On March 30, 2022 Vyant Bio, Inc. ("Vyant Bio", "Company") (Nasdaq: VYNT), is an innovative biotechnology company reported that reinventing drug discovery for complex neurodevelopmental and neurodegenerative disorders (Press release, Vyant Bio, MAR 30, 2022, View Source [SID1234611198]). The Company’s central nervous system ("CNS") drug discovery platform combines human-derived organoid models of brain disease, scaled biology, and machine learning. Vyant Bio’s current programs are focused on identifying repurposed and novel small molecule clinical candidates for rare CNS genetic disorders including Rett Syndrome ("Rett"), CDKL5 Deficiency Disorders ("CDD") and familial Parkinson’s Disease ("PD"). The Company’s approach to drug discovery focuses on de-risking candidate selection, thereby improving the known effectiveness of drugs discovered earlier in the development cycle. Today, Vyant Bio will report its Fourth Quarter and Year-End 2021 highlights and business updates in a conference call and webcast scheduled for 8:30am ET.

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"Vyant Bio is committed to transforming into a pure-play drug discovery and development biotech company. We are incorporating the use of quantitative biomarkers that we believe are unique to our Rett and CDD programs that should provide meaningful human-biology-derived, preclinical signals of potential drug efficacy before the commencement of clinical trials", stated Robert T. Fremeau PhD, the Company’s Chief Scientific Officer.

"We believe our drug discovery approaches will demonstrate a capital efficient and effective way to identify novel and repurposed therapeutics to treat patients with severe neurological conditions and will thereby maximize value to our shareholders," stated Jay Roberts, Chief Executive Officer of Vyant Bio.

Vyant Bio filed its Year-End 2021 on Form 10-K today with the Securities and Exchange Commission. Please refer to Vyant Bio’s Form 10-K for more detailed information with respect to our financial results for the year ended December 31, 2021.

YEAR-END 2021 FINANCIAL RESULTS

Cash and cash equivalents totaled $20.6 million as of December 31, 2021.

During the fourth quarter of 2021, we commenced the process to sell the vivoPharm business. Therefore the vivoPharm business is classified as a held-for-sale asset and its financial information as discontinuing operations as of and for the year ended December 31, 2021. Continuing operations as of and for the year ended December 31, 2021 consists of Vyant Bio and StemoniX, Inc.

Revenue from continuing operations for 2021 was $1.1 million as compared with $867 thousand in 2020. Cost of goods sold – service aggregated $408 thousand in 2021 and $384 thousand in 2020. Cost of goods sold – product aggregated $1.4 million and $717 thousand for the year ended December 31, 2021 and 2020, respectively. Substantially all of our revenue from continuing operations was generated from our Maple Grove facility. As we move this facility to internal research and development activities in 2021, the costs for this facility will have a corresponding shift to research and development costs in 2022.

Research and development costs for 2021 increased to $4.3 million as compared with $3.2 million in 2020 which was the result of increased headcount, lab and professional service costs. Selling, general and administrative expenses increased from $2.7 million in 2020 to $8.4 million in 2021 which reflects post-merger public company costs.

Overall, the net loss of $8.7 million in 2020 increased to $40.9 million in 2021. The 2021 net loss includes $26.0 million of non-cash expenses as well as $2.3 million of merger related expenses. The net loss from continuing operations aggregated $18.6 million and included non-cash expenses of $4.8 million as well as $2.3 million in merger related costs. The net loss of $22.3 million from discontinuing operations included a $20.2 million goodwill impairment charge and other non-cash expenses aggregating approximately $1.0 million.

Vyant Bio’s Conference Call and Webcast and Information

Vyant Bio’s management will host a conference call on Wednesday, March 30, 2022 at 8:30am ET to discuss the Fourth Quarter and Year End 2021 results and provide strategic business updates as well as answer questions. Event information is below:

Event: Investor Conference Call and Webcast for the Fourth Quarter and Year-End 2021
Date: Wednesday, March 30, 2022
Time: 8:30am ET
Dial In: Toll Free: 1.877.545.0523 Conference ID: 449263
Webcast: View Source

The live event will be recorded and available for replay. The conference call and webcast details are also included inside the Investors section of the Vyant Bio corporate website at www.vyantbio.com.

