On June 8, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase-R (PKR) activator mitapivat for the treatment of patients with thalassemia (Press release, Agios Pharmaceuticals, JUN 8, 2020, View Source [SID1234560897]). Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.

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"Receiving orphan drug designation is an important milestone as we continue to advance mitapivat for patients with thalassemia, a serious hemolytic anemia with limited treatment options," said Chris Bowden, M.D., chief medical officer at Agios. "We look forward to presenting updated data from our Phase 2 study of mitapivat in both alpha- and beta-thalassemia patients at the virtual European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress later this week."

The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

Mitapivat was previously granted orphan drug designation by the FDA and the European Medicines Agency for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia.

Mitapivat Clinical Development
Agios is conducting a Phase 2 study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat in adults with non-transfusion-dependent β- and α-thalassemia (NTDT). The trial is fully enrolled, and the primary endpoint is hemoglobin response. Preliminary Phase 2 data establishing proof-of-concept for mitapivat in thalassemia were disclosed at the end of 2019, and updated data from this trial will be presented at the 25th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress, which is being held virtually on June 11-14, 2020.

In addition, Agios has two ongoing global, pivotal trials in adults with PK deficiency that are fully enrolled.

ACTIVATE: A placebo-controlled trial with a 1:1 randomization evaluating patients who do not receive regular transfusions. The primary endpoint of the trial is the proportion of patients who achieve a sustained hemoglobin increase of ≥1.5 g/dL.
ACTIVATE-T: A single arm trial of regularly transfused patients with a primary endpoint of reduction in transfusion burden over six months compared to individual historical transfusion burden over prior 12 months.
Mitapivat is also being studied in sickle cell disease under a Cooperative Research and Development Agreement (CRADA) with the U.S. National Institutes of Health.

Mitapivat is not approved for use by any regulatory authority.

On June 8, 2020 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics, reported that it has selected development candidate, IDE397, for its Methionine adenosyltransferase 2a (MAT2A) Synthetic Lethality program (Press release, Ideaya Biosciences, JUN 8, 2020, View Source [SID1234560913]). The MAT2A program targets patients with tumors having methylthioadenosine phosphorylase (MTAP) deletions. With the nomination of IDE397 as a development candidate for the MAT2A program, IDEAYA has initiated IND-enabling studies and is targeting filing the Investigational New Drug (IND) application in the fourth quarter 2020.

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"With IDE397, we have achieved our preclinical target product profile of a differentiated compound, including potency, safety assessment, and physicochemical property characteristics. Based on the preclinical in vivo efficacy data we have generated, we believe IDE397 has the potential for evaluation in both monotherapy and combination studies in select solid tumor types with the genetic alteration of MTAP-deletion," said Michael Dillon, Ph.D., Chief Scientific Officer, IDEAYA Biosciences.

IDE397 Product Profile and MAT2A Program Summary:

Monotherapy activity with robust tumor growth inhibition and pharmacodynamic (PD) modulation in multiple endogenous MTAP-/- in vivo models
Monotherapy activity and tumor regression in HCT116 MTAP-/- xenograft model
No changes in liver enzymes or increased unconjugated bilirubin levels observed in preclinical studies
Favorable physicochemical properties and pharmacokinetics observed across multiple species
MAT2A program enabled by structure-based drug design; over 17 high resolution co-crystal structures have been resolved to enable lead optimization
About MAT2A and MTAP-Deletion:
MTAP-null cells lack the ability to metabolize 5-methylthioadenosine, or MTA, which is an essential step in a biochemical pathway involved in salvaging metabolite S-adenosyl methionine, or SAM. Increased levels of MTA partially inhibit the methyltransferase PRMT5 for which SAM is the substrate. This partial inhibition renders MTAP-null cells more dependent on the activity of methionine adenosyltransferase II alpha or MAT2A, an enzyme that is responsible for the synthesis of SAM. Because of this dependence, loss of MTAP results in synthetic lethality when MAT2A is pharmacologically inhibited.

MTAP deletions are prevalent in approximately 15% of all human tumors across various tumor types. Genetic profiling tests for the deletion of MTAP or for the commonly co-deleted gene CDKN2A are commercially available.

On June 8, 2020 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that it has commenced an underwritten public offering of its common stock (Press release, Fate Therapeutics, JUN 8, 2020, View Source [SID1234560898]). Fate Therapeutics intends to use the net proceeds from the offering to fund clinical trials and nonclinical studies, the manufacture of its clinical product candidates, the expansion of its cGMP compliant manufacturing operations, including the construction, commissioning and qualification of its new facility, the conduct of preclinical research and development, and for general corporate purposes. In connection with the offering, Fate Therapeutics expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock offered in the public offering. All shares of common stock to be sold in the offering will be offered by Fate Therapeutics. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

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Subject to the completion of the offering and the expiration or early termination of applicable waiting periods relating to certain antitrust filings under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, the Company also expects to sell to Johnson & Johnson Innovation-JJDC, Inc. in a private placement, a number of shares of common stock in an aggregate purchase price of up to $50.0 million, at a price per share equal to the price to the public in the underwritten public offering.

