On March 31, 2021 Sanofi reported that The U.S. Food and Drug Administration (FDA) has approved Sarclisa (isatuximab-irfc) in combination with carfilzomib and dexamethasone (Kd), for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received one to three prior lines of therapy (Press release, Sanofi, MAR 31, 2021, View Source [SID1234577459]).

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"In the Phase 3 IKEMA study, the addition of Sarclisa to carfilzomib and dexamethasone reduced risk of disease progression or death by 45%," said Thomas G. Martin, M.D., Associate Director, Myeloma Program, The University of California, San Francisco, Professor of Medicine, Adult Leukemia and Bone Marrow Transplantation Program and co-leader of the Hematopoietic Malignancies Program, Helen Diller Family Comprehensive Cancer Center. "This approval is an important advancement for patients whose disease has relapsed and reinforces the potential for Sarclisa to become a standard of care in relapsed or refractory multiple myeloma."

This marks the second FDA approval for Sarclisa, which is also approved in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of adults with RRMM who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.

"Treatment of patients with relapsed or refractory multiple myeloma remains challenging and the prognosis for patients experiencing multiple relapses unfortunately is poor," said Peter C. Adamson, M.D., Global Development Head, Oncology and Pediatric Innovation at Sanofi. "With this approval, Sarclisa is now included in two standard of care regimens for the treatment of patients with multiple myeloma as early as first relapse. Today’s milestone further supports our ambition for Sarclisa to become the anti-CD38 of choice for patients with relapsed or refractory multiple myeloma."

Sarclisa Phase 3 IKEMA pivotal trial results supporting approval

The FDA approval is based on data from the Phase 3 IKEMA study, a randomized, multi-center, open label clinical trial that enrolled 302 patients with relapsed multiple myeloma across 69 centers spanning 16 countries.1 In this study, Sarclisa added to Kd (Sarclisa combination therapy) reduced the risk of disease progression or death by 45% (hazard ratio 0.548, 95% CI 0.366-0.822, p=0.0032) versus standard of care Kd alone in patients with multiple myeloma. The median progression free survival (PFS) for Sarclisa combination therapy was not reached at the time of the pre-planned interim analysis. This study enrolled a difficult-to-treat patient population, including those who are elderly, have high cytogenetic risk or renal impairment. Overall, demographic and disease characteristics at baseline were balanced between the two treatment groups.2

Secondary endpoints of the IKEMA trial assessed the overall response rate (ORR) for Sarclisa combination therapy compared to Kd, including complete response (CR) and very good partial response (VGPR). There was no statistically significant difference in ORR, which remained similar for each arm at 86.6% for the Sarclisa combination therapy versus 82.9% for Kd (p=0.3859). The rate of CR was 39.7% in the Sarclisa combination therapy arm and 27.6% in the Kd arm. The rate of VGPR was 33% for patients receiving Sarclisa combination therapy and 28.5% for patients receiving Kd.2 At the time of the interim analysis, overall survival (OS) data were still immature.3

The most frequent adverse reactions (occurring in 20% or more of patients) for Sarclisa versus the control arm were upper respiratory tract infection (67% vs. 57%), infusion-related reactions (46% vs. 3.3%), fatigue (42% vs. 32%), hypertension (37% vs. 32%), diarrhea (36% vs. 29%), pneumonia (36% vs. 30%), dyspnea (29% vs. 24%), bronchitis (24% vs. 13%), and cough (23% vs. 15%). Serious adverse reactions that occurred in more than 5% of patients who received Sarclisa combination therapy were pneumonia (25%) and upper respiratory tract infections (9%). Permanent discontinuation of treatment because of adverse reactions (Grade 1-4) occurred in 8% of patients treated with Sarclisa combination therapy, and 2.8% of patients discontinued due to an infection.2

Multiple Myeloma: an incurable blood cancer, with significant burden

Multiple Myeloma (MM) is the second most common hematologic malignancy4, affecting more than 130,000 patients in the United States; approximately 32,000 Americans are diagnosed with multiple myeloma each year.5 Despite available treatments, MM remains an incurable malignancy, and is associated with significant patient burden. Since MM does not have a cure, most patients will relapse. Relapsed MM is the term for when the cancer returns after treatment or a period of remission. Refractory MM refers to when the cancer does not respond or no longer responds to therapy.