On March 30, 2022 Aravive, Inc. (Nasdaq: ARAV), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, reported that it has entered into definitive agreements with a single healthcare-focused institutional investor and Eshelman Ventures, LLC for the issuance and sale of an aggregate of 4,850,241 shares of its common stock (or common stock equivalents in lieu thereof) and warrants to purchase up to an aggregate of 4,850,241 shares of common stock in a registered direct offering priced at-the-market under Nasdaq rules (Press release, Aravive, MAR 30, 2022, View Source [SID1234611196]). The purchase price per share and accompanying warrant is $2.005 for the institutional investor and $2.325 for Eshelman Ventures, LLC.

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

The warrants to be issued to the institutional investor will be immediately exercisable, will expire five years following the issuance date and will have an exercise price of $1.88 per share. The warrants to be issued to Eshelman Ventures, LLC will be exercisable upon the approval by the stockholders of the Company of previously issued securities, will expire five years following the issuance date and will have an exercise price of $2.20 per share.

The Company’s Executive Chairman, Fredric N. Eshelman, Pharm.D., is the founder of Eshelman Ventures, LLC.

The closing of the offering is expected to occur on or about March 31, 2022, subject to the satisfaction of customary closing conditions. The gross proceeds from the offering are expected to be approximately $10 million. The Company intends to use the net proceeds from the offering to continue clinical development of batiraxcept in platinum resistant ovarian cancer and clear cell renal cell carcinoma, and for general corporate purposes.

The offering of the securities described above is being made only by means of a prospectus supplement and accompanying base prospectus. The Company has filed a shelf registration on Form S-3 (File No. 333-248612) (including a base prospectus) with the U.S. Securities and Exchange Commission ("SEC"), which was declared effective on November 20, 2020. A final prospectus supplement and accompanying base prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website, located at www.sec.gov. Electronic copies of the prospectus supplement and the accompanying base prospectus for the offering may also be obtained, when available, by contacting H.C. Wainwright & Co., LLC, at 430 Park Ave., 3rd Floor, New York, New York 10022, by telephone at (212) 856-5711, or by email at [email protected].

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

On March 30, 2022 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Alaunos Therapeutics, MAR 30, 2022, View Source [SID1234611195]).

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"Over the course of 2021 we successfully restructured to refocus Alaunos on advancing our novel TCR-T platform for the treatment of solid tumors," commented Kevin S. Boyle, Sr., Chief Executive Officer. "We are very pleased to announce that we’ve consented our first patient for treatment in our Phase 1/2 TCR-T Library trial at MD Anderson. We anticipate treating our first patient in the second quarter and will continue to focus in 2022 on generating meaningful clinical data."

James Huang, Chair of Alaunos, stated, "I am very pleased by the transformation of Alaunos over the past six months under Kevin’s leadership. As we prepare to enter the clinic, I am confident in his ability to lead the Company through its next phase of development. I believe that now is the right time for me to transition from Executive Chair to Chair of the board. I look forward to our continued collaboration on the board to guide Alaunos towards success."

Recent Developments and Upcoming Milestones

TCR-T Library Clinical Program: In January 2022, the Company announced that its Phase 1/2 TCR-T Library trial targeting KRAS, TP53, and EGFR mutations across six solid tumor indications is open for enrollment. The Company also announced that it had amended the study’s investigational new drug (IND) application to include an additional four TCRs, for a total of 10 evaluable TCRs across KRAS, TP53, and EGFR mutations.

