On April 14, 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 that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPD) to its p-STAT3 inhibitor, WP1066, for the treatment of ependymoma (Press release, Moleculin, APR 14, 2021, View Source [SID1234578018]).

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

Ependymoma is a rare type of tumor that can form in the brain or spinal cord. Ependymoma begins in the ependymal cells in the brain and spinal cord that line the passageways where the fluid (cerebrospinal fluid) that nourishes your brain flows. Ependymoma can occur at any age, but most often occurs in young children.

"The FDA’s recognition of the high prevalence and unmet needs in the treatment landscape for ependymoma, especially in pediatric patients is a significant milestone as we continue to advance and expand the WP1066 development program. We currently have Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as RPD designation for three other pediatric indications, and believe that ependymoma represents another important rare indication. We continue to be encouraged by the data WP1066 has demonstrated to date and believe it has the potential to be an effective therapy for pediatric patients with ependymoma," commented Walter Klemp, Chairman and CEO of Moleculin.

The Rare Pediatric Disease Priority Review Voucher program, which was created as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, is intended to incentivize the development of new therapies for rare pediatric diseases. Under the FDA’s rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a "rare pediatric disease," which is defined as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. Subject to FDA approval of WP1066 for the treatment of ependymoma, Moleculin would be eligible to receive a voucher that may be redeemed to receive priority review for a subsequent marketing application for a different product candidate or which could be sold or transferred.

About WP1066

WP1066 is an immune/transcription modulator capable of directly inhibiting certain key oncogenic transcription factors, including the activated form of a protein known as STAT3. The activated form of STAT3 referred to as p-STAT3, is considered a master regulator of tumor activity. In addition to inhibiting p-STAT3 and several other signaling proteins linked to tumor development, WP1066 has also been shown in animal models to stimulate a natural immune response and immune memory to fight tumor progression.

On April 14, 2021 Theratechnologies Inc. (Theratechnologies, or Company) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported business highlights and financial results for the first quarter ended February 28, 2021 (Q1 Fiscal 2021) (Press release, Theratechnologies, APR 14, 2021, View Source [SID1234578039]).

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"Theratechnologies is at a unique point in its evolution, and we have a tremendous opportunity ahead of us," said Paul Levesque, President and Chief Executive Officer at Theratechnologies. "We are particularly pleased with the swift advancement of our Phase 1 oncology program, which has dosed its first patient, and the continued progress we have made toward advancing our Phase 3 trial in NASH.

"We believe we are well-positioned for growth when we begin to emerge from the COVID-19 pandemic. In line with these efforts is our continued focus on strengthening our business with the addition of key talent and resources. While first-quarter revenues were relatively flat year-over-year, we believe that we have aligned our organization to provide an optimal landscape to further validate and drive value for our commercial and R&D opportunities," concluded Mr. Levesque.

On April 14, 2021 GeneLeap Bio., a subsidiary of Luye Life Sciences specializing in the development of nucleic acid therapeutics, reported that an ePoster entitled ‘Hydrogel encapsulated TLR9 agonists show sustained tumor growth inhibition and prolong survival of CT26 tumor-bearing mice’ at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Meeting (Press release, GeneLeap Bio, APR 14, 2021, View Source [SID1234578058]). The preclinical study shows that compared with using TLR9 agonist on its own, a hydrogel encapsulated TLR9 agonist, independently developed by GeneLeap Biotech, can significantly improve antitumor performance, while demonstrating tumor growth inhibitory effects similar to that of TLR9 agonist without hydrogel encapsulation.

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AACR Annual Meeting is one of the world’s biggest and most eminent conferences related to cancer research, covering the latest discoveries and drug developments across the spectrum of cancers and highlighting the work of the best minds in research and medicine from institutions all over the world. The meeting was held virtually this year, from April 10 to 15 and May 17 to 21.

TLR9 is one of the most promising targets in current cancer immunotherapies, whose agonist has emerged as a potential monotherapy or in combination with other anticancer therapies. As an innate immune stimulator, TLR9 agonist can be combined with checkpoint inhibitors to increase its anti-tumor effects. By activating the innate immune system, it provides increased immunological impetus against checkpoint inhibitor resistant and refractor tumors.

However, there are certain drawbacks in the clinical application of TLR9 agonist, such as increased toxicity seen with systemic administration. Local injection of the TLR9 agonist is well-tolerated but the drug can degrade in the local tissues or dissipate from the site of action rapidly, resulting in reduced efficacy. Thus, in such cases a weekly or biweekly dosing of TLR9 agonist is usually required to maintain the anti-tumor efficacy.