About Sarclisa

Sarclisa is a monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa.

This marks the second FDA approval for Sarclisa since March 2020 and comes more than three months ahead of the FDA’s target action date. In February, the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a positive opinion for a second indication for Sarclisa, in combination with carfilzomib and dexamethasone (Kd), for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. The use of Sarclisa in combination with Kd is not currently approved in the European Union (EU), but the final decision whether to expand the indication is expected from the European Commission in the coming months. In Europe, Sarclisa is indicated in combination with pom-dex for the treatment of adult patients with RRMM who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy. Outside of the U.S. and the EU, Sarclisa is approved in Switzerland, Canada, Australia, Japan, Russia, the UAE, South Korea, Taiwan and Brazil in combination with pom-dex for the treatment of certain adults with RRMM.

Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard and novel treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies and solid tumors. The use of Sarclisa in these additional settings is currently under clinical investigation and its safety and efficacy have not been fully evaluated by any regulatory authority.

IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS

What is SARCLISA?

SARCLISA is a prescription medicine used in combination with:

The medicines pomalidomide and dexamethasone, to treat adults who have received at least 2 prior therapies including lenalidomide and a proteasome inhibitor to treat multiple myeloma.
The medicines carfilzomib and dexamethasone, to treat adults with multiple myeloma who have already received 1 to 3 lines of treatment and they did not work or are no longer working.
It is not known if SARCLISA is safe and effective in children.

Important Safety Information

Do not receive SARCLISA if you have a history of a severe allergic reaction to isatuximab-irfc or any of the ingredients in SARCLISA (see the list of ingredients in the full Prescribing Information).

Before receiving SARCLISA, tell your healthcare provider about all of your medical conditions, including if you:

Have heart problems, if your healthcare provider prescribes SARCLISA in combination with carfilzomib and dexamethasone for you.
Are pregnant or plan to become pregnant. SARCLISA may harm your unborn baby. You should not receive SARCLISA during pregnancy.
Females who are able to become pregnant should use an effective method of birth control during treatment and for 5 months after your last dose of SARCLISA. Talk to your healthcare provider about birth control methods that you can use during this time.
Tell your healthcare provider right away if you think you are pregnant or become pregnant during treatment with SARCLISA.
Are breastfeeding or plan to breastfeed. It is not known if SARCLISA passes into your breast milk. You should not breastfeed during treatment with SARCLISA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines. Especially tell your healthcare provider if you have ever taken a medicine for your heart.

How will I receive SARCLISA?

SARCLISA will be given to you by your healthcare provider by intravenous (IV) infusion into your vein.
SARCLISA is given in treatment cycles of 28 days (4 weeks), together with either the medicines pomalidomide and dexamethasone, or carfilzomib and dexamethasone.
In cycle 1, SARCLISA is usually given weekly.
Starting in cycle 2, SARCLISA is usually given every 2 weeks.
If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment.
Your healthcare provider will give you medicines before each dose of SARCLISA to help reduce the risk of infusion reactions (make them less frequent and severe).
What are the possible side effects of SARCLISA?

SARCLISA may cause serious side effects, including:

Infusion reactions. Infusion reactions are common with SARCLISA and can sometimes be severe or life threatening.
Your healthcare provider will prescribe medicines before each infusion of SARCLISA to help decrease your risk for infusion reactions or to help make any infusion reaction less severe. You will be monitored for infusion reactions during each dose of SARCLISA.
Your healthcare provider may slow down or stop your infusion, or completely stop treatment with SARCLISA if you have an infusion reaction.
Get medical help right away if you develop any of the following symptoms of infusion reaction during or after an infusion of SARCLISA:

— shortness of breath, wheezing, or trouble breathing
— swelling of the face, mouth, throat, or tongue
— throat tightness
— palpitations
— dizziness, lightheadedness, or fainting
— headache
— cough
— rash or itching
— nausea
— runny or stuffy nose
— chills

Decreased white blood cell counts. Decreased white blood cell counts are common with SARCLISA and certain white blood cells can be severely decreased. You may have an increased risk of getting certain infections, such as upper and lower respiratory tract infections and urinary tract infections.