The study will be conducted at MD Anderson Cancer Center and will be an open label, dose escalation study, with patients to be treated in one of three dosing cohorts: 5 x 109, 40 x 109, or 10 x 1010 TCR-T cells. The trial will enroll patients with non-small cell lung, colorectal, endometrial, pancreatic, ovarian, and bile duct cancers that have a matching human leukocyte antigen (HLA) and hotspot mutation pairing. The primary endpoint of the study is to identify the maximum tolerated dose or recommended phase 2 dose. The Company has consented the first patient and expects to dose the first patient in the second quarter of 2022. Additional information about the study is available at www.clinicaltrials.gov using the identifier: NCT05194735.

human neoantigen T-cell Receptor Platform (hunTR): The Company’s robust and innovative TCR discovery engine has successfully identified proprietary TCRs under this platform. With further development and testing, the Company intends to further expand its TCR library with a selected group of these proprietary TCRs.

Filed patent for mbIL-15: In February 2022, the Company filed an international patent application related to its membrane bound IL-15 TCR T cell program, which covers vectors expressing mbIL-15 with TCRs that target hotspot mutations in solid tumors, including KRAS, TP53 and EGFR. The Company plans to showcase new preclinical data from this program at a major scientific conference later this year. The Company intends to file an IND application for this program in 2023.

Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute: In March 2022, the Company announced that it had extended its CRADA with the NCI for the evaluation of its TCR-T cells targeting solid tumors, which was established in January 2017. The agreement has been extended through January 9, 2023 and will focus on evaluating Alaunos’ TCR-T Library in a personalized TCR program.

Fourth Quarter Ended December 31, 2021 Financial Results

Research and Development Expenses: Research and development expenses were $8.2 million for the fourth quarter of 2021, compared to $14.0 million for the fourth quarter of 2020, a decrease of approximately 41%. The decrease was primarily due to reduced employee related expenses and lower program-related costs as a result of the winding down of the Company’s Controlled IL-12 and CAR-T programs.

General and Administrative Expenses: General and administrative expenses were $2.1 million for the fourth quarter of 2021, compared to $8.8 million for the fourth quarter of 2020, a decrease of approximately 76%. The decrease was primarily due to reduced professional services and employee related expenses.

Net Loss: Net loss was $11.8 million, or $(0.05) per share, for the fourth quarter of 2021, compared to a net loss of $22.8 million, or $(0.11) per share, for the same period in 2020.

Cash and Cash Equivalents: As of December 31, 2021, Alaunos had approximately $76.1 million in cash and cash equivalents. The Company anticipates its cash runway will be sufficient to fund operations into the second quarter of 2023.

Full Year 2021 Financial Results

Research and Development Expenses: Research and development expenses were $49.6 million for the full year ended December 31, 2021, compared to $52.7 million for the full year ended December 31, 2020. The decrease in research and development expenses was primarily due to reduced program-related costs of $9.2 million as a result of the winding down of the Company’s Controlled IL-12 and CAR-T programs. The decrease was partially offset by an increase of $4.3 million in employee related expenses, including a $2.2 million severance charge related to the Company’s strategic restructuring in the third quarter of 2021, and a $1.8 million increase in facilities and other related expenses primarily related to our expanded facilities in Houston.

General and Administrative Expenses: General and administrative expenses were $27.6 million for the full year ended December 31, 2021, compared to $27.7 million for the full year ended December 31, 2020. The decrease in general and administrative expenses was primarily due to reduced professional services of $4.4 million, partially offset by a $4.3 million increase in employee related expenses, including a $1.3 million severance charge related to the Company’s strategic restructuring in the third quarter of 2021.

Net loss: Net loss was $78.8 million, or $(0.37) per share for the full year ended December 31, 2021, compared to a net loss of $80 million, or $(0.38) per share for the full year ended December 31, 2020.

Conference Call and Webcast

Alaunos will host a conference call and webcast today, March 30, 2022 at 8:30am ET. Participants should dial 844-309-0618 (United States) or 661-378-9465 (International) with the conference code 9091406. A live webcast may be accessed using the link here, or by visiting the "Investors" section of the Alaunos website at www.alaunos.com. After the live webcast, the event will be archived on Alaunos’ website for approximately 90 days after the call.

