On January 18, 2023 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported that it intends to offer and sell $125,000,000 of shares of its common stock in an underwritten public offering (Press release, Deciphera Pharmaceuticals, JAN 18, 2023, View Source [SID1234626345]). In addition, Deciphera intends to grant the underwriters a 30-day option to purchase up to an additional $18,750,000 of shares of common stock offered in the public offering. All of the shares are being offered by Deciphera. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Deciphera intends to use the net proceeds from the offering to fund its planned Phase 3 INSIGHT study of QINLOCK versus sunitinib in second-line GIST patients with mutations in KIT exon 11 and 17/18 only; to fund the development of vimseltinib, including completion of its Phase 3 MOTION study of vimseltinib in tenosynovial giant cell tumor patients currently underway, additional clinical trials as well as clinical research outsourcing and manufacturing of clinical trial material and pre-commercial and medical affairs capabilities related to vimseltinib; to fund the development of DCC-3116, including multiple expansion cohorts in the ongoing Phase 1b combination dose escalation studies and potential Phase 2 expansion combination cohorts in multiple tumor types as well as clinical research outsourcing and manufacturing of clinical trial material; to fund the research and development of its pan-RAF program, as well as a potential new development candidate and other new research activities from its proprietary discovery engine of novel switch control inhibitors; and the remainder for working capital purposes, including general operating expenses.

J.P. Morgan, Jefferies, Cowen, and Guggenheim Securities are acting as joint book-running managers for the offering.

The securities described above are being offered by Deciphera pursuant to a shelf registration statement on Form S-3 (No. 333-266523) that was declared effective by the Securities and Exchange Commission (SEC) on August 10, 2022. 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 located at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, by contacting: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 866-803-9204, or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at 877-821-7388 or by email at [email protected]; Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, via telephone: +1 (833) 297-2926, or via email: [email protected]; or Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, New York 10017, by telephone at 212-518-9544 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 that state or jurisdiction.

On january 18, 2023 bluebird bio, Inc. (Nasdaq: BLUE) ("bluebird") reported that it has commenced an underwritten public offering of 20,000,000 shares of its common stock (Press release, bluebird bio, JAN 18, 2023, View Source [SID1234626343]). bluebird also intends to grant the underwriters a 30-day option to purchase up to an additional 3,000,000 shares of its common stock to be sold in the offering. The offering, actual size and terms are subject to market conditions, and there can be no assurance as to whether or when the offering may be completed. All shares in the offering are to be sold by bluebird.

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Goldman Sachs & Co. LLC and J.P. Morgan Securities LLC are acting as joint book running managers for the offering.

bluebird intends to use the net proceeds of the offering (i) to support commercialization and manufacturing for its two approved gene therapies, ZYNTEGLO and SKYSONA; (ii) to accelerate future commercialization activities for its gene therapy candidate, lovotibeglogene autotemcel (lovo-cel) for sickle cell disease, if approved; and (iii) to fund working capital and other general corporate purposes.

The offering is being made pursuant to an effective shelf registration statement on Form S-3, including a prospectus, that was filed with the U.S. Securities and Exchange Commission (the "SEC") on February 18, 2020 and was automatically effective upon filing. A preliminary prospectus supplement describing the terms of the offering will be filed with the SEC and will form a part of the effective registration statement. Copies of the preliminary prospectus supplement and accompanying prospectus relating to the public offering may be obtained, when available, by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by phone at (866) 471-2526, or by email at [email protected]; or J.P. Morgan Securities LLC, Attention: c/o Broadridge Financial Solutions, 155 Long Island Avenue, Edgewood, NY 11717, by phone at (866) 803-9204, or by email at [email protected]. Before you invest, you should read the preliminary prospectus supplement and accompanying prospectus and the other documents that bluebird has filed with the SEC for more complete information about bluebird and the proposed 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 state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Examples of forward-looking statements in this press release include, without limitation, statements regarding the consummation of the offering, the terms of the offering and the anticipated use of the net proceeds from the offering. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. These risks include, but are not limited to: the risk that we may not realize expected cost savings from the restructuring, including the anticipated decrease in operational expenses, at the levels we expect; we may encounter additional delays in the development of our programs, including the imposition of new clinical holds or delays in resolving existing clinical holds, that may impact our ability to meet our expected timelines and increase our costs; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause us to use cash more quickly than we expect or change or curtail some of our plans or both; our expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than our assumptions; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be seen in additional patients treated with our product candidates; the risk that additional insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time; the risk that our eli-cel, beti-cel and lovo-cel programs may be subject to further delays in their development, including but not limited to the imposition of new clinical holds; the risk that lovo-cel may not be approved within the priority review timeframe or at all; and the risk that any one or more of our products and product candidates, including eli-cel, beti-cel or lovo-cel, will not be successfully developed, approved or commercialized. The forward-looking statements included in this press release are made only as of the date of this press release and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