Your healthcare provider will check your blood cell counts during treatment with SARCLISA. Your healthcare provider may prescribe an antibiotic or antiviral medicine to help prevent infection, or a medicine to help increase your white blood cell counts during treatment with SARCLISA.
Tell your healthcare provider right away if you develop any fever or symptoms of infection during treatment with SARCLISA.

Risk of new cancers. New cancers have happened in people during treatment with SARCLISA. Your healthcare provider will monitor you for new cancers during treatment with SARCLISA.
Change in blood tests. SARCLISA can affect the results of blood tests to match your blood type. Your healthcare provider will do blood tests to match your blood type before you start treatment with SARCLISA. Tell all of your healthcare providers that you are being treated with SARCLISA before receiving blood transfusions.
Heart failure. Heart failure can happen during treatment with SARCLISA in combination with carfilzomib and dexamethasone. Tell your healthcare provider right away if you develop any of the following symptoms:
– trouble breathing

– cough

– swelling of your ankles, feet, or legs

The most common side effects of SARCLISA in combination with pomalidomide and dexamethasone include:

lung infection (pneumonia)
decreased red blood cell counts (anemia)
upper respiratory tract infection
decreased platelet counts (thrombocytopenia)
diarrhea
The most common side effects of SARCLISA in combination with carfilzomib and dexamethasone include:

upper respiratory tract infection
tiredness and weakness
high blood pressure
diarrhea
lung infection (pneumonia)
trouble breathing
trouble sleeping
bronchitis
cough
back pain
decreased red blood cells (anemia)
decreased platelet counts (thrombocytopenia)
These are not all the possible side effects of SARCLISA. For more information, ask your healthcare provider or pharmacist.

On March 31, 2021 CTI BioPharma Corp. (Nasdaq: CTIC) ("CTI"), a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies covering a spectrum of blood-related cancers, reported the pricing of its previously announced underwritten public offering of 14,260,800 shares of its common stock and 600 shares of its series X1 preferred stock (the "Series X1 Preferred") (Press release, CTI BioPharma, MAR 31, 2021, View Source [SID1234577458]). In addition, CTI has granted the underwriters a 30-day option to purchase up to an additional 2,139,120 shares of its common stock on the same terms and conditions. The offering price to the public of a share of common stock is $2.50 and the offering price to the public of a share of Series X1 Preferred Stock is $25,000. Each share of Series X1 Preferred will be convertible into 10,000 shares of common stock at the election of the holder, subject to beneficial ownership conversion limits applicable to the Series X1 Preferred.

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All of the securities in the offering are being sold by CTI. The gross proceeds to CTI from this underwritten public offering are expected to be approximately $50.7 million, before deducting the underwriting discount and other estimated offering expenses payable by CTI. The offering is expected to close on or about April 6, 2021, subject to the satisfaction of customary closing conditions.

CTI intends to use the proceeds from the proposed sale of its shares of common stock and Series X1 Preferred Stock for commercialization activities for pacritinib, general working capital and corporate purposes.

Stifel and JMP Securities are acting as joint book-running managers for the offering. BTIG is acting as lead manager for the offering.

The offering is being made pursuant to a registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and subsequently was declared effective by the SEC. A preliminary prospectus supplement and accompanying base prospectus relating to and describing the terms of the offering has been filed with the SEC and a final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s web site at www.sec.gov. When available, copies of the final prospectus relating to these securities may be obtained by sending a request to Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery St, Suite 3700, San Francisco, CA 94104, by telephone at (415) 364-2720 or by email at [email protected], or JMP Securities LLC, 600 Montgomery St, Suite 1100, San Francisco, CA 94111, Attn: Prospectus Department, telephone: 415-835-8900.

This press release does not constitute an offer to sell or the solicitation of an offer to buy any of these securities, nor will 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 other jurisdiction.

