On October 18, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported interim results from its U.S. Phase 1b/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma (STS) lung metastases, which documented preliminary clinical activity for Annamycin (Press release, Moleculin, OCT 18, 2021, View Source [SID1234591455]).

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The Phase 1b/2 study is a U.S. multi-center, open-label, single-arm study that in Phase 1b will determine the maximum tolerated dose (MTD) or the recommended Phase 2 dose (RP2D) and safety of Annamycin. The Phase 2 portion of the study will explore the efficacy of Annamycin as a single agent for the treatment of subjects with STS with lung metastases, who have failed prior chemotherapy, and for whom new chemotherapy is considered appropriate. A minimum of 3 subjects will be enrolled in each of up to 6 cohorts of the Phase 1b portion of the study, or until an MTD is identified, whichever comes first. A maximum of 25 subjects will be enrolled at the RP2D to further evaluate efficacy.

"To witness the activity of Annamycin in the treatment of STS lung metastases, even this early in a Phase 1 trial, we believe is encouraging. Four of the five patients that have completed scans to date demonstrated a response to treatment, including three with extended and, in one case, continuing stable disease and one patient with a substantial (>30%) reduction in tumor size. These interim data bolster our optimism about the potential for Annamycin to address the limitations with the current standard-of-care treatment options for STS lung metastases. Ultimately, we believe Annamycin has the potential to bring a new and effective treatment option to patients with a significant unmet need. With these data now in hand, we are cautiously optimistic as we begin patient enrollment and dosing in the next cohort," commented Walter Klemp, Chairman and CEO of Moleculin.

Mr. Klemp added, "We are also encouraged by the pace of recruitment so far in this trial. To have completed 2 full cohorts in just the first 4 months of the study with only one site open is faster than we would have expected, especially for a rare disease like STS lung metastases. And, with more sites joining the study before the end of the year, we believe there is the potential for this pace of recruitment to continue or even accelerate."

"As with all of the patients treated so far in our acute myeloid leukemia trials, no patients in this STS trial have exhibited any signs of cardiotoxicity," Mr. Klemp concluded. "This is an important point because, even though anthracyclines are considered a cornerstone chemotherapy for many types of cancer, including STS lung metastases, all currently approved anthracyclines are significantly cardiotoxic. Annamycin was designed to overcome this problem and we believe it has the potential to become the first non-cardiotoxic anthracycline approved for use. This could not only reduce the risk of many current anthracycline treatment regimens, but it could also enable longer treatment periods without cardiac risk."

The summary of interim data from the first two cohorts of the study are as follows:

First Cohort:

One subject is ongoing and is currently in cycle 5 of treatment and expects to enter cycle 6 of treatment at the end of this month. The end of cycle 4 scan revealed stable disease.
One subject discontinued after 6 cycles (4.5 months) due to progressive disease. However, stable disease was maintained up to that point.
One subject discontinued after cycle 1. The end of study scan was performed, and stable disease was observed. The subject was discontinued from the study because cycle 2 treatment was delayed greater than 6 weeks from the prior dosing.
Second Cohort:

One subject is ongoing and is currently in cycle 3 of treatment. The end of cycle 2 scan revealed partial response (PR, >30% reduction in tumor size).
One subject was discontinued from the study after 2 cycles after the end of cycle scan revealed progressive disease.
One subject received one cycle of treatment to-date and discontinued treatment for reasons unrelated to Annamycin. The end of study scans are scheduled in the coming weeks.
These are interim data and should be considered preliminary and subject to change.

The Company has now opened enrollment in the third cohort of the Phase 1b portion of the study. Three subjects minimum (6 maximum) for each dosing cohort will be enrolled until a maximum tolerated dose is identified. Therefore, up to 36 subjects may be enrolled in the Phase 1b portion of the study.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of STS lung metastases, in addition to Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia. For more information about the Phase 1b/2 study evaluating Annamycin for the treatment of STS lung metastases, please visit clinicaltrials.gov and reference identifier NCT04887298.

Study Design

In Phase 1b, Annamycin is administered as an intravenous (IV) infusion over 2 hours on Day 1, followed by 20 days off (1 cycle = 21 days). Subjects visit the study site every 21 days (±3 days) at which time safety monitoring, including adverse events (AEs), a physical examination, laboratory evaluations (clinical chemistry, complete blood count), vital signs, weight measurements, Eastern Cooperative Oncology Group (ECOG) performance status, and electrocardiograms (ECGs) will be performed, followed by an IV infusion of study drug. Additional laboratory evaluations (clinical chemistry, complete blood count) will be performed at 7 days (± 3 days) and 14 days (± 3 days) from the Annamycin infusion for additional safety monitoring during each cycle. Cardiac function will be followed by echocardiogram (ECHO) scans at screening, at the end of the first two cycles and then every other cycle thereafter, at the end of treatment visit, and if feasible, during follow up at 6 months (±1 month) and 1 year (±1 month) after study drug discontinuation. As long as the Investigator considers that the benefits of treatment with Annamycin continue to outweigh the risks, treatment will continue every 21 days until tumor progression is observed or unacceptable toxicity occurs.

