Grade < 2.

The expansion stage of the study is ongoing and continues to enroll patients with 2/3L NSCLC. In addition, this Phase 1b/2 study includes an ongoing expansion arm of abequolixron in combination with Yervoy (ipilimumab) in second- or third-line endometrial cancer as part of a clinical collaboration with Bristol Myers Squibb.

About Abequolixron (RGX-104)
Abequolixron is an orally administered small molecule agonist of the Liver X Receptor (LXR) which activates expression of the APOE tumor suppressor protein. APOE expression becomes dysregulated (silenced) in the tumors of select patients with solid cancers. APOE dysregulation results in increased tumor angiogenesis (tumor blood vessel growth) as well as a shifting of the tumor myeloid cell population from immune-stimulatory to immune-suppressive, which are both counteracted by abequolixron. Abequolixron is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for APOE dysregulation, including SCLC and NSCLC.

About Inspirna
Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates including abequolixron. Inspirna’s lead drug candidate, RGX-202, is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced or metastatic colorectal cancer (CRC).

Inspirna’s drug candidate abequolixron is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. Abequolixron is currently being tested in a Phase 1b/2 clinical trial in patients with advanced or metastatic lung cancer or endometrial cancer.

Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, Silicon Valley Bank, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna to Present at the Evercore ISI Annual HealthCONx Conference

November 17, 2022 08:00 AM Eastern Standard Time

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule therapeutics, announced today that Chief Executive Officer Masoud Tavazoie, M.D., Ph.D., and Chief Operating Officer David Darst, MBA, will participate in a fireside chat at the Evercore ISI Annual HealthCONx Conference on November 29, 2022 at 4:45pm ET hosted by Evercore ISI research analyst Umer Raffat.

Links to the live and archived versions of the discussion will be available on Inspirna’s website under the News section.

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates. Inspirna’s lead drug candidate, RGX-202, is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced or metastatic CRC.

Inspirna’s drug candidate RGX-104 is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. RGX-104 is currently being tested in a Phase 1b/2 clinical trial in patients with advanced or metastatic lung cancer or endometrial cancer.

Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr™, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, Silicon Valley Bank, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

The Bio Report: Targeting RNA Dysregulation in Cancer

Inspirna is putting RNA biology to work to address difficult to treat cancers that affect large numbers of people, such as colorectal cancer, small cell lung cancer, and non-small cell lung cancer. Its platform technology is being used to discover the underlying drivers of cancer that can be drugged by both small molecules and biologics to address RNA dysregulation. We spoke to Masoud Tavazoie, co-founder and CEO of Inspirna, about the role RNA dysregulation plays in certain cancers, Inspirna’s platform technology, and its clinical pipeline in development.

Inspirna Raises $50 Million in Series D Financing

Funding to further support company in advancing its clinical stage oncology programs

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule therapeutics, announced that it has raised $50 million in a Series D financing round, co-led by new investors Sands Capital and Vivo Capital, with additional participation from new investor Dreavent 6. Existing investors also participated in the financing round including Novo Holdings A/S, Sofinnova Partners, Sixty Degree Capital Fund, New York City Investment Fund LLC, Lepu Holdings, and additional investors. In addition, Michael Ginder, of Sands Capital, and Jack Nielsen, of Vivo Capital, will join Inspirna’s Board of Directors.

Proceeds from the financing will support the clinical development of Inspirna’s lead drug candidate RGX-202-01 (“RGX-202”), a first-in-class oral small molecule inhibitor of SLC6a8 to treat RAS mutant advanced colorectal cancer (CRC), and RGX-104, an oral small molecule activator of LXR/APOE to treat lung cancer and endometrial cancer. The financing will provide the company with the funds necessary to further evaluate efficacy and safety for RGX-202 in second-line RAS mutant CRC in an ongoing Phase 1b clinical trial, for which the company recently disclosed interim data in a poster presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting.

“I would like to thank both our new and existing investors for the confidence they continue to demonstrate in the potential of our unique drug candidates to make a profound impact on patients in the clinic,” said Masoud Tavazoie, MD, PhD, and Chief Executive Officer and co-founder of Inspirna. “We are currently evaluating RGX-202 and RGX-104 in clinical trials in high unmet medical need settings and, with renewed support from our investors, are now well-positioned to further build on the promising efficacy signals and safety profiles for these drug candidates.”

About Sands Capital

Sands Capital is an active, long-term investor in leading innovative businesses globally. Our approach combines analytical rigor and creative thinking to identify high-quality growth businesses that are creating the future. The firm maintains an integrated investment platform spanning venture capital, growth equity and public equity. Sands Capital is based in Arlington, Virginia.