Jefferies, SVB Leerink and Barclays are acting as joint book-running managers for the offering.

The securities described above are being offered by Fate Therapeutics pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-228513) that was previously filed by Fate Therapeutics with the Securities and Exchange Commission (the "SEC") and automatically became effective upon filing on November 21, 2018.

A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at View Source A copy of the preliminary prospectus supplement and accompanying prospectus can be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 547-6340; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525 ext. 6218 or by email at [email protected]; or Barclays Capital Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (888) 603-5847 or by email at [email protected].

This press release does 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 state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On June 8, 2020 Oncology Venture A/S (Nasdaq First North Stockholm: OV.ST) ("OV" or the "Company") reported that it has acquired the remaining 37% ownership in its priority Dovitinib program from investor Sass & Larsen ApS (Press release, Oncology Venture, JUN 8, 2020, View Source(8%20June%202020,from%20investor%20Sass%20%26%20Larsen%20ApS. [SID1234560934]). As a result of the transaction, the Company now has full control of its most advanced pipeline program.

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The price of the remaining 37 % of OV-SPV2 ApS is agreed to SEK 36 million and a potential royalty payment of 10 % for the first 24 months following the signature of the agreement. The royalty payment, if any, will be of net sales revenues. The payment of the SEK 36 million is made by conversion of the payment into Oncology Venture A/S shares. The conversion price is SEK 1.388 per nominal DKK 0.05 share corresponding to today’s closing price of OV’s share. In total Sass & Larsen will receive 25,936,599 OV shares.

The new shares hold no special rights and will rank pari passus with all other shares in OV. Following the conversion, the Company’s share capital will be nominal DKK 7,974,053.90 divided into 25,936,599 shares of nominal DKK 0.05 each.

In March 2020, the Company announced details of its pre-NDA meeting with the U.S. Food and Drug Administration (FDA) to discuss a potential path to approval for Dovitinib used to treat Renal Cell Carcinoma (RCC) (kidney cancer), the current lead indication for the drug. The Company’s proposal is to seek approval based on "non-inferiority" against the already approved compound Sorafenib (Bayer), based on prior Phase 3 trial results (by Novartis). At that time, the Company also announced its plan to file a New Drug Application (NDA) for the approval of Dovitinib for the treatment of RCC late in the second half of 2020.

Dovitinib, a pan-tyrosine kinase inhibitor (TKI) originally developed by Novartis, addresses a significant unmet need for improved therapies for the treatment of Renal Cell Carcinoma. Annual sales of Sorafenib, under the trade name Nexavar, were approximately USD $715 million in 2018. The global RCC market is projected to grow to USD $6.3 billion by 2022. In addition to the RCC market, Dovitinib has promising potential as a monotherapy in a number of other indications, including metastatic breast cancer, hepatocellular cancer, endometrial cancer and gastrointestinal stromal tumors, as well as in combination therapy with other approved drugs, including immune checkpoint inhibitors.

Steve Carchedi, CEO of Oncology Venture, stated "We are excited to gain full ownership of our priority Dovitinib program, as we advance towards U.S. submission of our first NDA and towards approval, marketing and sale of this key asset. Our acquisition of the remaining investor interest in this program now provides our Company and our shareholders with the full potential upside of this promising drug, as we bring it towards the market and to cancer patients."

On June 8, 2020 Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported a proposed public offering of $80 million of shares of its common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of its common stock (Press release, Replimune, JUN 8, 2020, View Source [SID1234560899]). All securities in the offering will be offered by Replimune. In addition, Replimune intends to grant the underwriters a 30-day option to purchase up to an additional $12 million of shares of its common stock from Replimune at the public offering price, less the underwriting discounts and commissions.

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J.P. Morgan Securities LLC, SVB Leerink LLC, and BMO Capital Markets Corp. are acting as joint book-running managers for the proposed offering. Wedbush Securities Inc. is acting as lead manager for the proposed offering. The proposed offering is subject to market and other customary closing conditions, and Replimune cannot assure you as to whether or when the proposed offering may be completed.

The proposed offering will be made only by means of a preliminary prospectus supplement and the accompanying prospectus. A copy of the preliminary prospectus supplement and the accompanying prospectus relating to the proposed offering will be filed with the Securities and Exchange Commission (the "SEC") and may be obtained, when available, by visiting EDGAR on the SEC website at www.sec.gov or from: J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by e-mail at [email protected]; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6218, or by e-mail at [email protected]; or BMO Capital Markets Corp., Attention: Equity Syndicate Department, 3 Times Square, 25th Floor, New York, NY 10036, by telephone at (800) 414-3627 or by e-mail at [email protected]. The final terms of the proposed offering will be disclosed in a final prospectus supplement to be filed with the SEC.

The shares of common stock and pre-funded warrants described above are being offered by Replimune pursuant to its shelf registration statement on Form S-3, including a base prospectus, that was previously filed by Replimune with the SEC on August 8, 2019 and declared effective by the SEC on August 15, 2019. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of 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 jurisdiction.