Tumor response is monitored every 6 weeks (±1 week) from Cycle 1 Day 1 during treatment, at the End of Treatment visit, and then every 3 months (±1 month) until disease progression using RECIST criteria. Those subjects who leave the study after a maximum response is achieved and who do not start another therapy will be followed every 3 months (±1 month) for progression-free survival (PFS). If a subject receives further therapy after discontinuing from the study, they will only be followed for overall survival (OS) and if feasible, follow-up ECHO scans at 6 months (±1 month) and 1 year (±1 month) after study drug discontinuation.

About Annamycin

Annamycin is the Company’s next-generation anthracycline that has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of acute myeloid leukemia (AML), and the Company believes that the use of Annamycin may not face the same usage limitations imposed on doxorubicin. Annamycin is currently in development for the treatment of AML and STS lung metastases.

On October 18, 2021 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported it has entered into an exclusive licensing agreement with Antares Pharma, Inc. to commercialize TLANDO in the United States (Press release, Lipocine, OCT 18, 2021, View Source [SID1234591454]). TLANDO is an oral testosterone product for testosterone replacement therapy ("TRT") in adult males indicated for conditions associated with a deficiency or absence of endogenous testosterone: primary hypogonadism (congenital or acquired) and hypogonadotropic hypogonadism (congenital or acquired).

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As previously announced, the U.S. Food and Drug Administration ("FDA") granted tentative approval to TLANDO. Upon the expiration of the exclusivity period granted to Clarus Therapeutics, Inc., with respect to its drug, JATENZO, under the Hatch-Waxman Act, which expires on March 27, 2022, TLANDO will be eligible for final approval. The FDA has affirmed the NDA resubmission for final approval will be a Class 1 resubmission which includes a two-month FDA review goal period.

Under the terms of the agreement, Lipocine will receive an immediate upfront cash payment of $11.0 million and, subject to certain conditions, an additional $5.0 million licensing payment in January 2025 and another $5.0 million licensing payment in January 2026. Lipocine will also be entitled to receive sales-based commercial milestone payments totaling up to $160.0 million based on TLANDO net sales and, if Antares Pharma exercises its option, TLANDO XR sales, in addition to tiered royalty payments at rates ranging from the mid-teens to up to 20% on net sales of TLANDO. Lipocine retains all rights for ex-US territories, and non TRT indications for TLANDO. Under the agreement, Antares Pharma will undertake all commercialization, post-marketing obligations, and sourcing of TLANDO in the U.S.

In addition, Lipocine has granted Antares Pharma an option to license, on or before March 31, 2022, TLANDO XR, a next-generation, potential once-daily oral product candidate for TRT, in the U.S. TLANDO XR met primary and secondary regulatory end points for TRT in an earlier Phase 2 clinical study. Upon exercise of the TLANDO XR option, Lipocine is entitled to receive an additional $4.0 million in license fees, clinical and regulatory milestone payments of up to an aggregate of $35.0 million, and tiered royalties on net sales at rates ranging from the mid-teens to up to 20%. Antares Pharma will be responsible for development costs, regulatory filings, commercialization, and post-marketing commitments for TLANDO XR.

"We are very pleased to be partnering with Antares Pharma, a strong market leader with one of the largest sales forces in the TRT space," said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. Dr. Patel further stated, "Our agreement with Antares Pharma demonstrates our commitment to ensure efficient and effective patient access to TLANDO in the U.S. We are confident in Antares Pharma’s capabilities, given its established marketing experience and demonstrated success in the TRT space. Consistent with our current core competency, this agreement allows us to focus diligently on progressing our innovative pipeline candidates with the goal of serving patients with serious unmet needs."

On October 18, 2021 Geron Corporation (Nasdaq: GERN), a late-stage biopharmaceutical company focused on the development and commercialization of treatments for hematologic malignancies, reported the completion of patient enrollment in the IMerge Phase 3 clinical trial to evaluate imetelstat, a first-in-class telomerase inhibitor, in lower risk myelodysplastic syndromes (MDS) (Press release, Geron, OCT 18, 2021, View Source [SID1234591453]). Based upon current planning assumptions, Geron expects top-line results for IMerge Phase 3 to be available at the beginning of January 2023.

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"Completing patient enrollment in IMerge Phase 3 brings us one step closer to delivering imetelstat as a potential treatment alternative for patients with lower risk MDS who are relapsed or refractory to ESAs. Achieving durable transfusion independence remains a significant medical need for these patients," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "I would like to thank all of the patients and their families, the investigators, clinical site staff, as well as our employees for supporting the achievement of this important milestone."

