KayoThera, Inc. Strengthens Financial Position with $5.2 Million in Grant and Expanded Series A Funding, Bringing Total Financial Support to $14 Million

On July 19, 2023 KayoThera, Inc. ("KayoThera"), an early-stage therapeutics company developing first-in-class, oral, small molecule inhibitors of the retinoid pathway, reported the strengthening of its financial resources through multiple grant awards and expansion of its previously announced Series A financing (Press release, KayoThera, JUL 19, 2023, View Source [SID1234633315]). The expanded financing was led by Accelerator Life Science Partners (ALSP), who also provided Series A funding for KayoThera. With the additional $5.2 million in new equity investments, non-dilutive grant funding, and initial seed funding from the New Jersey Health Foundation, KayoThera’s total financial support to date is $14 million.

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KayoThera has received grant funding from the National Institutes of Health Small Business Innovation Research (SBIR) program1,2 and the Andy Hill Cancer Research Endowment (CARE) Fund3. In addition, KayoThera secured additional Series A financing in an expansion round led by ALSP that also included participation from BioAdvance, Pier 70 Ventures, and WRF Capital. The additional resources secured by KayoThera will further support the Company’s therapeutic development programs.

"The grant awards are important validation of our development programs, and the expanded Series A financing reflects our investor’s confidence in our ability to innovate oral, small molecule inhibitors of the retinoid pathway," said Mark Esposito, Ph.D., Vice President, R&D, and Co-Founder of KayoThera. "With the additional financial resources announced today, we are well-positioned to advance our lead diabetes and oncology programs, each of which has first-in-class potential in disease indications with large patient populations and significant unmet medical need."

"Our belief that KayoThera offers a compelling investment opportunity and has the potential to transform the treatment of serious metabolic diseases and cancer was the key driver for our initial investment in the Company," said Thong Q. Le, Senior Managing Director of ALSP and CEO of KayoThera. "Since the initial Series A financing in March 2022, KayoThera has made significant progress in advancing its core technology and development programs. The additional funding announced today will allow the Company to continue making exciting advances in innovating small molecule inhibitors of the retinoid pathway and position itself for strategic partnering activity that will further help realize the commercial and clinical value of its science."

The retinoid pathway is known to play critical roles in several serious diseases. For example, the clinical use of retinoid activators leads to both adverse cardiometabolic events such as hyperlipidemias as well as increased cancer rates and faster cancer progression. KayoThera’s unique approach to drugging this pathway is based on biology discovered at Princeton University and represents the first platform to create drug-like retinoid inhibitors.

References
1 NIH NIDDK (National Institute of Diabetes and Digestive and Kidney Diseases) R43DK136420. Grant title: Development of first-in-class antagonists of the retinoid pathway as novel oral therapies for Type 2 Diabetes.
2NIH NCI (National Cancer Institute) R43CA278127. Grant title: Development of first-in-class antagonists of the retinoid pathway as novel oral immunotherapies for solid cancers.
3The Andy Hill Cancer Research Endowment (CARE) Fund FY23-LS-05. Grant title: Development of a First-in-Class Immunotherapy to Treat Advanced Solid Tumors Through IND-Enabling Safety Studies.

The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

Karyopharm Announces Updated Exploratory Subgroup Analysis from SIENDO Study in Patients with Advanced or Recurrent TP53 Wild-Type Endometrial Cancer to be Presented at ASCO Plenary Series on July 25th

On July 19, 2023 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that an updated exploratory subgroup analysis from the SIENDO study (NCT03555422) in patients with advanced or recurrent TP53 wild-type endometrial cancer will be presented at the virtual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Plenary Series (Press release, Karyopharm, JUL 19, 2023, View Source [SID1234633314]). The SIENDO exploratory subgroup data provides further support for the ongoing pivotal Phase 3 study of selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer (XPORT-EC-042; NCT05611931).

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ASCO Plenary Series Program

Title: Long-term follow up of selinexor maintenance in patients with TP53wt advanced or recurrent endometrial cancer: A pre-specified subgroup analysis from the phase 3 ENGOT-EN5/GOG-3055/SIENDO study.
Presenter: Brian Slomovitz, MD, Mount Sinai Medical Center
Session Date and Time: Tuesday July 25, 2023, 3:00pm – 4:00pm (ET)

This livestream event presented by ASCO (Free ASCO Whitepaper) is free to register at:
View Source

About the EC-042 Study

EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind study evaluating selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. The EC-042 Study was initiated in November 2022 and is expected to enroll up to 220 patients who will be randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The primary endpoint of the study is progression free survival (PFS), as assessed by an investigator with overall survival as a key secondary endpoint. Further, in connection with the EC-042 Study, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type.

About the SIENDO Study

Karyopharm’s evaluation of selinexor to treat patients with TP53 wild-type advanced or recurrent endometrial cancer is supported by data from an exploratory subgroup analysis from its ongoing SIENDO Study. The SIENDO Study (ENGOT-EN5/GOG-3055) is a multicenter, randomized, double-blinded Phase 3 study evaluating the efficacy and safety of oral selinexor versus placebo as a front-line maintenance therapy in patients with advanced or recurrent endometrial cancer following at least one prior platinum-based combination chemotherapy treatment (NCT03555422). Patients in this study with advanced or recurrent disease who had a partial response or a complete response after at least 12 weeks of taxane-platinum combination chemotherapy were randomized 2:1 to receive either maintenance therapy of 80 mg of selinexor or placebo taken once per week, until disease progression or death. The primary endpoint in the study is PFS from time of randomization until death or disease progression as assessed by an investigator, and prespecified exploratory endpoints included evaluation by p53 status and other molecular subtypes. Early data was presented at European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Plenary and Society for Gynecologic Oncology (SGO) meetings in 2022 and will be reported in future publications.