On March 30, 2022 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the Phase III SKYSCRAPER-02 study, evaluating the investigational anti-TIGIT immunotherapy tiragolumab plus Tecentriq (atezolizumab) and chemotherapy (carboplatin and etoposide) as an initial (first-line) treatment for people with extensive-stage small cell lung cancer (ES-SCLC), did not meet its co-primary endpoint of progression-free survival (Press release, Genentech, MAR 30, 2022, View Source [SID1234611194]). The co-primary endpoint of overall survival was not met at its interim analysis and is unlikely to reach statistical significance at the planned final analysis. Data suggest tiragolumab plus Tecentriq and chemotherapy was well-tolerated and no new safety signals were identified when adding tiragolumab. Data will be presented at an upcoming medical meeting.

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"Today’s outcome is disappointing as we had hoped to continue building on the advances of Tecentriq in extensive stage small-cell lung cancer, which remains difficult to treat. We are thankful to all the patients and healthcare professionals involved in the study," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "We look forward to seeing additional data from the upcoming Phase III trial in PD-L1-high non-small cell lung cancer based on the encouraging results from the CITYSCAPE study."

SCLC is the most aggressive form of any lung cancer and is characterized by rapid progression and poor survival. Tecentriq was the first cancer immunotherapy to show a survival benefit in ES-SCLC (Phase III IMpower133 study), and was the first approved treatment option in 20 years. More options are needed, particularly for hard-to-treat cancers like SCLC, and Genentech is committed to exploring innovative medicines to improve outcomes for people with lung cancer.

The tiragolumab program continues to explore advances in multiple clinical trials to build on Tecentriq, expand into earlier stages of disease, and seeks to provide new treatment options in advanced and difficult-to-treat cancers with high unmet medical need. Tiragolumab was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration in 2021 for the initial treatment of PD-L1-high metastatic non-small cell lung cancer, based on the results of the Phase II CITYSCAPE study – representing the only investigational anti-TIGIT therapy to be granted this designation. The Phase III SKYSCRAPER-01 trial is currently ongoing to confirm the CITYSCAPE results. Since 2020, Genentech has initiated five Phase III trials, including NSCLC (SKYSCRAPER-01, SKYSCRAPER-03), ES-SCLC (SKYSCRAPER-02), esophageal cancers (SKYSCRAPER-07, SKYSCRAPER-08), and multiple early trials in various tumor types.

About the SKYSCRAPER-02 study

SKYSCRAPER-02 is a global Phase III, randomized, placebo-controlled and double-blinded study evaluating tiragolumab plus Tecentriq (atezolizumab) and chemotherapy as an initial (first-line) treatment versus Tecentriq and chemotherapy alone in 490 people with extensive-stage small cell lung cancer. Co-primary endpoints are overall survival (OS) and progression-free survival (PFS) in the primary analysis set (all randomized patients whose cancer had not spread to the brain). Key secondary endpoints include OS and PFS in all randomized patients, and safety.

About tiragolumab

Tiragolumab is a novel immune checkpoint inhibitor with an intact Fc region. Tiragolumab selectively binds to TIGIT, a novel inhibitory immune checkpoint, which suppresses the immune response to cancer. Based on preclinical research, tiragolumab is thought to work as an immune amplifier with other cancer immunotherapies such as Tecentriq (atezolizumab). The TIGIT pathway is distinct but complementary to the PD-L1/PD-1 pathway. Dual blockade with tiragolumab and Tecentriq may help overcome immune suppression and restore the immune response.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called small cell lung cancer (SCLC). TECENTRIQ may be used with the chemotherapy medicines carboplatin and etoposide as your first treatment when your lung cancer:

TECENTRIQ may be used alone as a treatment for their lung cancer:
is a type called "extensive-stage small cell lung cancer," which means that it has spread or grown.
It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues in any area of the body and can affect the way they work. These problems can sometimes become severe or life threatening and can lead to death. Patients can have more than one of these problems at the same time. These problems may happen anytime during their treatment or even after their treatment has ended.