Patients from the IMerge Phase 2 clinical trial achieved durable transfusion independence with imetelstat treatment, including transfusion-free periods greater than one year, irrespective of the disease subgroup, such as ringed sideroblast positive or ringed sideroblast negative. Such durability provides significant and meaningful clinical benefit to lower risk MDS patients given their chronic anemia and the debilitating impact of serial blood transfusions. In addition, depletion of cytogenetic abnormalities and reductions in key driver mutations associated with lower risk MDS were observed, and these results were also correlated with transfusion independence. Based on the IMerge Phase 2 data, taken together, the durability, molecular and cytogenetic data provide strong evidence for disease-modifying activity of imetelstat, which has the potential to differentiate it from other currently approved and investigational treatments in lower risk MDS today.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

For further information about IMerge Phase 3, visit ClinicalTrials.gov/NCT02598661.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of diverse blood disorders that develop because bone marrow cells do not mature into healthy blood cells. There are approximately 60,000 people living with the disease in the United States. Approximately 70% of MDS patients are categorized in the lower risk groups at diagnosis, according to the International Prognostic Scoring System that assigns relative risk of progression to acute myelogenous leukemia and overall survival by taking into account the presence of a number of disease factors, such as cytopenias and cytogenetics. The predominant clinical problem in lower risk MDS is chronic anemia, and many patients become dependent on red blood cell transfusions which leads to iron overload, heart and kidney complications, decreases in quality of life and shorter overall survival.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in myeloid hematologic malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk myelofibrosis whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

On October 18, 2021 The board of directors of Eli Lilly and Company (NYSE: LLY) reported that it has declared a dividend for the fourth quarter of 2021 of $0.85 per share on outstanding common stock (Press release, Eli Lilly, OCT 18, 2021, View Source [SID1234591452]).

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The dividend is payable on December 10, 2021 to shareholders of record at the close of business on November 15, 2021.

On October 18, 2021 Cullinan Oncology, Inc. (Nasdaq: CGEM) ("Cullinan" or "the Company"), a biopharmaceutical company focused on developing a diversified pipeline of targeted and immuno-oncology therapies, reported the appointment of Nadim Ahmed as President and Chief Executive Officer, effective today (Press release, Cullinan Oncology, OCT 18, 2021, View Source [SID1234591451]). Mr. Ahmed will also join the Company’s Board of Directors. Mr. Ahmed succeeds Owen Hughes, who resigned as CEO and as a member of the Company’s Board of Directors. Mr. Hughes will take on the role of Strategic Advisor to the company to support the transition.

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"Owen has successfully built-out Cullinan’s organization and pipeline since its inception, while also leading the Company through multiple capital raises, including its IPO earlier this year," said Anthony Rosenberg, Chairman of the Board of Directors at Cullinan. "Cullinan, via both internal discovery and external collaboration, has constructed a diverse portfolio of novel oncology programs that are advancing to later stages of development. Nadim brings to Cullinan a proven track record in oncology drug development and commercialization success. His experience at Celgene during a period of tremendous growth will be invaluable to Cullinan as we advance Pearl into later-stage clinical development, while also moving our first two immuno-oncology programs, MICA and Florentine, into clinical trials by year-end 2021. We will provide an update for Pearl in Q4 2021 as originally planned. On behalf of Cullinan, I want to welcome Nadim to Cullinan and thank Owen for his contributions. We look forward to him supporting the transition."

"I am excited to be leading the incredibly talented team at Cullinan to advance our deep, multi-modality pipeline as we strive to bring important medicines to cancer patients," said Mr. Ahmed. "With a world-class leadership team in place and a robust cash position, Cullinan is well-poised to execute on its evolution into a company with multiple clinical-stage novel oncology programs."

Mr. Ahmed has more than twenty-five years of leadership experience in oncology across a range of development and commercialization roles, including recently at Bristol Myers Squibb (BMS), where he served as President, Hematology and at Celgene Corporation (Celgene) where he served as President, Global Hematology & Oncology through its acquisition by BMS. As President, Hematology at BMS, Mr. Ahmed oversaw multiple product launches and participated as a member of the company’s Leadership Team. Prior to BMS, Mr. Ahmed served in leadership roles at Celgene across both development and commercialization functions. During this time at Celgene, he successfully launched multiple cancer medicines, including Pomalyst, Abraxane and several key indications for Revlimid. Mr. Ahmed has worked with various treatment modalities, including small molecules, biologics and cell therapy in hematology and solid tumors. Mr. Ahmed holds a Master of Science degree from Loughborough University, UK and a Bachelor of Science degree from University College London, UK.