On February 3, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported the closing of its previously announced sale of a number of its bioengineering assets and the related sale of $35 million of its common stock to an affiliate of Third Security LLC (Press release, Intrexon, FEB 3, 2020, View Source [SID1234553806]). Additionally, effective February 1, 2020, the Company has changed its name to Precigen, Inc. from Intrexon Corporation and its Nasdaq stock symbol to PGEN from XON. The new Precigen enters 2020 with a promising portfolio of investigational gene and cell therapies, derived from the Company’s transformative therapeutic platforms, including UltraCAR-T, AdenoVerse cytokine therapies, multifunctional therapeutics and off-the-shelf AdenoVerse immunotherapies, as well as innovative approaches from our subsidiaries ActoBio Therapeutics, Exemplar Genetics, and Triple-Gene.

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"With multiple data read-outs on track for our internal pipeline and partnered programs, we believe 2020 will be an exciting year for Precigen," said Helen Sabzevari, Ph.D., President and CEO of Precigen. "At the same time, we plan to further optimize our portfolio this year through a renewed commitment to financial rigor and fiscal discipline."

"We are particularly enthusiastic about the opportunity to advance our UltraCAR-T platform as current CAR-T treatments rely on a long, complex, and relatively inefficient manufacturing process that delays treatment for cancer patients and leads to high pricing. By contrast, UltraCAR-T is designed to overcome many of these obstacles by eliminating the need for ex vivo expansion, reducing overall manufacturing time, and enabling patients to be treated with CAR-T therapy only one day after non-viral gene transfer at the cancer center. UltraCAR-T has the potential to democratize patient care by simplifying this life-saving cancer immunotherapy which should improve availability throughout the world," added Dr. Sabzevari.

Two non-healthcare businesses remain with Precigen and the Company will continue to evaluate strategic and operational options for these businesses.

Dr. Sabzevari presented an overview of Precigen at the 38th Annual J.P. Morgan Healthcare Conference on January 14, 2020, and a replay of the presentation is available on Precigen’s website in the Events section at View Source

On February 3, 2020 Anchiano Therapeutics Presented the Corporate Presentation (Presentation, Anchiano Therapeutics, FEB 3, 2020, View Source [SID1234553804]).

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On February 3, 2020 Nevro Corp. (NYSE: NVRO), a global medical device company that is providing innovative, evidence-based solutions for the treatment of chronic pain, reported that the Company will release its financial results for the fourth quarter and full year ended December 31, 2019 after the market closes on February 25, 2020 (Press release, Nevro, FEB 3, 2020, View Source [SID1234553795]).

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Investors interested in listening to the conference call may do so by dialing (833) 286-5807 in the U.S. or (647) 689-4452 internationally, using Conference ID: 1864387. In addition, a live webcast, as well as an archived recording, will be available on the "Investors" section of the Company’s website at: www.nevro.com.

On February 3, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported that following a meeting with the U.S. Food and Drug Administration ("FDA" or "the Agency"), CTI has reached agreement on an accelerated approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia (platelet counts <50,000/µL) (Press release, CTI BioPharma, FEB 3, 2020, View Source [SID1234553794]). CTI will be amending the PACIFICA pivotal Phase 3 trial protocol to allow for the primary analysis of SVR rates on the first 168 patients, with an end-of-study analysis of TSS and OS following the full enrollment of 348 patients. If the primary endpoint of SVR is met following the planned review of data from the first 168 patients, CTI intends to submit a New Drug Application (NDA) under the FDA’s subpart H regulations, subject to review of all available efficacy and safety data. Conversion to a regular approval of pacritinib would be anticipated following the successful end-of-study assessment of the secondary efficacy endpoints, and the completion of post-marketing requirements.

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"Since the initiation of the PACIFICA trial in September 2019, we have been working diligently with the FDA to identify an expedited approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "Severely thrombocytopenic myelofibrosis patients (platelet counts <50,000/µL) have reduced survival and very limited therapeutic options. Pacritinib has now demonstrated clinical benefit in this population in three clinical trials, including two prior randomized Phase 3 studies, so we believe that pacritinib has the potential to change the treatment paradigm in this area of serious unmet medical need."

Based on the new trial design, CTI expects to report primary SVR data by the end of 2021, with a potential NDA filing in early 2022 if the SVR data is positive. Final study efficacy data is expected in 2023.

