On December 14, 2022 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company utilizing a cost-efficient, CRO-independent product development platform to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies, reported that the IDMC for the ongoing ENVASARC Phase 2 pivotal trial recommended continued accrual as planned in both cohorts: single agent envafolimab and envafolimab in combination with Yervoy (ipilimumab) (Press release, Tracon Pharmaceuticals, DEC 14, 2022, View Source [SID1234625272]).

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The IDMC reviewed interim safety and efficacy data from 18 patients enrolled into each cohort who completed a minimum of 12 weeks of efficacy evaluations (two on-treatment scans). The double-digit ORR assessed by blinded independent central review in each cohort more than satisfied the prespecified futility rule. Envafolimab monotherapy and in combination with Yervoy was well tolerated, with only a single related serious adverse event reported in 36 patients. Responses were noted in patients regardless of weight at the 600 mg dose of envafolimab that was instituted following the previous IDMC review of interim safety and efficacy data from patients in the ENVASARC trial treated at the 300 mg dose of envafolimab.

"We are pleased with the activity of the 600 mg dose of envafolimab that has demonstrated a double-digit objective response rate both as a single agent and in combination with Yervoy, even at this early 12-week time point," said James Freddo, M.D., TRACON’s Chief Medical Officer. "We are also encouraged with the safety data showing that envafolimab monotherapy and in combination with Yervoy are well tolerated. This interim analysis is a positive milestone and we look forward to the next interim analysis which will be conducted after the 46th patient in each cohort has completed a minimum of 12 weeks of efficacy evaluations. Importantly, accrual in ENVASARC remains ahead of projections and we are excited by the emerging data and for envafolimab’s potential to become a differentiated treatment for sarcoma patients."

"Achieving a double-digit ORR with a well tolerated safety profile, as both monotherapy and in combination, positions envafolimab to become a potential treatment option for patients with refractory UPS and MFS," said Charles Theuer, M.D., Ph.D., TRACON’s Chief Executive Officer. "The sole approved treatment for these patients is Votrient, which achieved a 4% ORR and carries a black box warning for fatal liver toxicity."

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology and licensed by TRACON, is the first approved subcutaneously injected PD-(L)1 inhibitor. Envafolimab was approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. In September 2022, TRACON received fast track designation from the U.S. Food and Drug Administration for envafolimab (KN035) for patients with locally advanced, unresectable or metastatic undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) who have progressed on one or two prior lines of chemotherapy.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at 30 top cancer centers in the United States and the United Kingdom that began dosing in December 2020. TRACON expects the trial to enroll more than 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into a cohort of treatment with single agent envafolimab at 600 mg every three weeks and 80 patients enrolled into a cohort of treatment with envafolimab at 600 mg every three weeks with Yervoy. The primary endpoint is objective response rate by central review with duration of response a key secondary endpoint.

On December 14, 2022 Tiziana Life Sciences Ltd. (Nasdaq: TLSA) ("Tiziana" or the "Company"), a biotechnology company enabling breakthrough CNS immunomodulation approaches to enhance the functionality of Treg-based therapies, reported that it has received a notification letter from Nasdaq Stock Market LLC that the Company has been granted an additional 180-day compliance period, with a new deadline of June 12, 2023, to regain compliance with Nasdaq’s minimum bid price rule (Press release, Tiziana Life Sciences, DEC 14, 2022, View Source [SID1234625271]).

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Nasdaq’s extension notice has no immediate effect on the continued listing status of the Company’s common stock on The Nasdaq Capital Market LLC under the trading symbol, "TLSA." If at any time during the additional 180-day extension, the bid price of the Company’s common stock closes at, or above, $1.00 per share for a minimum of ten (10) consecutive business days, the Company will regain compliance with this Nasdaq rule and this matter will be closed.

The Company was first notified by Nasdaq of its failure to maintain a minimum bid price of $1.00 per share under Rule 5550(a)(2) on June 14, 2022, and was given until December 12, 2022 to regain compliance. As part of its request for an extension, the Company transferred the listing of its common stock from the Nasdaq Global Market to the Nasdaq Capital Market.

The Company intends to monitor the closing bid price of its common stock between now and June 12, 2023, and has available options within the second compliance period to rectify the deficiency and regain compliance with the minimum bid price requirement. The Company’s common stock will continue to be listed and trade on the Nasdaq Capital Market during this period.

This announcement is made in compliance with Nasdaq Listing Rule 5810(b), which requires prompt disclosure of receipt of a deficiency notification.

On December 14, 2022 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reproted the closing of its previously announced underwritten public offering of 7,840,909 shares of its common stock at a price to the public of $22.00 per share (Press release, Syndax, DEC 14, 2022, View Source [SID1234625270]). This includes the exercise in full by the underwriters of their option to purchase up to 1,022,727 additional shares of common stock. The aggregate gross proceeds to Syndax from this offering were $172.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Syndax. Following the closing of the Offering, Syndax has 68,100,918 shares issued and outstanding as of December 14, 2022.

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Goldman Sachs & Co. LLC, J.P. Morgan and Cowen acted as joint book-running managers for the offering. BTIG and B. Riley Securities acted as co-lead managers for the offering.