Patients should call or see their healthcare provider right away if they develop any new or worse signs or symptoms, including:

Lung problems

cough
shortness of breath
chest pain
Intestinal problems

diarrhea (loose stools) or more frequent bowel movements than usual
stools that are black, tarry, sticky, or have blood or mucus
severe stomach-area (abdomen) pain or tenderness
Liver problems

yellowing of the skin or the whites of the eyes
severe nausea or vomiting
pain on the right side of their stomach area (abdomen)
dark urine (tea colored)
bleeding or bruising more easily than normal
Hormone gland problems

headaches that will not go away or unusual headaches
eye sensitivity to light
eye problems
rapid heartbeat
increased sweating
extreme tiredness
weight gain or weight loss
feeling more hungry or thirsty than usual
urinating more often than usual
hair loss
feeling cold
constipation
their voice gets deeper
dizziness or fainting
changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness
Kidney problems

decrease in their amount of urine
blood in their urine
swelling of their ankles
loss of appetite
Skin problems

rash
itching
skin blistering or peeling
painful sores or ulcers in mouth or nose, throat, or genital area
fever or flu-like symptoms
swollen lymph nodes
Problems can also happen in other organs.

These are not all of the signs and symptoms of immune system problems that can happen with Tecentriq. Patients should call or see their healthcare provider right away for any new or worse signs or symptoms, including:

Chest pain, irregular heartbeat, shortness of breath, or swelling of ankles
Confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs
Double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight
Persistent or severe muscle pain or weakness, muscle cramps
Low red blood cells, bruising
Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include:

chills or shaking
itching or rash
flushing
shortness of breath or wheezing
dizziness
feeling like passing out
fever
back or neck pain
Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if patients undergo transplantation either before or after being treated with Tecentriq. A healthcare provider will monitor for these complications.

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider will check patients for these problems during their treatment with Tecentriq. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may also need to delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus
have received an organ transplant
have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)
have received radiation treatment to their chest area
have a condition that affects their nervous system, such as myasthenia gravis or Guillain-Barré syndrome
are pregnant or plan to become pregnant. Tecentriq can harm an unborn baby. Patients should tell their healthcare provider right away if they become pregnant or think they may be pregnant during treatment with Tecentriq. Females who are able to become pregnant:
A healthcare provider should do a pregnancy test before they start treatment with Tecentriq
They should use an effective method of birth control during their treatment and for at least 5 months after the last dose of Tecentriq
are breastfeeding or plan to breastfeed. It is not known if Tecentriq passes into the breast milk. Patients should not breastfeed during treatment and for at least 5 months after the last dose of Tecentriq
Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

feeling tired or weak
decreased appetite
nausea
cough
shortness of breath
The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

feeling tired or weak
nausea
hair loss
constipation
diarrhea
decreased appetite
Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information about the benefits and side effects of Tecentriq.

Report side effects to the FDA at 1-800-FDA-1088 or View Source

Report side effects to Genentech at 1-888-835-2555.

Please see the Tecentriq full Prescribing Information, including the Medication Guide, for additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentech’s approved PD-L1 checkpoint inhibitor, the company’s broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit View Source

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

On March 30, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial stage company engaged in the discovery, development and sale of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the hormone cortisol, reported overall survival ("OS") data from its 178-patient, randomized, controlled, Phase 2 study of relacorilant plus nab-paclitaxel in patients with recurrent, platinum-resistant ovarian cancer (Press release, Corcept Therapeutics, MAR 30, 2022, https://ir.corcept.com/news-releases/news-release-details/relacorilant-plus-nab-paclitaxel-extends-survival-women [SID1234611193]).