About Vivo Capital

Founded in 1996, Vivo Capital is a leading global healthcare investment firm with a diverse, multi-fund investment platform in venture capital, growth equity, buyout, and public equities. The Firm has approximately $6.4 billion in assets under management and has invested in over 290 public and private companies worldwide. Headquartered in Palo Alto, California, with additional offices in Asia, the Vivo team consists of more than 50 multi-disciplinary professionals. Vivo invests broadly in healthcare across all fund strategies, including biotechnology, pharmaceuticals, medical devices, and healthcare services, with a focus on the largest healthcare markets globally.

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing several first-in-class drug candidates. Inspirna’s lead drug candidate, RGX-202-01, is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202-01 is currently being tested in a Phase 1b clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced or metastatic CRC.

Inspirna’s drug candidate RGX-104 is an oral small molecule activator of LXR/APOE that inhibits angiogenesis and tumor myeloid derived suppressor cells to enhance the immune response against tumors. RGX-104 is currently being tested in a Phase 1b/2 clinical trial in APOE biomarker enriched patients with advanced or metastatic lung cancer or endometrial cancer.

Inspirna identifies novel cancer targets using its microRNA-based target discovery platform, RNA-DRIVEr™, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Sands Capital, Vivo Capital, Lepu Holdings Limited, Sixty Degree Capital, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Dreavent 6, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna to Present Phase 1b RGX-202-01 Clinical Trial Data at the 2022 ESMO World Congress on Gastrointestinal Cancer

Favorable efficacy and safety support further development of RGX-202-01 in advanced or metastatic second-line colorectal cancer

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule cancer therapeutics, announced today data from the ongoing Phase 1b clinical trial studying RGX-202-01 in combination with FOLFIRI and bevacizumab (FOLFIRI/BEV) in second-line advanced colorectal cancer (CRC) at the 2022 European Society for Medical Oncology (ESMO) World Congress on Gastrointestinal Cancer being held June 29 through July 2, 2022 in Barcelona, Spain.

“These clinical data continue to demonstrate the clear potential of RGX-202-01 to improve on the standard of care for patients with advanced or metastatic colorectal cancer, especially for those whose tumors harbor KRAS mutations,” said Andrew Hendifar, M.D., Assistant Professor at Cedars-Sinai Medical Center and Principal Investigator on the study. “RGX-202-01 employs a novel mechanism by inhibiting the creatine transporter SLC6a8, which enables cells to generate ATP as well as other nucleotides by importing phospho-creatine (p-creatine). These results also show that KRAS mutant tumors are highly sensitive to the effects of SLC6a8 inhibition by RGX-202-01. The drug is also very well-tolerated, enabling a safe and effective combination therapy with FOLFIRI/BEV to provide further optionality for patients.”

RGX-202-01 is an oral, potential first-in-class small molecule inhibitor of SLC6a8, a creatine transporter that drives colorectal cancer and certain other cancers’ progression. It is currently being evaluated in a Phase 1b dose escalation and expansion study in combination with FOLFIRI/BEV in second-line, advanced or metastatic CRC. The primary endpoint of the study is to determine maximum tolerated dose (MTD), overall response rate (ORR), and treatment-emergent adverse events (TEAEs). In the dose escalation stage of the study, two dose levels of RGX-202-01 with FOLFIRI/BEV have been evaluated in patients with advanced or metastatic CRC who have progressed on available oxaliplatin based first line therapy. In the ongoing expansion stage, additional patients with CRC are being treated at the dose of 3000mg PO BID to provide further characterization of the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of the treatment.

Key findings to be presented at ESMO World Congress on Gastrointestinal Cancer:

  • Previously reported data from the study (at ASCO 2022) as of a data cutoff of April 28, 2022 included 19 patients who were enrolled in the study, including eight total patients in the dose escalation stage treated with either 2400mg twice daily (BID) of RGX-202-01 plus FOLFIRI/BEV (n = 4) or 3000mg BID RGX-202-01 plus FOLFIRI/BEV (n = 4), and 11 patients treated in the expansion stage with 3000mg BID RGX-202-01 plus FOLFIRI/BEV.
  • 17 patients were evaluable for response per RECIST v1.1 at data cutoff, of which 10 patients had KRAS mutant tumors and seven patients had KRAS wild-type tumors.
    • In the KRAS mutant population, five patients (50%) had confirmed partial responses (PR) and five patients (50%) had stable disease (SD).
    • In the KRAS wild-type population, one patient (14%) had an unconfirmed PR, five patients (71%) had SD, and one patient (14%) had progressive disease (PD).
    • Preliminary median progression-free survival (mPFS) was 11.8 months in the enrolled patients with KRAS mutant tumors.
    • Tumor regression was observed to deepen over time in patients with KRAS mutant tumors, with first radiographic achievement of PR appearing as late as 40 weeks post-treatment induction.
  • Updates since April 28, 2022 (data cutoff date) through June 10, 2022:
    • Two patients with KRAS mutant tumors had follow up imaging: patient 102-3112 remains in PR status and patient 102-2407 now has SD.
    • Two patients with KRAS WT tumors had follow up imaging: patient 102-3121 remains in SD status and patient 103-3127 maintained SD radiographically but was declared clinical PD as per the Investigator.
    • The ORR remains unchanged (50% in KRAS mutant group, 14% in the KRAS WT group).
    • Two grade 3 TEAEs (neutropenia, pulmonary embolus) deemed unrelated to RGX-202-01.
    • No grade 4 or 5 TEAEs reported.