ON January 18, 2023 RenovoRx, Inc. (Nasdaq: RNXT), a biopharmaceutical company focused on the localized treatment of solid tumors, reported initial results from a pharmacokinetic (PK) substudy within the phase III un-blinded randomized control TIGeR-PaC clinical trial to be presented at the 2023 ASCO (Free ASCO Whitepaper) Gastrointestinal (ASCO GI) Cancers Symposium this week (Press release, Renovorx, JAN 18, 2023, View Source [SID1234626342]). The TIGeR-PaC clinical trial is evaluating intra-arterial (IA) administration of gemcitabine (chemotherapy) using the proprietary RenovoRx Trans-Arterial Micro-Perfusion (RenovoTAMP) platform for targeted treatment of Locally Advanced Pancreatic Cancer (LAPC). The substudy provides clinical support that RenovoTAMP may increase local drug delivery and thus concentration at the tumor site while decreasing the debilitating side effects often associated with systemic intravenous (IV) delivery, which is the current standard of care.

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Three additional abstracts supporting the use of RenovoTAMP with gemcitabine for treatment of LAPC will also be presented at the ASCO (Free ASCO Whitepaper) GI on January 19-21, 2023 in San Francisco, California, and available online.

"Intra-arterial Gemcitabine vs IV Gemcitabine PK Substudy in Patients with Locally Advanced Pancreatic Cancer," presented by Amer H. Zureikat, MD, et al., evaluates RenovoTAMP for IA delivery of gemcitabine (chemotherapy) directly into tumors for higher local drug concentration and decreased systemic drug concentration and associated side effects. The PK substudy evaluates a sample of LAPC patients (N=13) participating in the TIGeR-PaC study and demonstrates that the cohort had an average greater than 50% reduction in systemic drug exposure with IA delivery of gemcitabine using RenovoTAMP when compared with IV administration. The substudy concludes that RenovoTAMP may increase local gemcitabine concentration, which may be beneficial in decreasing gemcitabine-related systemic side effects. Five TIGeR-PaC clinical sites participated in this substudy.

"The upcoming presentation of our TIGeR-PaC clinical trial substudy at ASCO (Free ASCO Whitepaper) GI highlights significant potential benefits for patients with LAPC," said Ramtin Agah, MD, Chief Medical Officer and Founder of RenovoRx. "Targeted local delivery of standard dose gemcitabine via the RenovoTAMP therapy platform may be associated with significantly less systemic drug exposure. The clinical implications may be decreased side effects and enhanced chemotherapy delivery. Ongoing studies are quantifying the resulting impact on improving patients’ quality of life and extending lifespan."

"This substudy data, and the additional three studies to be presented at ASCO (Free ASCO Whitepaper) GI, enhance the strong clinical momentum of our therapy platform as we prepare for our most significant milestone to date: the initial interim analysis for our randomized phase III TIGeR-PaC trial." said Shaun Bagai, CEO of RenovoRx.

