On May 6, 2021 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of cortisol, reported positive results from its 178-patient, controlled, Phase 2 trial of relacorilant plus nab-paclitaxel in patients with recurrent platinum-resistant ovarian cancer (Press release, Corcept Therapeutics, MAY 6, 2021, https://ir.corcept.com/news-releases/news-release-details/addition-relacorilant-nab-paclitaxel-improves-progression-free [SID1234579334]).

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Women with platinum resistant ovarian cancer (n=60) who received 150 mg of relacorilant the day before, the day of and the day after their weekly nab-paclitaxel infusion exhibited a statistically significant improvement in progression free survival (hazard ratio 0.66, p-value 0.038) compared to women (n=60) who received nab-paclitaxel monotherapy; their median progression free survival was 1.8 months longer (5.6 vs 3.8 months). Safety data and tolerability data for the two groups were comparable. (See Figure 1)

A photo accompanying this announcement is available at View Source

A third cohort of patients (n=58) received a daily dose of relacorilant (100 mg per day, with titration to 150 mg per day at the investigator’s discretion) in addition to nab-paclitaxel. These patients’ median progression-free survival increased 1.5 months compared to the control arm, to 5.3 months, but this improvement did not reach statistical significance (hazard ratio 0.83).

"It’s impressive how this therapy extends the benefits of taxanes," said Professor Pamela Munster, MD, Director of the University of California San Francisco’s Early Phase Clinical Trials Unit and Co-leader of its Center for BRCA Research. "Patients with platinum-resistant ovarian cancer have few good treatment options. A therapy that significantly extends time to tumor progression without additional side effect burden would be an important advance."

Corcept has begun planning a pivotal Phase 3 trial.

About the Trial

This controlled, multi-center, Phase 2 trial enrolled 178 patients with platinum-resistant ovarian cancer in one of three study arms. Sixty patients received 150 mg relacorilant the day before, the day of and the day after their infusion of nab-paclitaxel. Fifty-eight patients received 100 mg relacorilant every day, with titration up to 150 mg permitted at the investigator’s discretion. The sixty patients in the control arm received nab-paclitaxel alone. All patients received nab-paclitaxel on Days 1, 8 and 15 of each 28-day cycle. Patients in the control arm received 100 mg/m2 of nab-paclitaxel. Because relacorilant increases nab-paclitaxel plasma levels, the nab-paclitaxel dose in patients receiving relacorilant was 80 mg/m2. The trial’s primary endpoint was progression free survival. Secondary endpoints include overall survival, objective response rate and duration of response. The trial is being conducted at 28 sites in the United States and Europe. Additional information about the study (NCT03776812) can be obtained at www.ClinicalTrials.gov.

About Relacorilant

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

On May 6, 2021 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for the quarter ended March 31, 2021 and highlighted recent corporate progress (Press release, MEI Pharma, MAY 6, 2021, View Source [SID1234579350]).

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"The first several months of 2021 have been very eventful for MEI, highlighted by the completion of enrollment in the follicular lymphoma efficacy population arm of the zandelisib TIDAL study. In addition, we recently reported preclinical data at AACR (Free AACR Whitepaper) 2021, demonstrating the ability of voruciclib to downregulate MYC and synergize with KRAS inhibitors in KRAS mutant cancers," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "While we continue to work diligently to advance the clinical development of voruciclib and ME-344, we anticipate additional important milestones from the zandelisib program this calendar year, including top-line TIDAL data by the end of 2021, the initiation of our Phase 3 COASTAL study evaluating zandelisib in combination with rituximab in patients with second line follicular or marginal zone lymphomas expected to start around mid-year, and clinical data updates from the ongoing Phase 1b study at the ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper) and ICML annual meetings."