On March 31, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported the completion of its previously-announced sale of a portfolio of select products to Orifarm Group ("Orifarm") for a total value of up to $670 million USD (Press release, Takeda, MAR 31, 2021, View Source [SID1234577457]). The portfolio includes approximately 130 over-the-counter (OTC) and prescription pharmaceutical products sold in Europe, and two manufacturing sites located in Denmark and Poland. This divestment agreement was first announced in April 2020.

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The divested portfolio includes OTC products and food supplements, plus select products within the Cardiovascular, Anti-inflammatory, Respiratory, and Endocrinology therapeutic areas, primarily sold in Denmark, Norway, Belgium, Poland, Finland, Sweden, the Baltics and Austria. The portfolio generated FY2020 net sales of approximately $240 million USD, driven by sales of cough/cold and vitamin OTC brands, along with prescription products such as Warfarub and Levaxin, and other strong regional brands. The products, while addressing key patient needs in these countries, are outside of the business areas Takeda has chosen as core to its global long-term growth.

As previously announced, Takeda and Orifarm have also entered into manufacturing and supply agreements under which Takeda will continue to manufacture select products on behalf of Orifarm. In addition, approximately 600 employees from the manufacturing sites, sales and marketing professionals and others supporting the divested portfolio and manufacturing sites have transitioned to Orifarm.

Takeda remains focused on executing its long-term growth strategy to optimize our business mix around our key business areas, and simplifying our operations to better serve patients by delivering innovative treatments in these areas.

The Company intends to use the proceeds from the sale to reduce its debt and accelerate deleveraging towards its target of 2x net debt/adjusted EBITDA within Fiscal Years 2021–2023.

Takeda has exceeded its $10 billion non-core asset divestiture target and has announced 12 deals since January 2019 to date for a total aggregate value of up to approximately $12.9 billion.

On March 31, 2021 Kiromic BioPharma (NASDAQ: KRBP), a pre-clinical stage biotechnology company using its proprietary DIAMOND artificial intelligence ("A.I.") platform to improve drug discovery and development with a therapeutic focus on immune-oncology, reported its financial results for the year ended December 31, 2020, and provided an update on its corporate developments (Press release, Kiromic, MAR 31, 2021, View Source [SID1234577456]).

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"Kiromic BioPharma achieved important scientific and operational milestones during the year that we believe have us well positioned for preparing our staff and our facilities for the first in-human dosing in Q3 2021," said Dr. Maurizio Chiriva-Internati, CEO and President of Kiromic BioPharma. "We are thankful to our employees and collaborators who have maintained this high level of execution in the middle of the hard challenges posed by the COVID-19 pandemic."

Our approach and goal is to defeat cancer by developing immunotherapies that rely on improving target discovery and validation. With better targets, we believe our therapies will be more effective than the current array of immunotherapies using older targets.

Corporate and Scientific Highlights

Initial Public Offering Completion – On October 20, 2020, we completed our IPO, raising $15 million of gross proceeds, significantly strengthening the Company’s balance sheet to support the continued development of our promising pipeline of targeted cancer therapies.

Two IND Application Filings – On December 17, 2020 we filed two applications with the U.S. Food and Drug Administration ("FDA"). The first IND was for a Phase 1 clinical trial of intravenously ("IV") administered allogeneic CAR-T for epithelial ovarian carcinoma ("EOC") and malignant pleural mesothelioma ("MPM"). The second IND was for a Phase 1 clinical trial of an intrapleural/intraperitoneal (IP) administered allogeneic CAR-T for EOC and MPM.

Since filing the original INDs in December 2020, the Company has had communications with the FDA, and numerous consults with scientific board and clinical advisors regarding resubmission. In March 2021, we announced that we planned to resubmit the two INDs.

The revised INDs will be for first in-human dosing of our Off-the-Shelf, Allogeneic Gamma-Delta T cell therapy for metastatic and progressive locally advanced solid malignancies.

The revised INDs have protocols which retain approximately 80% of the original INDs.