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Women treated with relacorilant the day before, the day of and the day after their regular nab-paclitaxel infusions (the trial’s "intermittent" arm) experienced a 33% reduction in risk of death compared to women treated with nab-paclitaxel alone (hazard ratio: 0.67; p-value: 0.066). Their median OS was 13.9 months, compared to 12.2 months for women receiving nab-paclitaxel monotherapy. (See Figure 1)

Corcept Therapeutics Incorporated

The women who enrolled in this trial were very sick and had experienced disease progression following prior therapy (median number of prior therapies: three). By chance, more women with either primary platinum-refractory disease or who had already received four or more prior lines of therapy, both indicators of a very poor prognosis, were assigned to the intermittent arm. As is typical of late-stage clinical trials, such women will not be enrolled in Corcept’s upcoming Phase 3 trial.

Excluding primary platinum-refractory patients and women who had already received four or more prior lines of therapy, women treated with relacorilant intermittently experienced a 48% reduced risk of death compared to women treated with nab-paclitaxel alone (hazard ratio: 0.52; p-value: 0.010). Their median OS was 13.9 months, compared to 12.2 months for women receiving nab-paclitaxel monotherapy. (See Figure 2)

Corcept Therapeutics Incorporated

"Corcept has introduced a novel oncologic therapeutic platform, cortisol modulation. These results constitute a potentially important medical advance," said Thomas Herzog, MD, Deputy Director at the University of Cincinnati Cancer Center, member of the Board of Directors of the Gynecologic Oncology Group (GOG) Foundation and Associate Director of GOG Partners. "In this large, randomized study, women with recurrent, platinum-resistant ovarian cancer who were administered relacorilant at the time they received nab-paclitaxel exhibited meaningful improvements in progression free survival, duration of response and overall survival – without increased side effects – compared to women who received nab-paclitaxel alone. For this patient population, relacorilant plus nab-paclitaxel has the potential to become a new standard of care."

"We are excited to receive these survival data, which have continued to improve as the trial has progressed," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "If our Phase 3 trial replicates the results in progression free survival, duration of response and overall survival that we’ve seen in Phase 2, it will be an unprecedented success for patients with ovarian cancer. No approved therapy has been shown to significantly extend survival compared to standard chemotherapy in women with platinum-resistant ovarian cancer. We plan to meet with the FDA as soon as possible to define the best path forward and to open our Phase 3 trial in the second quarter of 2022."

On March 31, 2022, Corcept will host an event for investors and analysts regarding its ovarian cancer program. Click here to register. In addition, the company plans to present these results at a major oncology conference in 2022.

About Platinum-Resistant Ovarian Cancer

Ovarian cancer is the fifth most common cause of cancer death in women.1 Patients whose disease returns less than six months after receiving platinum-containing therapy are described as having "platinum-resistant" disease. In the United States, approximately 20,000 women with platinum-resistant disease are candidates to start a new therapy each year.2 There are few treatment options and median overall survival following recurrence of disease is 12 months or less with single-agent chemotherapy.3 No approved therapy has been shown to significantly extend overall survival in patients with recurrent, platinum-resistant ovarian cancer compared to standard chemotherapy.4

About Corcept’s Ovarian Cancer Program

The data in this release come from Corcept’s 178-patient, randomized, controlled, Phase 2 trial of relacorilant plus nab-paclitaxel in patients with recurrent, platinum-resistant ovarian cancer.5 The women who entered the trial had experienced disease progression on prior lines of therapy. The median number of prior treatments was three.

Study participants were randomized 1:1:1 to receive either (i) nab-paclitaxel plus 150 mg of relacorilant given the day before, the day of, and the day after each weekly nab-paclitaxel infusion ("intermittent" arm), (ii) nab-paclitaxel plus 100 mg relacorilant given daily ("continuous" arm) or (iii) nab-paclitaxel alone ("comparator" arm).

OS was assessed after a pre-determined number of patient deaths had occurred. At the time of database cutoff, 128 of the 178 women who enrolled in the study had died. Fourteen additional women in the intermittent arm and nine additional women in the comparator arm are expected to contribute to the final overall survival results. One woman in the intermittent arm and one in the continuous arm have yet to experience tumor progression – for both patients it has been over twenty months since they initiated therapy with relacorilant.

