On July 25, 2019 Wayshine Biopharm, a clinical-stage pharmaceutical company, reported that its First-in-Class CNS penetrable EGFR/EGFRvIII inhibitor, namely WSD0922, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of glioma (including Glioblastoma and Anaplastic Astrocytoma) (Press release, Wayshine Biopharm, JUL 25, 2019, View Source [SID1234537798]). Orphan drug designation was created to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

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WSD0922 has previously received IND approval from FDA for the treatment of Glioblastoma, Anaplastic Astrocytoma and cancers with CNS metastasis patients. Phase I/IIA to evaluate safety, tolerability, pharmacokinetics and anti-tumor activity of WSD0922 is ongoing at Minnesota, Arizona and Florida, the three campuses of Mayo Clinic.

"Orphan Drug Designation by the FDA for glioma is another significant milestone in the WSD0922 development program," commented Dr. Wei Zhong, Ph.D., CEO and Founder of Wayshine Biopharm. " We are very pleased that the FDA has granted broader indication than the indication proposed (GBM and AA). Fast grant for orphan drug designation by the FDA based on encouraging preclinical data truly reflects our innovation and commitments and the clinical potential of WSD0922 has been recognized and endorsed by the FDA, for this substantial unmet medical need and expansion to lower grade brain cancer."

Orphan drug designation by the FDA is granted to promote the development of drugs that target diseases affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan drug designation qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, eligibility for orphan drug grants, seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, and a waiver of certain administrative fees.

On July 25, 2019 Cytokinetics, Incorporated (Nasdaq:CYTK) reported that it is scheduled to report second quarter results on August 8, 2019 at 4:00 PM Eastern Time (Press release, Cytokinetics, JUL 25, 2019, View Source [SID1234537787]). Following the announcement, Cytokinetics’ senior management will host a conference call at 4:30 PM Eastern Time to discuss operational and financial results and the company’s outlook for the future.

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The conference call will be simultaneously webcast and can be accessed from the homepage and in the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 1778276.

An archived replay of the webcast will be available via Cytokinetics’ website until August 15, 2019. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 1778276 from August 8, 2019 at 7:30 PM Eastern Time until August 15, 2019.

On July 25, 2019 Snap Bio, Inc. and Torrey Pines Investment (TPI) reported that their joint early drug discovery program within the framework of the i2020 Accelerator has resulted in achieving the projected milestone in developing several small molecule lead candidates for treatment of Hepatocellular carcinoma (HCC) and Hepatoblastoma (Press release, Snap Bio, JUL 25, 2019, View Source [SID1234537776]). The milestone is marked with an outstanding selectivity profile and promising overall animal data in rodents for the lead candidates.

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This timeline-driven achievement was enabled­­ through the synergistic efforts of TPI’s i2020 Accelerator network, which includes ChemDiv, Inc. with its pertinent technical life-sciences expertise and its unique integrated drug discovery platform.

"The i2020 Accelerator was created to help take early stage research programs with differentiated biology and established development paradigms to the level of advanced leads and clinical candidates," comments Ronald Demuth, President of Torrey Pines Investment. "We were excited to start working with Snap Bio, their unique platform that integrates structure-based drug design and advanced synthetic biology allows for scaffold modification to be taken to a new level with improved optimization for all the key properties of highly effective and selective treatments. Snap Bio has been a great fit for our i2020 Accelerator portfolio, and we are glad to be seeing the first fruits of our collaboration thanks to the collective efforts of our global net of partners, including ChemDiv."

"With the help of TPI and the i2020 Accelerator, Snap Bio has discovered novel bispecific inhibitors, opening the door to a platform that offers small molecules with unique combinations of well-defined activities," comments Mark Burk, President and CEO at Snap Bio. "Bispecific small molecules can simultaneously and selectively inhibit two mechanistically distinct targets, which can provide important polypharmacological benefits, such as higher efficacy, fewer side effects, and reduced drug resistance. In addition, relative to drug combination therapies, bispecific inhibitors can target multiple disease pathways without pharmacokinetic disparity, leading to easier, faster, cheaper, and lower risk clinical trials."