Anticipated Calendar Year 2021 Drug Candidate Pipeline Developments

Zandelisib – Oral PI3K delta inhibitor for the treatment of various B-cell malignancies

Reporting of topline data from the Phase 2 TIDAL study in the fourth quarter from the follicular lymphoma primary efficacy population. The complete data from the follicular lymphoma arm of the Phase 2 TIDAL study data are intended to be submitted to FDA to support an accelerated approval application.
Initiation around mid-2021 of enrollment in COASTAL, a Phase 3 study evaluating zandelisib in combination with rituximab in follicular and marginal zone lymphoma patients who received one or more prior lines of treatment. This study is intended to support FDA approval for additional indications and act as the required confirmatory study for the potential accelerated approval of zandelisib in patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma.
Clinical data updates from the Phase 1b study of zandelisib at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and European Hematology Association (EHA) (Free EHA Whitepaper) annual meetings, including the combination with zanubrutinib.
Voruciclib – CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia

Program updates, including data from the Phase 1 program evaluating voruciclib in patients with acute myeloid leukemia and B-cell malignancies.
ME-344 – Tumor selective mitochondrial inhibitor

Initiation of a Phase 2 pilot study of ME-344 in solid tumors in the first half of calendar 2022.
Recent and Third Quarter Fiscal Year 2021 Corporate Highlights

In April 2021, MEI completed enrollment in the follicular lymphoma primary efficacy population of the global Phase 2 TIDAL study evaluating zandelisib. Topline data from the study is on track to be reported in the fourth quarter. The complete Phase 2 TIDAL study data are intended to be submitted to FDA to support accelerated approval applications.

In April 2021, MEI reported preclinical data demonstrating that voruciclib, an orally administered cyclin-dependent kinase (CDK) inhibitor that is potent against CDK9, downregulates MYC by inhibiting MYC transcription and stabilization, and synergizes with KRAS inhibitors in KRAS mutant cancers. The research was featured as an E-Poster Session presentation titled, "Voruciclib, a CDK9 inhibitor, downregulates MYC and inhibits proliferation of KRAS mutant cancers in preclinical models" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021.

In January 2021, MEI announced that the Phase 1b trial arm exploring zandelisib in combination with zanubrutinib in collaboration with BeiGene, Ltd. completed the dose optimization stage in patients with B-cell malignancies and is expanding into disease specific B-cell malignancy cohorts. The Safety Review Committee recommended moving forward with a dosing regimen found to be generally well tolerated and active following a planned safety analysis.
Third Quarter Fiscal Year 2021 Financial Results

As of March 31, 2021, MEI had $164.6 million in cash, cash equivalents, and short-term investments with no outstanding debt.
For the quarter ended March 31, 2021, cash used in operations was $15.6 million, compared to $10.3 million for the same period in 2020. The increase in cash used in operations primarily relates to costs associated with our clinical development programs. For the nine months ended March 31, 2021, cash used in operations was $20.7 million, compared to $34.9 million for the same period in 2020. The year-to-date decrease in cash used in operations reflects $20.9 million of cash received from the Japanese taxing authorities as a refund of withholding tax associated with the Kyowa Kirin commercialization agreement signed in April 2019, offset by increased costs associated with our clinical development programs.
Research and development expenses were $17.9 million for the quarter ended March 31, 2021, compared to $9.0 million for the quarter ended March 31, 2020. The increase was primarily related to increased development costs associated with zandelisib, including increased activity in the TIDAL study and start-up costs related to the Phase 3 study, as well as increased personnel costs to support clinical trial activities.
General and administrative expenses were $6.2 million for the quarter ended March 31, 2021, compared to $3.9 million for the quarter ended March 31, 2020. The increase primarily relates to personnel costs and general corporate expenses incurred during the quarter ended March 31, 2021.
MEI recognized revenues of $2.4 million for the quarter ended March 31, 2021, compared to $1.2 million for the quarter ended March 31, 2020. The increase in revenue primarily related to the license agreement with Kyowa Kirin and included the recognition of fees allocated to research and development obligations.
Net loss was $31.3 million, or $0.28 per share, for the quarter ended March 31, 2021, compared to net loss of $4.3 million, or $0.04 per share for the quarter ended March 31, 2020. The Company had 112,591,778 shares of common stock outstanding as of March 31, 2021, compared with 105,998,677 shares as of March 31, 2020.
The adjusted net loss for the quarter ended March 31, 2021, excluding non-cash expenses related to changes in the fair value of the warrants (a non-GAAP measure), was $22.0 million, compared to an adjusted net loss of $12.1 million for the quarter ended March 31, 2020.