Relative to women in the comparator arm, women in the intermittent arm experienced significantly improved progression free survival (PFS) (median PFS: 5.6 months versus 3.8 months; hazard ratio: 0.66; p-value: 0.038) and duration of response (DoR) (median DoR: 5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006). They also lived longer (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.67; p-value: 0.066). Safety and tolerability of relacorilant plus nab-paclitaxel was comparable to nab-paclitaxel monotherapy. (See Table 1)

Phase 2 Trial Results – Intermittent and Comparator Arms (All Patients)
Intermittent Comparator Intermittent vs Comparator
Median PFS* (95% CI) 5.6 months 3.8 months Hazard Ratio: 0.66 (0.44, 0.98)
p-value: 0.038
Median DoR* (95% CI) 5.6 months 3.7 months Hazard Ratio: 0.36 (0.16, 0.77)
p-value: 0.006
Median OS* (95% CI) 13.9 months 12.2 months Hazard Ratio: 0.67 (0.43, 1.03)
p-value: 0.066
Table 1: Summary of results of intermittent and comparator arms – all patients.
*PFS: progression free survival; OS: overall survival; DoR: duration of response.

Excluding women with primary platinum-refractory disease and patients who had received four or more prior lines of treatment, women in the intermittent arm experienced significantly improved PFS (median PFS: 5.6 months versus 3.8 months, hazard ratio: 0.58; p-value: 0.016) and OS relative to women in the comparator arm (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.52; p-value: 0.010). The women in the intermittent arm also experienced a significant improvement in DoR relative to those in the comparator arm (median DoR: 5.6 months versus 3.6 months, hazard ratio: 0.26; p-value: 0.001). Safety and tolerability of relacorilant plus nab-paclitaxel was comparable to nab-paclitaxel monotherapy. (See Table 2)

Phase 2 Trial Results – Intermittent and Comparator Arms (Planned Phase 3 Patient Population)
Intermittent Comparator Intermittent vs Comparator
Median PFS* (95% CI) 5.6 months 3.8 months Hazard Ratio: 0.58 (0.37, 0.91)
p-value: 0.016
Median DoR* (95% CI) 5.6 months 3.6 months Hazard Ratio: 0.26 (0.11, 0.62)
p-value: 0.001
Median OS* (95% CI) 13.9 months 12.2 months Hazard Ratio: 0.52 (0.31, 0.86)
p-value: 0.010
Table 2: Summary of results of intermittent and comparator arms – excluding patients with primary platinum-refractory disease and patients who had received four or more prior lines of treatment.
*PFS: progression free survival; OS: overall survival; DoR: duration of response.

About Corcept’s Oncology Programs

There is substantial evidence that cortisol activity at the glucocorticoid receptor ("GR") allows certain solid tumors to resist treatment and that modulating cortisol’s activity may help anti-cancer treatments achieve their intended effect.

Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy. In some cancers, cortisol inhibits cellular apoptosis – the tumor-killing effect many treatments are meant to stimulate. In other cancers, cortisol activity promotes tumor growth. Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers.

Corcept is conducting clinical trials of its proprietary selective cortisol modulators in combination with three different anti-cancer treatments in patients with ovarian, adrenal and prostate cancers. Corcept’s first controlled study in oncology – relacorilant plus nab-paclitaxel for the treatment of patients with ovarian cancer – has demonstrated statistically significant and clinically meaningful results.

About Relacorilant

Relacorilant is a non-steroidal, selective glucocorticoid receptor modulator that does not bind to the body’s other hormone receptors. Corcept is studying relacorilant in a variety of serious disorders, including ovarian and adrenal cancer and Cushing’s syndrome. Relacorilant is proprietary to Corcept and is protected by composition of matter and method of use patents, as well as orphan drug designation in the United States for the treatment of pancreatic cancer and both the United States and the European Union for the treatment of Cushing’s syndrome.