On January 18, 2023 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, reported that the first patient has been dosed in the ReSPECT-GBM Phase 2b dose expansion clinical trial evaluating rhenium (186Re) obisbemeda for the treatment of recurrent glioblastoma (GBM) (Press release, PLUS THERAPEUTICS, JAN 18, 2023, View Source;_hsmi=242229049&_hsenc=p2ANqtz-_yNe2WheQKNsEW3EUnRcfAl-UnEZJRUrRbt9ZO3byyP_mt1dFqHsUnsVrfmUqhmgg0ilEZE5aWncX5JJ-VdXWUovDNog&utm_content=242229049&utm_source=hs_email [SID1234626347]).

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This Phase 2b multi-center trial is designed to evaluate the safety, tolerability, distribution and efficacy of rhenium (186Re) obisbemeda infused directly into the tumor via convection-enhanced delivery catheters in patients with recurrent GBM progressing after conventional treatment.

"In the Phase 1/2a dose escalation trial, we showed that a rhenium (186Re) obisbemeda radiation dose of 22.3 mCi in an infused volume of 8.8 mL can be safely administered and that there is a statistically significant correlation between overall survival and both absorbed radiation dose to the tumor and percent tumor volume in the treated volume," said Andrew J. Brenner, M.D., Ph.D., Professor of Medicine, Neurology, and Neurosurgery, Kolitz/Zachry Endowed Chair Neuro-Oncology Research, and Co-Leader of the Experimental and Developmental Therapeutics Program at The University of Texas Health Science Center at San Antonio and principal investigator of the ReSPECT-GBM clinical trial. "The strength of this correlation is unusually positive for a Phase 1/2a trial and we are optimistic that these safety and efficacy signals will be confirmed in the ongoing Phase 2b trial."

The Phase 2b trial is expected to enroll up to 31 additional patients with small- to medium-sized tumors (20 mL or less) in approximately 24 months. The trial is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH).

"We have successfully completed two of our key near-term clinical development goals, specifically to manufacture cGMP rhenium (186Re) obisbemeda and move our glioblastoma program into Phase 2b," said Norman LaFrance, M.D., Chief Medical Officer and Senior Vice President at Plus Therapeutics. "The treatment of our first patient went well with excellent targeted tumor delivery of high dose rhenium (186Re) obisbemeda, similar to that seen at this same dose in our Phase 1/2a trial. Moving forward this year, our focus will be to expand the trial sites and ramp up enrollment to accelerate clinical development of this novel treatment option."

As disclosed at the Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology in November 2022, results from the Phase 1 ReSPECT-GBM clinical trial of rhenium (186Re) obisbemeda in 24 patients with recurrent GBM demonstrated that a statistically significant improvement in overall survival correlated with both the average absorbed dose of radiation to the tumor and the percent volume of tumor treated. The treatment was safe and well tolerated without dose limiting toxicities.

The U.S. FDA granted both Orphan Drug designation and Fast Track designation to rhenium (186Re) obisbemeda for the treatment of GBM. More information about the ReSPECT-GBM trial may be found at ReSPECT-Trials.com and ClinicalTrials.gov (NCT01906385).

About Recurrent Glioblastoma (GBM)

GBM affects approximately 14,490 patients annually in the U.S. and is the most common and lethal form of brain cancer. The average life expectancy with GBM is less than 24 months, with a one-year survival rate of 40.8% and a five-year survival rate of only 6.9%. There is no clear standard of care for recurrent GBM and even the few currently approved treatments provide only marginal survival benefit and are associated with significant side effects, which limit dosing and prolonged use. Approximately 90% of patients experience GBM tumor recurrence at or near the original tumor location, yet there are no FDA-approved treatments in the recurrent or progressive setting that can significantly extend a patient’s life.

About Rhenium (186Re) obisbemeda

Rhenium (186Re) obisbemeda is a novel injectable radiotherapy specifically formulated to deliver highly targeted high dose radiation in CNS tumors in a safe, effective and convenient manner to optimize patient outcomes. Rhenium (186Re) obisbemeda has the potential to reduce risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue and gamma energy for live imaging.

On January 18, 2023 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported the positive recommendation from its safety review committee to advance to the fifth dose level (650 mg/m2) in its camsirubicin Phase 1b trial in patients with advanced soft tissue sarcoma (ASTS) (Press release, Monopar Therapeutics, JAN 18, 2023, View Source [SID1234626346]). This decision was made following a review of safety data from the patients in the first four dose cohorts.

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"Clearance to go to this higher dose level is an important milestone for the trial as this class of drugs is known to have dose-dependent anti-tumor activity," said Chandler Robinson, MD, Monopar’s Chief Executive Officer. "We continue to see a favorable safety profile compared to doxorubicin, and the Phase 1b data to-date shows an improvement in median progression free survival from what was observed in the prior camsirubicin Phase 2 trial (265mg/m2). We are looking forward to evaluating the 650 mg/m2 dose level, which is nearly 2.5x higher than the highest dose evaluated in any prior camsirubicin clinical trial."

Further information about this actively enrolling, open-label, dose-escalation Phase 1b clinical trial is available at www.ClinicalTrials.gov under study identifier NCT 05043649.

About Camsirubicin

Camsirubicin is a novel, proprietary analog of the widely used cancer drug doxorubicin. It has been previously investigated in ASTS patients in a Phase 1 and a single-arm Phase 2 clinical trial. In these studies, no camsirubicin-treated patients developed the irreversible cardiotoxicity common to doxorubicin at higher cumulative doses. The most frequent adverse event observed in the Phase 1 study was neutropenia, which was mitigated in the Phase 2 study using prophylactic G-CSF. Based on encouraging clinical results from prior clinical trials, the current Phase 1b trial is designed to test camsirubicin at progressively higher doses than previously administered while using concomitant prophylactic G-CSF to prevent neutropenia.

About Soft Tissue Sarcoma

Soft tissue sarcomas (STS) are a diverse type of cancer that typically develop in the connective tissue of the body. According to the American Cancer Society, in 2021, an estimated 13,460 new STS cases were diagnosed in the U.S. alone, and about 5,350 people will not survive their disease. These tend to be the advanced cases; those with sarcomas that are unresectable and/or have metastasized. The average life expectancy from time of diagnosis for those patients with advanced disease (ASTS) is about 12 to 15 months. Doxorubicin is the current standard of care in the 1st-line setting for ASTS, and has been for decades, since there have been no 1st-line therapeutic advancements that have improved overall survival for this patient population.

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