On May 6, 2021 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the first quarter of 2021 and recent corporate updates (Press release, Mirati, MAY 6, 2021, View Source [SID1234579367]).

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"We are pleased to see the significant progress across our clinical pipeline, including sitravatinib and our novel KRASG12C inhibitor, adagrasib. We initiated several potentially registration-enabling studies evaluating adagrasib in lung and colorectal cancers, as monotherapy and in combination with other therapies," said Charles M. Baum, M.D., Ph.D., president and chief executive officer, Mirati Therapeutics, Inc. "We are excited to submit our first NDA with adagrasib in the second half of this year for patients with non-small cell lung cancer who have a KRASG12C mutation."

Baum added, "Mirati continues to advance an innovative pipeline of drug candidates. The company expects to file IND applications for two potentially first-in-class therapies – our KRASG12D inhibitor, MRTX1133, in 2022, and a synthetic lethal MTA cooperative PRMT5 inhibitor, in the first half of 2022."

Pipeline Updates

Adagrasib

Initiated two randomized registration-enabling Phase 3 trials, KRYSTAL-10 and KRYSTAL-12, evaluating adagrasib with cetuximab (ERBITUX)1 versus chemotherapy in patients with second-line colorectal cancer (CRC), and a confirmatory trial of adagrasib versus docetaxel in patients with previously treated non-small cell lung cancer (NSCLC), respectively.
Initiated a potentially registration-enabling Phase 2 cohort in the KRYSTAL-01 study evaluating adagrasib as a monotherapy in patients with first-line NSCLC who have an STK11/KRASG12C co-mutation.
Initiated a potentially registration-enabling Phase 2 cohort in the KRYSTAL-01 study evaluating adagrasib as a monotherapy in patients with CRC who have received three or more lines of therapy.
The Company expects to share updated monotherapy data in the second half of 2021 from the registration-enabling Phase 1/2 KRYSTAL-01 study, evaluating adagrasib as a monotherapy in NSCLC, and Phase 1 data in CRC.
The Company expects to share initial proof-of-concept combination data in the second half of 2021 from the Phase 1/2 KRYSTAL-01 study, evaluating adagrasib with pembrolizumab (KEYTRUDA)2 in NSCLC, and with cetuximab (ERBITUX) in CRC.
The Company expects to provide an update in the first half of 2022 on additional combination data in NSCLC, including adagrasib plus a SHP2 inhibitor.
Sitravatinib

Continued to enroll patients in the Phase 3 SAPPHIRE clinical trial evaluating sitravatinib plus nivolumab (OPDIVO)3 in second or third line non-squamous NSCLC.
Preclinical

Continued to evaluate the optimal formulation and drug delivery approach for the potential first-in-class investigational KRASG12D inhibitor, MRTX1133, to improve the therapeutic exposure for patients with KRASG12D mutations. The Company expects to file an IND application for MRTX1133 in 2022.
Announced initial preclinical results evaluating the Company’s investigational synthetic lethal PRMT5 inhibitor program in methylthioadenosine phosphorylase (MTAP)-deleted cancer models at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The Company expects to file an IND application for this program in the first half of 2022. (View Release)
Corporate Updates

Announced the Company will contribute a $4 million grant to Stand Up To Cancer to develop new approaches to treat patients with KRAS mutant cancers. (View Release)
Announced Mirati Board of Directors elected a new independent director, Ms. Shalini Sharp. (View Release)
Ended the first quarter with approximately $1.3 billion in cash, cash equivalents, and short-term investments.
Financial Results for the First Quarter 2021

