On December 30, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported the filing of an investigational new drug (IND) application with the U.S. Food and Drug Administration to initiate the clinical program of TransCon TLR7/8 Agonist (Press release, Ascendis Pharma, DEC 30, 2020, View Source [SID1234573331]).

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TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained activation of intratumoral antigen presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines in the tumor.

"The filing of our first oncology IND for TransCon TLR7/8 Agonist – which is designed to provide intratumoral, sustained release of resiquimod over several weeks from a single administration with minimal systemic exposure and potent immune response against cancer cells – is a major milestone for Ascendis," said Juha Punnonen, M.D., Ph.D., Senior Vice President and Head of Oncology at Ascendis Pharma. "We believe TransCon TLR7/8 Agonist represents a potential paradigm shift in the treatment of cancer through sustained release of an immunostimulatory molecule over several weeks inside the tumor, thereby employing the patients’ own immune systems to destroy cancer cells with reduced risk of systemic adverse events."

About TransCon Technology

TransCon refers to "transient conjugation." The proprietary TransCon platform is an innovative technology to create new therapies that are designed to optimize therapeutic effect, including efficacy and safety and through dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.

On December 30, 2020 Cancer Research UK reported that scientists have finally found a way to target the KRAS mutation – a common mutation found in several hard-to-treat cancers (Press release, Cancer Research UK, DEC 30, 2020, View Source [SID1234573621]).

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By targeting two components of the KRAS signalling pathway, a new drug was much better at stopping the growth of pancreatic tumours with a KRAS mutation in mice compared to standard KRAS inhibitors, which only block one component of the pathway.

The drug, which has already shown promise in one sarcoma patient whose tumour had a KRAS mutation, will next be tested in patients with a variety of cancers in a larger Phase II clinical trial. The results are published in Annals of Oncology(link is external) today (Wednesday).

Changes to the KRAS gene, which is important in controlling cell growth, can lead to cells growing and dividing out of control to form tumours. Mutations in KRAS are found across 25% of cancers and are commonly found in several hard-to-treat cancers, including pancreatic, lung and colorectal tumours*.

Despite the prominent role of KRAS mutations in cancers and clear need for new treatments, scientists have been working for decades trying to find ways to target this pathway. Researchers say the main issue is because current KRAS inhibitors only target one component of the signalling pathway.

Now, a team of researchers led by Professors Caroline Springer and Richard Marais at the Cancer Research UK Manchester Institute have developed a new drug called CCT3833, which blocks two pathways in the KRAS signalling pathway called RAF and SRC.

The scientists tested CCT3833 in pancreatic, colorectal and lung cancer cell lines that had KRAS gene changes, as well as pancreatic tumours in mice, and compared the new drug with other standard KRAS inhibitors**.

Results showed that CCT3833 was able to restrict the growth of the cancer cells. It was also more effective in killing the cancer cells compared with other KRAS inhibitors. This was even more apparent when using 3D spherical tumour models, or ‘mini tumours’, which bridge the environment of cells grown in the lab and tumours in the body.

When tested in mouse models of pancreatic cancer with KRAS mutations, CCT3833 was more effective than other drugs in preventing cancer growth. CCT3833 also reduced tumour size.­

The study also describes the results of an early phase clinical trial of the drug tested in 31 patients with solid tumours. Whilst primarily designed to test safety, one patient in the trial who had a KRAS-mutant tumour that was growing larger after failing to respond to standard treatment had their tumour reduce in size for 8 months after receiving CCT3833.

The team is now planning to investigate the drug in further clinical trials.

Professor Caroline Springer, director of the drug discovery unit at the Cancer Research UK Manchester Institute, said: "Faulty KRAS signalling in cancer was identified over 35 years ago and yet, we still cannot block this pathway effectively. Our new drug stops the function of two critical growth pathways in KRAS-driven cells and has shown to be more effective than current drugs that only block one part of the pathway. We now need to run broader clinical trials to see how many cancer patients could benefit from this drug."

Professor Richard Marais, director of the Cancer Research UK Manchester Institute, said: "Cancers with a KRAS gene change have been notoriously difficult to treat. Because current drugs that target KRAS are only able to block one part of the pathway, cancer cells can easily outmanoeuvre treatments. It’s extremely encouraging to see that the impressive effects of the drug in the lab are already also showing promise in early-stage trials that include hard-to-treat cancers."

Michelle Mitchell, chief executive of Cancer Research UK, said: "This study has built upon almost 20 years of Cancer Research UK-funded research, and shows the importance of investing in basic research in the lab, which unpicks the biology of cancer. Breakthroughs in science are all about small steps that can lead to great change, and these results have brought us closer to a potential new option for people with these difficult-to-treat cancers."