The poster is available at the Annals of Oncology website and inspirna.com/our-science/.

Poster Presentation Details

Title: Phase 1b study of RGX-202-01, a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)

Date and time: July 1, 2022 at 10:30 – 10:50am CEST and 4:30 – 5pm CEST

Abstract Category: Clinical Colon Cancer

Abstract Subcategory: Metastatic Disease

Abstract ID: 291

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancers of high unmet need. The company is pursuing two first-in-class small molecule drug candidates, RGX-202 and RGX-104. Inspirna’s lead drug candidate RGX-202 is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients with gastrointestinal cancers where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced CRC. Inspirna is also developing a first in class small molecule that activates ApoE, RGX-104 (abequolixron), in high unmet medical need lung cancer settings and in advanced endometrial cancer in collaboration with Bristol-Myers Squibb.

Inspirna identifies novel cancer targets using its RNA-based target discovery platform, RNA-DRIVEr™, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Sixty Degree Capital, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna to Present Phase 1b RGX-202-01 Clinical Trial Data at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting

ORR of 50% and median PFS of 11.8 months in patients with KRAS mutant advanced colorectal cancer (CRC) treated in the second-line

Favorable safety profile with a majority of TEAEs Grade 2 or lower, and no DLTs observed

Overall efficacy and safety data support further development of RGX-202-01 in combination with standard of care in advanced or metastatic second-line CRC

NEW YORK, NY – May 26, 2022 – Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, announced today new data from the ongoing Phase 1b clinical trial studying RGX-202-01 in combination with FOLFIRI and bevacizumab (FOLFIRI/BEV) in second-line advanced colorectal cancer (CRC) at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting being held June 3-7 in Chicago, Illinois.

“These exciting new data show the potential of RGX-202-01 to drive meaningful responses in patients with advanced or metastatic colorectal cancer, especially in the KRAS mutant setting where there is a clear opportunity to improve on the standard of care,” said Andrew Hendifar, M.D., Assistant Professor at Cedars-Sinai Medical Center and principal investigator on the study. “RGX-202-01 employs a novel mechanism by inhibiting SLC6a8, part of a pathway that becomes activated by colorectal cancer cells in order for these cells to survive, proliferate, and metastasize. Importantly, along with its preliminary signal of efficacy, these results also demonstrate that RGX-202-01 is very well-tolerated, enabling it to be combined with FOLFIRI/BEV and provide further optionality in this difficult-to-treat indication.”

RGX-202-01 is an oral, potential first-in-class small molecule inhibitor of SLC6a8, a creatine transporter that drives colorectal cancer and certain other cancers’ progression. It is currently being evaluated in a Phase 1b dose escalation and expansion study in combination with FOLFIRI/BEV in second-line, advanced or metastatic CRC. The primary endpoint of the study is to determine maximum tolerated dose (MTD), overall response rate (ORR), and treatment-emergent adverse events (TEAEs). In the dose escalation stage of the study, two dose levels of RGX-202-01 with FOLFIRI/BEV have been evaluated in patients with advanced or metastatic CRC who have progressed on available oxaliplatin based first line therapy. In the ongoing expansion stage, additional patients with CRC are being treated at the dose of 3000mg PO BID to provide further characterization of the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of the treatment.

Key findings to be presented at ASCO 2022:

  • The data cutoff for the presentation is April 28, 2022. As of data cutoff, 19 patients were enrolled in the study, including eight total patients in the dose escalation stage treated with either 2400mg twice daily (BID) of RGX-202-01 plus FOLFIRI/BEV (n = 4) or 3000mg BID RGX-202-01 plus FOLFIRI/BEV (n = 4), and 11 patients treated in the expansion stage with 3000mg BID RGX-202-01 plus FOLFIRI/BEV.
  • The dose escalation stage did not reach an MTD.
  • No dose limiting toxicities (DLT) were observed at either 2400mg BID or 3000mg BID doses.
  • There were only two Grade 4 TEAEs, and one of those was considered unrelated to treatment with RGX-202-01, and no Grade 5 TEAEs were observed.
  • 17 patients were evaluable for response per RECIST v1.1 at data cutoff, of which 10 patients had KRAS mutant tumors and seven patients had KRAS wild-type tumors.
  • In the KRAS mutant population, five patients (50%) had confirmed partial responses (PR) and five patients (50%) had stable disease (SD).
  • In the KRAS wild-type population, one patient (14%) had an unconfirmed PR, five patients (71%) had SD, and one patient (14%) had progressive disease (PD).
  • Preliminary median progression-free survival (mPFS) was 11.8 months in the enrolled patients with KRAS mutant tumors.
  • Tumor regression was observed to deepen over time in patients with KRAS mutant tumors, with first radiographic achievement of PR appearing as late as 40 weeks post-treatment induction.
  • Overall, results show ORR and mPFS exceed expected benefit with standard-of-care alone in second-line CRC.

“We are very encouraged by the data reported today showing a strong signal of activity and meaningful responses, especially in patients harboring KRAS mutant tumors,” said Masoud Tavazoie, M.D., Ph.D., Chief Executive Officer of Inspirna. “The results not only support our efforts to continue advancing RGX-202-01 in CRC, but also validate the ability of our RNA-DRIVEr™ platform to discover and develop new drug candidates with the potential to address cancers of high unmet medical need. We look forward to sharing these results at ASCO and further advancing RGX-202-01 drug development.”

The abstract is available for viewing on the ASCO website, and the poster will be available at inspirna.com/our-science/ following the session.

Poster Presentation Details

Title: Phase 1b study of RGX-202-01, a first-in-class oral inhibitor of the SLC6A8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)

Date and time: Saturday, June 4, 2022, 8:00 a.m. CDT

Session: Gastrointestinal Cancer—Colorectal and Anal

Abstract ID: 3579

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancer high unmet progression. The company is pursuing several first-in-class drug candidates to treat cancers of need. Inspirna’s lead drug candidate RGX-202 is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients with gastrointestinal cancers where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced CRC. Inspirna expects to present data from this clinical study in mid-2022.

Inspirna identifies novel cancer targets using its RNA-based target discovery platform, RNA-DRIVErTM, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Sixty Degree Capital, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna Announces Phase 1b RGX-202 Clinical Trial Data Selected for Poster Presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting

April 27, 2022 10:00 AM Eastern Daylight Time

NEW YORK–(BUSINESS WIRE)–Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, announced today that it will present a poster at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting being held June 3-7 in Chicago, Illinois.

Poster Presentation Details
Title: Phase 1b study of RGX-202-01, a first-in-class oral inhibitor of the SLC6A8/CKB pathway, in combination with FOLFIRI and bevacizumab (BEV) in second-line advanced colorectal cancer (CRC)
Date and time: Saturday, June 4, 2022, 8:00 a.m. CDT
Session: Gastrointestinal Cancer—Colorectal and Anal
Abstract ID: 3579

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancer high unmet progression. The company is pursuing several first-in-class drug candidates to treat cancers of need. Inspirna’s lead drug candidate RGX-202 is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients with gastrointestinal cancers where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced CRC. Inspirna expects to present data from this clinical study in mid-2022.

Inspirna identifies novel cancer targets using its RNA-based target discovery platform, RNA-DRIVErTM, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Sixty Degree Capital, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton
212-600-1902
inspirna@argotpartners.com

Inspirna to Present at 21st Annual Needham Virtual Healthcare Conference

New York, NY – April 7, 2022 – Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, announced today that CEO Masoud Tavazoie, M.D., Ph.D., will present at the 21st Annual Needham Virtual Healthcare Conference at 10:15 A.M. EST on April 14, 2022.

Links to the live and archived versions of the presentation will be available on Inspirna’s website under the News section.

About Inspirna

Inspirna, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancer high unmet progression. The company is pursuing several first-in-class drug candidates to treat cancers of need. Inspirna’s lead drug candidate RGX-202 is an orally-administered small molecule that targets the CKB/SLC6A8 pathway. This pathway becomes activated in the tumors of select patients with gastrointestinal cancers where it enables the generation of the energy molecule ATP in response to tumor hypoxia. RGX-202 is currently being tested in a Phase 1b clinical trial in combination with standard-of-care FOLFIRI and bevacizumab for the second line treatment of patients with advanced CRC. Inspirna expects to present data from this clinical study in mid-2022.

Inspirna identifies novel cancer targets using its RNA-based target discovery platform, RNA-DRIVErTM, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, and a leadership team comprised of experienced drug developers. The Company is funded by leading biotechnology investors, including Novo Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Sixty Degree Capital, K2 HealthVentures, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.inspirna.com.

Contacts

Argot Partners
Maeve Conneighton 
212-600-1902 
inspirna@argotpartners.com