Research and development expenses for the first quarter of 2021 were $104.1 million, compared to $71.7 million for the same period in 2020. The increase in research and development expenses is primarily due to an increase in expense associated with the development of adagrasib, an increase in preclinical and early discovery activities, as well as an increase in salaries and other employee-related expense, which includes an increase in share-based compensation expense. The Company recognized research and development-related share-based compensation expenses of $14.5 million during the first quarter of 2021, compared to $11.8 million for the same period in 2020.
General and administrative expenses for the first quarter of 2021 were $28.4 million, compared to $18.0 million for the same period in 2020. The increase is primarily due to an increase in sponsorship agreements expense, professional service expense, and salaries and other employee-related expenses. The Company recognized general and administrative-related share-based compensation expenses of $10.2 million in the first quarter of 2021, compared to $9.7 million for the same period in 2020.
Net loss for the first quarter of 2021 was $135.7 million, or $2.67 per share basic and diluted, compared to a net loss of $86.7 million, or $2.02 per share basic and diluted for the same period in 2020.
Cash, cash equivalents, and short-term investments were $1.3 billion at March 31, 2021.

On May 6, 2021 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplants to more patients, reported financial results for the first quarter ended March 31, 2021 and recent program highlights (Press release, Magenta Therapeutics, MAY 6, 2021, View Source [SID1234579383]).

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"Magenta continues to make progress across our program portfolio, building momentum towards several key anticipated clinical and data milestones throughout 2021, including the initial Phase 2 trial results evaluating MGTA-145 plus plerixafor in Multiple Myeloma patients in an autologous transplant setting and filing an IND for first-in-human Phase 1/2 clinical trial of MGTA-117, our first product candidate from our targeted conditioning platform," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "We are optimistic about our programs’ ability to transform clinical practice of stem cell transplants and gene therapies with new first-line medicines, which we believe could increase access to curative transplants across multiple disease areas."

Program Highlights:

MGTA-145 Upcoming Scientific Conference Presentations:

Magenta will present preliminary MGTA-145 Phase 2 data in Multiple Myeloma patients at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held virtually June 4-8, 2021, and at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, to be held virtually June 9-17, 2021.
MGTA-145 Stem Cell Mobilization and Collection

Recent and Planned Activity:

Phase 2 clinical trial in Multiple Myeloma is ongoing at Stanford University, evaluating the ability of MGTA-145, in combination with plerixafor, to mobilize stem cells for collection prior to autologous stem cell transplant. This 25-patient, investigator-initiated trial will also measure engraftment and disease outcome measures, with stem cell mobilization as the primary endpoint. Preliminary results will be presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) and final clinical data from this trial are expected in the second half of 2021.
Phase 2 clinical trial in collaboration with the National Marrow Donor Program/Be The Match, evaluating MGTA-145, in combination with plerixafor, in the mobilization and collection of stem cells from allogeneic donors for transplant in patients with Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL) and Myelodysplastic Syndromes (MDS). Initial data from this trial are expected in the second half of 2021.
Initiate Phase 2 clinical trial in Sickle Cell Disease in collaboration with bluebird bio in the second half of 2021 to evaluate the utility of MGTA-145, in combination with plerixafor, for the mobilization and collection of stem cells in patients with Sickle Cell Disease where mobilization and collection is difficult and there is a clear unmet medical need.

MGTA-117 and CD45-ADC Targeted Conditioning Programs

Recent and Planned Activity:

MGTA-117 Investigational New Drug (IND) filing anticipated mid-2021. If the IND is accepted by the FDA, Magenta plans to initiate a Phase 1/2 clinical trial evaluating MGTA-117 in patients with AML and MDS. Magenta expects to assess initial safety and pharmacokinetic data internally in the fourth quarter of 2021, and also expects to be able to provide an update regarding the study’s progress within the dose escalation study design.
Magenta recently completed its GLP toxicology studies, its GMP manufacturing process and has finished its pre-IND communications with the FDA.
Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2021, were $132.3 million, compared to $148.8 million as of December 31, 2020. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the first quarter of 2023.