The shares were offered pursuant to a "shelf" registration statement previously filed and declared effective by the Securities and Exchange Commission (SEC). A final prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC and are available on the SEC’s website located at View Source Copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained from: Goldman Sachs and Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing [email protected]; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (833) 297-2926, or by email at [email protected].

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 14, 2022 Qu Biologics Inc., a clinical stage biopharmaceutical company developing Site Specific Immunomodulators (SSIs), a novel platform of immunotherapies designed to restore innate immune function, reported a new collaboration with Associate Professor Jonas Fuxe and his research team at the Karolinska Institute (KI) in Stockholm, Sweden, to characterize the molecular targets of Qu’s SSI therapy at the tissue level that lead to resolution of disease (Press release, Qu Biologics, DEC 14, 2022, View Source;utm_medium=rss&utm_campaign=qu-biologics-and-karolinska-institute-initiate-important-new-collaboration-to-validate-molecular-mechanisms-of-qu-biologics-first-in-class-immunotherapy-platform [SID1234625268]). This collaboration will include assessment of tissue biopsies from the gastrointestinal (GI) tract of patients with moderate-to-severe Crohn’s disease (CD) who experienced histological healing upon treatment with QBECO, Qu’s GI-targeting SSI.

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Epithelial cells on mucosal surfaces are an important immune interface between microbial invaders and human tissue. Loss of epithelial barrier function is commonly seen in diseases associated with chronic inflammation and can be exceedingly challenging to resolve. Compromised intestinal epithelial barrier function is present in inflammatory bowel diseases (IBD), such as ulcerative colitis and Crohn’s disease, which are currently treated with lifelong aggressive immune suppressive medication to manage symptoms. Despite the arsenal of immune suppressive medication used to treat IBD, most patients experience disease progression. QBECO SSI treatment, which is designed to restore normal innate immune function, immune homeostasis, and barrier function in the GI tract, provides a novel potentially transformative alternative to immunosuppressive therapies for patients living with IBD.

Associate Professor Fuxe, acting Head of the Division of Pathology and from January 1, 2023, Head of the Department of Laboratory Medicine at KI, is a renowned scientist in epithelial cell biology. Qu’s collaboration with his team will initially focus on validating, at the tissue level, data demonstrating that the expression of genes associated with damaging, unproductive inflammation that disrupt epithelial barrier function can be normalized in the GI tract of patients with moderate-to-severe Crohn’s disease upon treatment with QBECO SSI. This partnership is anticipated to further understanding of epithelial integrity and plasticity in the context of immune dysfunction and how SSI treatment helps restore barrier function through targeting innate immune modulation in the GI tract. In addition, this collaboration may help identify patients, based on the tissue profiling of their disease, who may benefit the most from SSI treatment for inflammatory conditions associated with a loss of barrier function, such as Crohn’s disease. Such an advance would enable precision medicine for patients living with CD.

Both the Fuxe team at KI and Qu’s science team are excited about the scientific and medical potential of this collaboration. Dr. Jonas Fuxe states, "We have a long-standing interest in identifying mechanisms and targets that may prevent loss of epithelial integrity in chronic inflammatory and cancer diseases. We are excited by the collaboration with Qu Biologics, which timely opens new avenues to explore links between epithelial and immune dysfunction." Immunologist Dr. Shirin Kalyan, VP Scientific Innovation of Qu Biologics, notes, "Molecular characterization of how QBECO SSI leads to disease resolution in the GI tract in patients with IBD has the potential to identify which patients are most likely to benefit from this innovative approach to restore gastrointestinal health, which could be life-changing for these patients. We are very excited to be working with Associate Professor Fuxe and his team at KI to address this critically important issue." Dr. Hal Gunn, CEO of Qu Biologics, adds, "Given KI’s reputation and high regard in the field of immunology, we are very pleased to be collaborating with Dr. Fuxe and his team on this important project."

On December 14, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on Panbela’s application for orphan designation of ivospemin (SBP-101) in combination with gemcitabine and nabPaclitaxel in patients with metastatic pancreatic ductal adenocarcinoma (PDA) (Press release, Panbela Therapeutics, DEC 14, 2022, View Source;utm_medium=rss&utm_campaign=panbela-receives-positive-ema-opinion-on-orphan-designation-for-ivospemin-sbp-101-in-combination-with-gemcitabine-and-nab-paclitaxel-in-patients-with-metastatic-pancreatic-ductal-adenocarcinoma [SID1234625267]). Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to SBP-101.

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"We are pleased to receive a notification of positive opinion for orphan drug designation from EMA’s Committee Orphan Medicinal Products following its December plenary meeting," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "The opinion is timely, as we only a few weeks ago announced first patients enrolled in Europe in our ASPIRE trial. We continue to advance the global clinical trial, as we aim for interim analysis in early 2024."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives, including a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.

Panbela is continuing to focus on site initiation and enrollment to ultimately deliver a more effective treatment for pancreatic cancer, a deadly disease with few treatment options. The Company expects that a significant number of global sites will be open by year-end with the full complement of sites open by early-to-mid 2023.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101

Ivospemin Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. For more information, please visit View Source

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigatorinitiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.