On January 06, 2020 Atossa Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that its corporate name change from "Atossa Genetics Inc." to "Atossa Therapeutics, Inc." became effective at 12:01 a.m. ET today, January 6, 2020 (Press release, Atossa Genetics, JAN 6, 2020, View Source;utm_medium=twitter [SID1234629125]). The Company’s common stock will continue to trade on the NasdaqCM exchange under the ticker symbol "ATOS."

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"Over the last several years, we have transitioned the Company’s focus on developing therapies to treat breast cancer, breast density and other breast conditions," said Steve Quay, Ph.D., M.D., president and CEO of Atossa. "Changing our name to Atossa Therapeutics, Inc. more clearly reflects our focus on developing therapies and continues to honor Princess Atossa, the great queen of the Achaemenid Empire, who reigned in the fifth century BCE and who is the earliest recorded woman with breast cancer. She is featured in the The Emperor of All Maladies: A Biography of Cancer, the Pulitzer Prize-winning book by Siddhartha Mukherjee on the history of cancer."

On January 6, 2020 Gene Techno Science (Kidswell Bio) reported a joint development agreement with Sapporo Medical University for a development of an anticancer drug using antibodies with an ability of entering cancer cell (Press release, Kidswell Bio, JAN 6, 2020, View Source [SID1234625474]).

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1. Purpose and background for concluding this agreement
This agreement aims to develop new anticancer drugs for rare and refractory cancers as a new pipeline in the new biologic business, which is the future growth driver for our company. In recent years, cancer treatment has dramatically improved due to the emergence of drugs that selectively inhibit abnormal proliferation signals of cancer cells or antibody drugs that selectively recognize only cancer cells. On the other hand, the therapeutic effect achieved only with single antibody drug is limited, so in clinical practice, the combination treatment of other existing anticancer drugs and antibody drugs is a common treatment. Recently, development of antibody-drug conjugates combining cytotoxic small molecules to an antibody that recognizes cancer cells, has been actively conducted. In Japan, two antibody-drug conjugates have been launched in the market and show a great therapeutic effect.

Assistant professor Miki Yamaguchi of Research Institute of Frontier Medicine, Sapporo Medical University School of Medicine, possesses antibodies by immunizing mice, which bind to molecules expressed only on the surface of cancer cells with an ability to enter cancer cells. GTS will sort out antibodies with superior ability in a recognition specificity and entering to cancer cells, and combine them to antibodydrug conjugates combining small molecule drugs or immunotoxin combining cytotoxic protein. GTS aims to develop anti-cancer drugs that show excellent therapeutic effects even by the single administration.

GTS will conduct this research and development based on the expertise of handling proteins accumulated in the past developments of biosimilar products and append this drug as a new pipeline in the new biologic business to secure new revenue sources in the future. Through the contribution to society by developing new anticancer drugs for rare and refractory cancers, we will accelerate to raise our enterprise value and aim to achieve GTS3.0 at an early stage.

2. Contents of this agreement
Joint development agreement for an anticancer drug using antibodies with an ability of entering cancer cells which Sapporo Medical University possesses.

3. Outline of counterparties to this agreement
1. Name Sapporo Medical University
2. Location South-1, West-17, Cyuo-ku, Sapporo, Japan
3. Chairperson and President Taiji Tsukamoto
4. Department Department of Molecular Medicine, Research Institute
for Frontier Medicine, Sapporo University School of
Medicine

4. Future outlook
The impact on the business results for the fiscal year ending March 2020 is expected to be minimal.

On January 6, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics reported that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma (Press release, BridgeBio, JAN 6, 2020, View Source [SID1234576234]). In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

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Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

"We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma," said Susan Moran, MD, MSCE, chief medical officer for QED. "Fast Track Designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly."

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

"Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them."

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected]

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at View Source

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

On January 6, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics reported that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma (Press release, BridgeBio, JAN 6, 2020, View Source [SID1234576234]). In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

"We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma," said Susan Moran, MD, MSCE, chief medical officer for QED. "Fast Track Designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly."

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

"Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them."

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected]

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at View Source

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

On January 6, 2020 Black Diamond Therapeutics reported that its series A raise is going for a $100 million IPO (Press release, Black Diamond Therapeutics, JAN 6, 2020, View Source [SID1234553607]).

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It’s been a whirlwind 12 months, with two quick-fire raises coupled with a series of big-name execs coming to the biotech, which nabbed Yumanity Therapeutics, Merck KGaA and SR One veterans back in September to help head up its research work.

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This trio of new research leads are Christopher Roberts, Ph.D., chief scientific officer; Matt Lucas, Ph.D., vice president of chemistry; and Tai-An Lin, Ph.D., VP and head of biology.

Roberts oversees all of Black Diamond’s research and early development. Lucas will run its chemistry work in Cambridge, Massachusetts, while Lin is heading up its labs in New York.

The new CSO comes to the biotech from SR One, the corporate venture capital arm of GlaxoSmithKline, where he was an entrepreneur in residence. Lucas, meanwhile, was the senior director of chemistry at Parkinson’s disease biotech Yumanity.

Lin has been director of the immuno-oncology translational innovation platform and experimental medicine at Merck KGaA/EMD Serono, with stints before this on a series of Big Pharma drug discovery teams at Bristol-Myers Squibb, Roche and Janssen.

The Versant Ventures biotech raised $85 million in a series B at the start of the year, coming just weeks after Black Diamond exited stealth mode. It broke cover with a $20 million in series A and a platform designed to identify and target allosteric mutant oncogenes.

Together, these cash grabs teed up the allosteric oncogenic mutation specialist up to move two or three candidates into the clinic in the coming years: It now wants an extra $100 million from the public markets to help in this mission.

The biotech’s founders David Epstein and Elizabeth Buck designed the platform to unlock the therapeutic potential of oncogenes activated by allosteric mutations, which, unlike kinase domain mutations, are yet to make a mark on cancer care.

If the platform works as Black Diamond hopes, it will generate single molecules capable of treating baskets of mutations previously seen as unactionable from a therapeutic perspective.

The company is now focused on getting its lead program, BDTX-189, into the clinic to test this platform. According to its Securities and Exchange Commission filing, the biotech submitted its IND for BDTX-189 in November 2019 and got the FDA green light a month later.

Black Diamond is plotting a combined phase 1/2 clinical trial in the first half of 2020. "The Phase 1 portion of the trial will evaluate escalating doses of BDTX-189 and is designed to determine the recommended Phase 2 dose and to assess preliminary indications of anti-tumor activity," it said in the filing.

"The Phase 2 portion will determine the objective response rate, or ORR, and duration of response in patients with solid tumors that have an allosteric HER2 mutation or EGFR or HER2 exon 20 insertion mutation determined using next-generation sequencing, or NGS. Depending on results, we plan to pursue an accelerated approval of BDTX-189 for a tumor-agnostic indication in patients with mutations of the ErbB family."

It’s also working on a preclinical candidate it hopes can combat glioblastoma, a particularly aggressive form of brain cancer with few treatments and poor outcomes.

For this candidate, Black Diamond said: "Our lead molecules are designed to be potent, allosteric EGFR selective and to be brain penetrant. We have observed measurable brain exposure in animal models. We are completing preclinical characterization of our glioblastoma candidate leads and plan to select a development candidate in 2020."

The Cambridge, Massachusetts-based company plans to list on the Nasdaq under the symbol "BDTX."