About Endometrial Cancer

Endometrial cancer is the most common cancer of the female reproductive organs in the U.S., with approximately 66,000 new cases expected in 2023 leading to nearly 13,000 deaths.1 In 2020, there were approximately 130,000 new cases and 29,000 deaths in Europe from endometrial cancer, while on a global scale there were 417,000 new cases and approximately 97,000 deaths.2 Since 2002, the incidence of new cases and deaths from endometrial cancer have risen.3 Risk factors include obesity, Type 2 diabetes, high-fat diets, use of tamoxifen and oral estrogens, and delayed menopause.4 There are no approved therapies in the maintenance setting for patients with advanced or recurrent endometrial cancer. 5

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, China, South Korea, Canada, Israel and Taiwan. XPOVIO and NEXPOVIO is marketed by Karyopharm’s partners, Antengene, Menarini, Neopharm and FORUS, in China, South Korea, Singapore, Australia, Hong Kong, Germany, Austria, Israel and Canada.

Please refer to the local Prescribing Information for full details.

Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

SELECT IMPORTANT SAFETY INFORMATION
Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1–888–209–9326 or FDA at 1–800–FDA–1088 or www.fda.gov/medwatch.

Genprex Announces $7.5 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules

On July 19, 2023 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that it has entered into definitive agreements with healthcare-focused institutional investors for the sale and issuance of 7,425,744 shares of the Company’s common stock and warrants to purchase up to 7,425,744 shares of common stock at a combined offering price of $1.01 per share of common stock and accompanying warrant, in a registered direct offering priced at-the-market under the Nasdaq rules (Press release, Genprex, JUL 19, 2023, View Source [SID1234633313]). The warrants have an exercise price of $0.885 per share, will become exercisable commencing the date of issuance, and will expire five years following the issuance date. The closing of the offering is expected to occur on or about July 21, 2023, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds to Genprex from this offering are expected to be approximately $7.5 million, before deducting the placement agent’s fees and other offering expenses. Genprex intends to use the net proceeds from this offering for working capital and general corporate purposes.

A "shelf" registration statement (File Number 333-271386) relating to the offered securities was filed with the Securities and Exchange Commission ("SEC") on April 21, 2023 and was declared effective on June 9, 2023. The offering of the securities is being made only by means of a prospectus, including a prospectus supplement, forming a part of an effective registration statement. A prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC. Electronic copies of the prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at www.sec.gov or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 856-5711 or e-mail at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, 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.

Circle Pharma Achieves Major Milestone with the Selection of a Clinical Development Candidate Directly Inhibiting Cyclins A and B

On July 19, 2023 Circle Pharma, a biopharmaceutical company advancing the discovery and development of intrinsically cell-permeable macrocycles, reported the selection of CID-078 for its first clinical development program (Press release, Circle Pharma, JUL 19, 2023, View Source [SID1234633312]). CID-078 is the first-and-only-in-class dual inhibitor of Cyclins A and B, which play essential roles in regulating cell cycle progression. Inhibiting Cyclins A and B selectively induces synthetic lethality in certain cancers exhibiting cell cycle dysregulation while sparing healthy cells.

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Circle Pharma recently presented pre-clinical data for its dual Cyclin A/B inhibitors at this year’s American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data demonstrated activity across a wide range of human tumor cell lines including tumor regression in xenograft models of small cell lung carcinoma (SCLC) and ovarian cancer. SCLC has a five-year survival rate of 3.5%, and its treatment has seen minimal progress in the last 30 years. Ovarian cancer patients commonly face poor outcomes and resistance to the current standard of care. In advance of human clinical trials for CID-078, further preclinical studies using patient-derived models (PDX) are underway in breast cancer (including triple negative breast cancer), non-small cell lung cancer, gastric cancer, pancreatic cancer, and other tumor types.

"The selection of CID-078 represents a major milestone for our team; we are excited to advance this molecule into clinical development and to realize its potential to bring new hope for the many cancer patients who currently have woefully inadequate treatment options," said David Earp, J.D., Ph.D., President and Chief Executive Officer of Circle Pharma. "This achievement is also a reflection of the capabilities of our macrocycle discovery platform as the cyclins have until now been considered undruggable."

ABOUT CID-078
CID-078 is an orally bioavailable macrocycle with dual cyclin A and B inhibitory activity that drives synthetic lethality in multiple tumor types. In biochemical and cellular studies, CID-078 has been shown to potently and selectively disrupt the protein-protein interaction between Cyclins A and B and their key substrates, including E2F (a substrate of Cyclin A) and Myt1 (a substrate of Cyclin B). Preclinical studies have demonstrated the ability of CID-078 to cause pronounced tumor regression in multiple xenograft models. Circle Pharma plans to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for CID-078 and initiate clinical development in 2024.

Cerus Corporation to Release Second Quarter 2023 Financial Results on August 2, 2023

On July 19, 2023 Cerus Corporation (Nasdaq: CERS) reported that its second quarter 2023 financial results will be released on Wednesday, August 2, 2023, after the close of the stock market (Press release, Cerus, JUL 19, 2023, View Source [SID1234633311]). The Company will host a conference call and webcast at 4:30 P.M. ET that afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook.

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To listen to the live webcast and view the presentation slides, please visit the Investor Relations page of the Cerus website at View Source Participants may register for the call here. While not required, interested participants are encouraged to join 10 minutes prior to the start of the event.

A replay will be available on Cerus’ website and will be available approximately three hours after the call through August 16, 2023.