On December 30, 2020, Lumos Pharma, Inc. (the "Company") reported that it entered into a Controlled Equity OfferingSM Sales Agreement (the "Sales Agreement") with Cantor Fitzgerald & Co., as agent (the "Agent"), pursuant to which the Company may offer and sell from time to time through the Agent up to $50.0 million of shares of the Company’s common stock, $0.01 par value (the "Shares") (Filing, 8-K, NewLink Genetics, DEC 30, 2020, View Source [SID1234573332]). The offering and sale of the Shares has been registered under the Securities Act of 1933, as amended (the "Securities Act"), pursuant to the Company’s Registration Statement on Form S-3 (File No. 333-226366) (the "Registration Statement"), which was originally filed with the Securities and Exchange Commission ("SEC") on July 26, 2018 and declared effective by the SEC on July 22, 2019, the base prospectus contained within the Registration Statement, and a prospectus supplement that was filed with the SEC on December 30, 2020.

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Under the Sales Agreement, the Agent may sell the Shares by any method permitted by law and deemed to be an "at-the-market" offering as defined in Rule 415(a)(4) promulgated under the Securities Act, including sales made directly on or through The Nasdaq Global Market, on any other existing trading market for the Shares, in negotiated transactions at market prices prevailing at the time of sale or at prices related to such prevailing market prices and/or any other method permitted by law. The Company will notify the Agent of the number of Shares to be issued, the time period during which sales are requested to be made, any limitation on the number of Shares that may be sold in any one day and any minimum price below which sales may not be made. The Company intends to use the proceeds of the offering as described in the prospectus supplement.

The Sales Agreement contains customary representations, warranties and agreements by the Company, as well as indemnification and contribution obligations of the Company for certain liabilities under the Securities Act. Under the terms of the Sales Agreement, the Company will pay the Agent a commission of up to 3.0% of the gross sales price of the Shares sold through it under the Sales Agreement. In addition, the Company has agreed to reimburse certain expenses incurred by the Agent in connection with the offering.

The Company is not obligated to make any sales of the Shares under the Sales Agreement. The Sales Agreement may be terminated by the Agent or the Company at any time upon notice to the other party, as set forth in the Sales Agreement, or by the Agent at any time in certain circumstances, including the occurrence of a material and adverse change in the Company’s business or financial condition that makes it impractical or inadvisable to market the shares or to enforce contracts for the sale of the Shares.

The foregoing description of the material terms of the Sales Agreement is not intended to be complete and is qualified in its entirety by reference to the Sales Agreement, which is filed herewith as Exhibit 10.1 and incorporated herein by reference.

Wilson Sonsini Goodrich & Rosati P.C., counsel to the Company, has issued a legal opinion relating to the Shares. A copy of such legal opinion, including the consent included therein, is attached as Exhibit 5.1 hereto.

This Current Report on Form 8-K shall not constitute an offer to sell or a solicitation of an offer to buy any securities, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.

On December 29, 2020 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported that Sean George, co-founder and chief executive officer of Invitae, will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 10:50 a.m. Eastern/7:50 a.m. Pacific (Press release, Invitae, DEC 29, 2020, View Source [SID1234573312]). Following the company presentation, management will participate in a virtual breakout session at 11:10 a.m. Eastern/8:10 a.m. Pacific.

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

The 2021 J.P. Morgan Healthcare Conference is being held virtually via webinar. A live webcast of both the presentation and the breakout session may be accessed at the following direct link or by visiting the investors section of the company’s website at ir.invitae.com.

Public listeners can access an audio and slide recording of the session, which will be available shortly after the conclusion of the presentation and breakout session on the investors section of the company’s website at ir.invitae.com.

On December 29, 2020 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that it has entered into a securities purchase agreement with an existing investor to sell an aggregate of 520,961 shares of common stock in a registered direct offering at a price of $9.61 per share, which was the closing price of the Company’s common stock on December 28, 2020 (Press release, Tracon Pharmaceuticals, DEC 29, 2020, View Source [SID1234573313]). The aggregate gross proceeds to TRACON from the offering are expected to be $5.0 million. The closing of the offering is expected to occur on or about December 30, 2020.

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TRACON intends to use the net proceeds from the offering to fund the ENVASARC pivotal study of envafolimab in sarcoma, further business development efforts, and for working capital and general corporate purposes.

The offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-229990) previously filed with the U.S. Securities and Exchange Commission (the "SEC"). A prospectus supplement describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website located at View Source Before investing in the offering, interested parties should read in their entirety the prospectus supplement and the accompanying prospectus and the other documents that the Company has filed with the SEC that are incorporated by reference in such prospectus supplement and the accompanying prospectus, which provide more information about the Company and the offering.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.