Three additional clinical data abstracts presented by researchers at ASCO (Free ASCO Whitepaper) GI with data from the induction phase of the TIGeR-PaC study help to advance the science behind pancreatic cancer. In one abstract, Dr. Amer H. Zureikat, et al. investigates Mesenteric Venous Thrombosis (MVT), often identified on routine imaging studies performed with LAPC, and concludes that severe MVT is more prevalent in this patient population than previously reported. Anticoagulation is also underutilized in this cohort; however, chemotherapy may have a beneficial effect in downstaging MVT beyond anticoagulation. In a second abstract, Dr. Karyn A. Goodman, et al. performs an exploratory analysis to compare the toxicity and efficacy between patients receiving either stereotactic body radiation therapy (SBRT) or intensity-modulated radiation therapy (IMRT) during the induction phase (prior to randomization) of the TIGeR-PaC study. When compared to IMRT, SBRT demonstrates improved tolerability for treatment of patients with LAPC with comparable clinical efficacy. It was this finding that led to the modification of the TIGeR-PaC study design in 2021. Finally, a third abstract presented by Michael J. Pishvaian, et al. focuses on the TIGeR-PaC trial design and status.

The poster presentations for the four RenovoRx abstracts to be presented at the ASCO (Free ASCO Whitepaper) GI Symposium will be available on RenovoRx’s website once available: View Source

On January 18, 2023 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported that REZLIDHIA (olutasidenib) has been added by the National Comprehensive Cancer Network (NCCN) to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for acute myeloid leukemia (AML) (Press release, Rigel, JAN 18, 2023, View Source [SID1234626341]). REZLIDHIA (olutasidenib) is now included as a recommended targeted therapy for adult patients with relapsed/refractory (R/R) AML with isocitrate dehydrogenase-1 (IDH1) mutation.

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"We are pleased that REZLIDHIA was quickly added to the NCCN Guidelines for AML, receiving important recognition as an appropriate treatment option for adult R/R AML patients with an IDH1 mutation," said Raul Rodriguez, president and CEO of Rigel. "Inclusion in the NCCN Guidelines for AML reinforces the strength of the data, which supports the safety and efficacy of REZLIDHIA."

In December 2022, the U.S. Food and Drug Administration (FDA) approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test. REZLIDHIA is an oral, small molecule, inhibitor of mutated IDH1 designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells.

The NCCN is a not-for-profit alliance of 32 leading U.S. cancer centers devoted to patient care, research, and education. The NCCN Guidelines are the recognized standard for clinical direction and policy in cancer care and are the most thorough and frequently updated clinical practice guidelines available in any area of medicine. For more information visit: View Source

NCCN and the NCCN Guidelines are registered trademarks of National Comprehensive Cancer Network.

About AML
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.1

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.2 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.3 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About REZLIDHIA
INDICATION
REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, can occur with REZLIDHIA treatment.

Symptoms may include dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney

injury, hypotension, fever, and weight gain. If differentiation syndrome is suspected,

withhold REZLIDHIA and initiate treatment with corticosteroids and hemodynamic

monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS
Differentiation Syndrome
REZLIDHIA can cause differentiation syndrome. In the clinical trial of REZLIDHIA in patients with relapsed or refractory AML, differentiation syndrome occurred in 16% of patients, with grade 3 or 4 differentiation syndrome occurring in 8% of patients treated, and fatalities in 1% of patients. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with REZLIDHIA included leukocytosis, dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, fever, edema, pyrexia, and weight gain. Of the 25 patients who experienced differentiation syndrome, 19 (76%) recovered after treatment or after dose interruption of REZLIDHIA. Differentiation syndrome occurred as early as 1 day and up to 18 months after REZLIDHIA initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, temporarily withhold REZLIDHIA and initiate systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and until resolution of signs and symptoms. If concomitant leukocytosis is observed, initiate treatment with hydroxyurea, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms. Differentiation syndrome may recur with premature discontinuation of corticosteroids and/or hydroxyurea treatment. Institute supportive measures and hemodynamic monitoring until improvement; withhold dose of REZLIDHIA and consider dose reduction based on recurrence.