Concurrent with this press release, CTI is announcing a $60 million rights offering. For further details, see the concurrent press release relating to the rights offering.

About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 17,000 people. Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months. Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, which can lead to dose reductions, and as a result, may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months. There are currently no approved therapies available to treat myelofibrosis patients with severe thrombocytopenia or patients who have failed ruxolitinib treatment, thereby making this a significant unmet medical need.

About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

On February 3, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported its intent to raise $60 million through a fully backstopped rights offering (Press release, CTI BioPharma, FEB 3, 2020, View Source [SID1234553793]). Under the terms of the rights offering, investors as of February 13, 2020 in CTI BioPharma’s common stock and preferred stock will receive a subscription right entitling them to purchase their pro rata share of the $60 million offering amount. The rights offering will be fully backstopped by BVF Partners L.P., Stonepine Capital, L.P., OrbiMed Private Investments VI, LP and New Enterprise Associates, Inc. each of which have agreed to purchase, at a minimum, its respective as-converted pro rata share of the offering amount, plus an additional amount of securities that are not subscribed for by other purchasers in the rights offering, for a total of up to $60 million.

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Under the proposed rights offering (the "Rights Offering"), CTI BioPharma plans to distribute non-transferable subscription rights to purchase a portion of a share of CTI BioPharma’s common stock ("Common Stock") or series X convertible preferred stock ("Series X Preferred") to each stockholder of record of Common Stock, including Common Stock issuable upon conversion of the outstanding shares of CTI Biopharma’s series O convertible preferred stock ("Series O Preferred") at $1.00 per share to such security holders of record as of the close of business on February 13, 2020 (the "Record Date"). The subscription rights will be exercisable for shares of Common Stock (or an equivalent number of Series X Preferred), with participation to be allocated among holders of its Common Stock and Series O Preferred on a pro rata basis (assuming full conversion of the Series O Preferred into shares of Common Stock), subject to the aggregate offering threshold and ownership limitations. The subscription rights may be exercised only during the anticipated subscription period of Friday, February 14, 2020, through 5:00 PM (Eastern Time) on Monday, March 2, 2020, unless extended. Any participant in the Rights Offering that, by exercise of its subscription right would become a holder of greater than 9.9% of the outstanding number of shares of Common Stock following the Rights Offering may elect to instead purchase Series X Preferred. CTI BioPharma intends to sell the Series X Preferred at $10,000 per share, and any such holder so electing would have a right to purchase one 10,000th of a share of Series X Preferred for each share of Common Stock it had a right to purchase under the subscription rights. Each share of Series X Preferred is, subject to certain limitations, convertible into 10,000 shares of Common Stock at the election of the holder. The Series X Preferred generally have no voting rights, except as required by law, and will participate pari passu, on an as-converted basis, with any distribution of proceeds to holders of Common Stock and Series O Preferred in the event of CTI BioPharma’s liquidation, dissolution or winding up.

The Rights Offering will be made pursuant to CTI BioPharma’s effective shelf registration statement on file with the Securities and Exchange Commission and only by means of a prospectus supplement and accompanying prospectus. CTI BioPharma expects to mail subscription certificates evidencing the subscription rights and a copy of the prospectus supplement and accompanying prospectus for the Rights Offering shortly following the Record Date.

This press release is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities in any jurisdiction, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.

Certain existing shareholders of CTI BioPharma (the "Investors"), including BVF Partners L.P., CTI BioPharma’s largest shareholder, currently owning approximately 11.95% of CTI BioPharma’s outstanding Common Stock (and 23.1% on an as-converted basis), Stonepine Capital, L.P., OrbiMed Private Investments VI, LP and New Enterprise Associates, Inc. currently owning 6.23%, 8.62% and 5.59% of CTI BioPharma’s outstanding Common Stock, respectively, will backstop the Rights Offering and have agreed to purchase up to $60 million of Common Stock at a subscription price per share of $1.00 (or Series X Preferred at a price of $10,000 per share) in a private placement promptly at the conclusion of the Rights Offering, with the dollar amount to be purchased in such private placement reduced by the dollar amount sold by CTI BioPharma (including to the Investors and their respective affiliates) in the Rights Offering.

Concurrent with this press release, CTI is announcing the outcome of its recent meeting with the U.S. Food and Drug Administration and the subsequent amendment to the PACIFICA trial. For further details, see the concurrent press release relating to this meeting.