About Torrey Pines Investment and i2020

Torrey Pines Investment (San Diego, CA) is a specialty life-science investment firm that invests in development stage molecules, diagnostics, and therapeutics in areas of high unmet medical need. [View Source By rapidly de-risking novel science and enriching partner pipelines with best-in-class molecules, i2020 platform helps accelerate early stage drug discovery platforms towards advanced lead and clinical candidate level within a two-year framework. i2020 Accelerator’s vast scientific and global resource network tailored specifically to the needs of early drug development programs allows it to take on projects in a wide array of therapeutic areas, from immunology to infectious diseases and beyond. By successfully leveraging agile development principles and flexible partnering business models, i2020 Accelerator plans to create and invest in multiple small-molecule early drug discovery programs. [www.i2020accelerator.com]

On July 25, 2019 4D pharma plc (AIM: DDDD), a pharmaceutical company leading the development of Live Biotherapeutics, reported an update on its oncology programmes (Press release, 4d Pharma, JUL 25, 2019, View Source [SID1234537775]). New preclinical data on the Company’s oncology candidates, MRx0518 and MRx1299, were presented at 1st Microbiome Movement Oncology Response Summit in Boston, USA by Research Director Imke Mulder. A clinical progress update on MRx0518 was also provided by 4D’s Chief Scientific Officer, Alex Stevenson.

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MRx1299

MRx1299 is a second-generation Live Biotherapeutic identified by 4D’s MicroRx discovery platform. One of the target host pathways identified for this Live Biotherapeutic is its potent histone deacetylase (HDAC) inhibition mediated by bacterial short-chain fatty acids (SCFAs). HDAC inhibition is thought to have multiple modes of anti-cancer activity, acting on both tumour and immune cells. Treatment with MRx1299 and its metabolites gives cytotoxic T lymphocytes the ability to reduce tumour growth in preclinical cancer models.

MRx0518

0MRx0518, the Company’s lead Live Biotherapeutic candidate in oncology, induces strong innate and adaptive immune responses in vitro and in vivo. New data presented demonstrates the ability of orally administered MRx0518 to modulate the frequency of immune cell populations with anti-tumour activity in the gastrointestinal tract, systemically and in the tumour microenvironment, acting through signalling pathways known to drive anti-cancer immunity.

The Company has previously published data on the mechanism of action of MRx0518 (Lauté-Caly et al. 2019), demonstrating that the bacterial flagellin protein, FliC, strongly activates immunostimulatory NF-κB signalling, via the TLR5 receptor. Research presented at the conference indicates a role for additional host receptors, including TLR9, in mediating the immuno-stimulatory effects of MRx0518.

Alex Stevenson, 4D’s Chief Scientific Officer, commented, "The identification of our new Live Biotherapeutic candidate, MRx1299, strengthens our leading oncology portfolio and expands the scope of oncology indications for our Live Biotherapeutics. MRx1299 has a different mechanism of action to MRx0518 and as such may be suitable for the treatment of different types of cancer. Studies of MRx1299’s efficacy in preclinical models of additional tumour types are ongoing, as is scale-up and process development."

He added, "The additional preclinical data on MRx0518 strengthens our knowledge of its mechanism of action and capacity to launch an immune response against cancer cells. In parallel, data generated from our ongoing clinical studies will assess the ability of MRx0518 to induce these anti-tumour immunological effects in patients. Our Phase I/II study of MRx0518 in combination with KEYTRUDA (pembrolizumab) in patients with solid tumours will provide us with specific biomarker data from both parts of the study. We anticipate the first of this data to be available by the end of 2019."

A copy of the presentations given can be found at View Source

On July 25, 2019 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported that it will release its second quarter 2019 financial results on Thursday, August 1, 2019 (Press release, Insmed, JUL 25, 2019, View Source [SID1234537774]).

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Insmed management will host a conference call for investors beginning at 8:30 a.m. ET on Thursday, August 1, 2019 to discuss the financial results and provide a business update.

Shareholders and other interested parties may participate in the conference call by dialing (888) 317-6003 (domestic) or (412) 317-6061 (international) and referencing conference ID number 5579948. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately one hour after its completion through August 8, 2019 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing replay access code 10133256. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.