Hepatotoxicity
REZLIDHIA can cause hepatotoxicity, presenting as increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased blood alkaline phosphatase, and/or elevated bilirubin. Of 153 patients with relapsed or refractory AML who received REZLIDHIA, hepatotoxicity occurred in 23% of patients; 13% experienced grade 3 or 4 hepatotoxicity. One patient treated with REZLIDHIA in combination with azacitidine in the clinical trial, a combination for which REZLIDHIA is not indicated, died from complications of drug-induced liver injury. The median time to onset of hepatotoxicity in patients with relapsed or refractory AML treated with REZLIDHIA was 1.2 months (range: 1 day to 17.5 months) after REZLIDHIA initiation, and the median time to resolution was 12 days (range: 1 day to 17 months). The most common hepatotoxicities were elevations of ALT, AST, blood alkaline phosphatase, and blood bilirubin.

Monitor patients frequently for clinical symptoms of hepatic dysfunction such as fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. Obtain baseline liver function tests prior to initiation of REZLIDHIA, at least once weekly for the first two months, once every other week for the third month, once in the fourth month, and once every other month for the duration of therapy. If hepatic dysfunction occurs, withhold, reduce, or permanently discontinue REZLIDHIA based on recurrence/severity.

ADVERSE REACTIONS
The most common (≥20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased, alanine aminotransferase increased, potassium decreased, sodium decreased, alkaline phosphatase increased, nausea, creatinine increased, fatigue/malaise, arthralgia, constipation, lymphocytes increased, bilirubin increased, leukocytosis, uric acid increased, dyspnea, pyrexia, rash, lipase increased, mucositis, diarrhea and transaminitis.

DRUG INTERACTIONS

Avoid concomitant use of REZLIDHIA with strong or moderate CYP3A inducers.
Avoid concomitant use of REZLIDHIA with sensitive CYP3A substrates unless otherwise instructed in the substrates prescribing information. If concomitant use is unavoidable, monitor patients for loss of therapeutic effect of these drugs.
LACTATION
Advise women not to breastfeed during treatment with REZLIDHIA and for 2 weeks after the last dose.

GERIATRIC USE
No overall differences in effectiveness were observed between patients 65 years and older and younger patients. Compared to patients younger than 65 years of age, an increase in incidence of hepatotoxicity and hypertension was observed in patients ≥65 years of age.

HEPATIC IMPAIRMENT
In patients with mild or moderate hepatic impairment, closely monitor for increased probability of differentiation syndrome.

On January 18, 2023 Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported that strengthens its Executive Management with the inclusion of Jan Stenvang, who is appointed Chief Scientific Officer (CSO) reporting directly to CEO Francois Martelet (Press release, Scandion Oncology, JAN 18, 2023, View Source;ph-d–chief-scientific-officer-and-member-of-executive-mana,c3699655 [SID1234626340]).

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A co-founder of Scandion, Jan Stenvang led the initial research and discoveries upon which Scandion is based. He has headed the company’s research and early development activities ever since and has played a pivotal role in e.g. the planning and execution of the development of Scandion’s lead compound SCO-101. In his new role he will continue this work, also focusing on potential new indications that Scandion could pursue.

With a master’s degree and Ph.D. in Molecular and Cellular Biology, Jan Stenvang has specialized in translational cancer research particularly focusing on drug resistance and biomarker identification. Before co-founding Scandion, he spent most of his career in academia as e.g. Group Leader and Associate Professor at Copenhagen University and also as Group Leader at the biotech company Santaris Pharma. Combined, Jan Stenvang has more than 20 years of experience in cancer research.

"We are delighted to appoint Jan CSO and member of Executive Management allowing us to utilize his expertise even more in the management and development of Scandion. Jan is a distinguished researcher and expert in the field of cancer drug resistance and his contributions will be crucial in our work to bring forward new and better cancer treatments", says Francois Martelet, CEO of Scandion.

"It is a great pleasure for me to be taking this new role and also contribute as a member of Executive Management in Scandion. We have the potential to make a huge difference for patients and I am excited to contribute to the research and development of medicines that can revert cancer drug resistance", says Jan Stenvang.