To address this medical need, GeneLeap Biotech has made a breakthrough by encapsulating TLR9 agonists in injectable hydrogels containing poloxamer for a slow and sustained release of TLR9 agonist over a period of time. GeneLeap’s researchers have conducted preclinical studies in a mouse tumor model to examine whether a single dose of Hydrogel encapsulated TLR9 agonist can be effective over a longer period in immune activation without toxicity.

The current preclinical study showed that a single administration of hydrogel encapsulated TLR9 agonist demonstrates tumor growth inhibition and non-toxicity similar to that of a multiple dose of TLR9 agonist without hydrogel encapsulation. This indicates that for those patients who are resistant or refractory to checkpoint inhibitors, hydrogel encapsulated TLR9 agonist could enhance the immune response to checkpoint inhibitors for a longer time. "Based on the positive preclinical data obtained in a mouse tumor model, we believe this novel formulation – hydrogel encapsulation – will ease frequency of dosing schedules of TLR9 agonists in clinic," said Dr. Jason Zhang, CSO of GeneLeap Biotech.

This preclinical study is also of pioneering significance in exploring further applications of hydrogel formulations. "To our knowledge, this is the first study using hydrogel encapsulation to improve the frequency of dosing of TLR9 agonists in cancer patients," said CEO of GeneLeap Biotech, Dr. Sean Fu. "It helps us enhance understanding of hydrogel formulations and has laid a good foundation for more in-depth clinical research in the area of therapeutic modalities including mRNA and oligonucleotides. As a pharmaceutical biotech focusing on frontier science, GeneLeap Biotech is committed to promoting pioneering science, developing nucleic-acid drugs for tumors and severe infectious diseases, and providing patients with innovative treatments to meet their health needs."

On April 14, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that HOOKIPA’s management team will participate and host 1:1 meetings at the virtual Kempen Life Science Conference, taking place April 21, 2021 (Press release, Hookipa Pharma, APR 14, 2021, View Source [SID1234578019]).

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Additional information will be available within the Investors & Media section of HOOKIPA’s website at View Source

On April 14, 2021 Veracyte (Nasdaq: VCYT) reported a key milestone in its companion diagnostics program with Acerta Pharma, the hematology research and development arm of AstraZeneca (LSE/STO/NYSE: AZN) (Press release, Veracyte, APR 14, 2021, View Source [SID1234578040]). The first patient has been enrolled and randomized in Acerta Pharma’s Phase 3 ESCALADE trial, which is using Veracyte’s LymphMark lymphoma subtyping test to identify patients with untreated diffuse large B-cell lymphoma (DLBCL) who may benefit from Acerta and AstraZeneca’s acalabrutinib (Calquence) in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) therapy.

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The randomized, double-blind, placebo-controlled ESCALADE study is designed to evaluate the efficacy and safety of acalabrutinib with R-CHOP therapy as compared to placebo plus R-CHOP in patients aged 18-65. The study is expected to enroll up to 600 participants at centers around the world. Study investigators are using the investigational LymphMark genomic test to select patients with non-germinal center B-cell (non-GCB) subtype DLBCL, an aggressive form of the disease that is associated with worse outcomes following the traditional chemoimmunotherapy regimen (R-CHOP).

"The initiation of this global study marks an important step in our multi-year companion diagnostics program with Acerta Pharma," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "By distinguishing DLBCL patients based on the genomic underpinning of their disease, we believe the LymphMark test will help identify patients early on who are more likely to respond to targeted therapies such as Calquence that may improve outcomes."

DLBCL patients respond to treatment differently based on the molecular subtype of their tumors. The LymphMark test uses gene-expression profiling of RNA extracted from surgical tissue to classify the "cell of origin" subtype of DLBCL tumors. The World Health Organization recommends gene-expression profiling for patients with DLBCL, which may potentially be mitigated by more specific treatments that are under development.i By identifying the specific subtypes in individual patients’ tumors, physicians can help ensure the right patients are included in clinical trials evaluating these therapies.

Non-Hodgkin lymphoma (NHL) ranks among the top-10 common cancers worldwide, with over 500,000 new cases estimated in 2018.ii DLBCL is the most common type of NHL, accounting for approximately 30 percent of lymphomas.iii

The LymphMark test is intended for use on the nCounter Analysis System. Veracyte acquired the LymphMark test, as well as the exclusive global diagnostic rights to the nCounter system, in December 2019.