Research and Development Expenses: Research and development expenses were $11.7 million in the first quarter of 2021, compared to $14.0 million in the first quarter of 2020. The decrease was driven primarily by the completion of the GMP manufacturing campaign to support the upcoming IND for MGTA-117, and the discontinuation of the Phase 2 trial of MGTA-456 in inherited metabolic diseases in June 2020.

General and Administrative Expenses: General and administrative expenses were $7.0 million for the first quarter of 2021, compared to $7.3 million for the first quarter of 2020.

Net Loss: Net loss was $17.5 million for the first quarter of 2021, compared to net loss of $20.0 million for the first quarter of 2020.

On May 6, 2021 ITM AG, a leading radiopharmaceutical company, reported the establishment of a Chinese subsidiary, ITM (Shanghai) Precision Medical Co., Ltd., in Shanghai, China (Press release, ITM Isotopen Technologien Munchen, MAY 6, 2021, View Source [SID1234579401]). The new subsidiary will be part of the ITM Group as a wholly foreign-owned entity (WFOE) to further expand ITM’s growing operations in China, which is a key emerging market for novel radiomolecular precision oncology. The ITM Shanghai team will especially focus on expanding and accelerating clinical development, regulatory filings and access of targeted therapeutic and diagnostic radiopharmaceuticals for Chinese patients. ITM’s Chinese subsidiary will further facilitate and extend dialogue with local authorities and industry partners across China to help facilitate and grow the access to Targeted Radionuclide Diagnostics and Therapies.

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The foundation of the Chinese ITM subsidiary is an important step in ITM’s long-term China market access strategy which was initiated over a decade ago through the partnership with China Isotope & Radiation Corporation (CIRC) for supply and production of ITM’s medical radioisotopes and radiolabeling equipment. The strategic collaboration with CIRC was further expanded by a technology license agreement, signed in November 2019 enabling CIRC’s production and distribution of Germanium-68/Gallium-68 Generators and no-carrier-added Lutetium-177 (EndolucinBeta) in China based on established global standards. This expansion of the ITM/CIRC collaboration was a critical step towards securing and scaling the supply of theranostic medical radioisotopes as a robust basis for the clinical development and commercialization of targeted radiopharmaceutical imaging and therapy products in China.

"Cancer remains the leading cause of death in China creating a high demand for innovative, precise and economically viable treatment options. We are proud that we have been able to play a part in laying the foundation for radiomolecular precision oncology in China over the past decade. Together with CIRC, now joining the Processing and Production Network, we will be able to provide security of supply and scalability of medical radioisotopes for the exponentially growing use of targeted radiopharmaceuticals in precision oncology," said Steffen Schuster, Chief Executive Officer of ITM. "The demand in China in particular has substantially increased, especially due to the strong efforts that have been made by the Chinese Government and the medical industry to exponentially grow PET/CT installations for diagnostic purposes in recent years. Our new subsidiary will further facilitate the clinical development of radiomolecular imaging solutions and therapies for Chinese patients and is a testament to ITM’s long-term commitment to the evolution of radiomolecular precision oncology in China."

Mr. Schuster added: "It has always been our goal to build a global collaboration network leveraging our diagnostic and therapeutic radiopharmaceutical portfolio in order to help cancer patients live longer and better for which the establishment of our ITM China presence is an important milestone."

ITM’s precision oncology pipeline combines its proprietary, highly pure radioisotopes with tumor-specific targeting molecules for a range of hard-to-treat cancer indications, such as neuroendocrine tumors, prostate cancer, glioblastoma, osteosarcoma and bone metastases, as well as folate receptor α positive tumors like lung, ovarian or breast cancer. The company’s lead candidate, no-carrier-added Lutetium-177-Edotreotide (n.c.a. 177Lu-Edotreotide) is currently being evaluated in a phase III clinical trial, COMPETE, in patients with neuroendocrine tumors of gastroenteric and pancreatic origin (GEP-NETs). By leveraging its radiomolecular precision oncology treatments, it is ITM’s stated goal to give cancer patients worldwide better answers than "maybe".