Longwood University Licensing Agreement – On November 30, 2020, we executed a licensing agreement for chPD1 with Longwood University. This marks a major milestone for Kiromic CAR-T development. With chPD1, we believe our chimeric PD1 CAR-T will be able to overcome the challenging tumor micro-environment (TME) which has plagued other CAR-T programs, while making Kiromic the only CAR-T development program with a built-in capability to meet other CAR-T programs head-on who do not have a bundled chPD1 CAR-T.

GMP Facility Completion – As of September 30, 2020, the key features of the GMP facility have been completed, clearing the path for the production of off-the-shelf Gamma-Delta-T cells, a novel approach to CAR-T cell therapy, which will be evaluated in the upcoming clinical trials.
FY 2020 Financial Highlights

Cash Position: Cash and cash equivalents were $10,150,500 as of December 31, 2020, compared to $1,929,100 as of December 31, 2019. The increase was primarily due to cash inflows of $15,805,600 attributable to financing activities related to the issuance of common stock from the initial public offering, issuance of Series B Preferred Stock and proceeds net of repayments from the Paycheck Protection Program loan. These inflows were offset by outflows of $6,126,600 and $1,457,600 attributable to operating activities and investing activities, respectively.

R&D Expenses: Research and development expenses were $5,052,900 for the year ended December 31, 2020, compared to $1,201,700 for the year ended December 31, 2019. The increase was primarily attributable to augmented headcount, increased square footage to our Houston, TX leased facilities, in-vitro experimentation costs, and intellectual property costs.

G&A Expenses: General and administrative expenses were $14,144,000 for the year ended December 31, 2020, compared to $2,503,700 for the year ended December 31, 2019. This increase was primarily due to increased stock compensation expenses and personnel expenses.

Net Loss: Net loss was $19,200,200 for the year ended December 31, 2020, compared to a net loss of $3,727,900 for the year ended December 31, 2019.

Dr. Chiriva-Internati continued, "Developing live-cell therapies by leveraging artificial intelligence is central to transforming the cost and efficiency of the immune-oncology field and improving the potential for off-the-shelf therapies for cancer patients. We believe our approach will help us design more efficient pre-clinical validation studies and more targeted clinical trials, thereby accelerating our drug candidates’ time to approval and eventually to market. DIAMOND is central to our process in achieving this outcome rapidly and with reduced costs," concluded Dr. Chiriva-Internati.

On March 31, 2021 Guided Therapeutics, Inc. (OTCQB: GTHP), the maker of a rapid and painless testing platform based on its patented biophotonic technology, reported that it had closed its Series F Preferred Stock Financing on March 26, 2021 (Press release, Guided Therapeutics, MAR 31, 2021, View Source [SID1234577454]). This oversubscribed sale of 2,114 Series F Preferred Shares resulted in gross proceeds to the Company of $2,114,000, netting approximately $1,980,000 after legal costs, fees and commissions of approximately $134,000 (6.3%). In addition, as board approved non-cash transactions, three of the Company’s officers exchanged and forgave a total of $321,554 in debt owed to them by the Company into 235 Series F Preferred Shares under the same terms as the non-affiliated cash investors.

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The two most important uses of proceeds from the Series F Financing included 1) making the final payment of $750,000 to GPB Holdings, LLC, completed on January 8, 2021, and 2) allocate most of the remainder to completing the U.S. FDA clinical trial for the LuViva Advanced Cervical Scan. The clinical sites currently are reviewing the study protocol that was developed by the Company, which implemented recommendations made by the U.S. FDA. It is expected the trial will begin in the second quarter of 2021. The Company’s primary objective this year is completing the clinical trial of approximately 400 women and filing the results with the FDA.

The Series F Preferred shares are convertible at a fixed price of 25 cents per share, carry a 6% annual dividend and could potentially include an additional dividend of 15% after twelve months if the Company is unable to uplist to a national exchange or complete its FDA study. The dividends can be paid in cash or common shares at the option of the Company. Aspen Capital acted as Financial Advisor to the Company with respect to certain Canadian investments.

"Now that the Series F Financing has closed, netting sufficient funds, we can turn our full attention to completing the FDA study without delay," said Gene Cartwright, Guided Therapeutics CEO, adding, "At the same time, we have made great strides in cleaning up our balance sheet and have significantly reduced our liabilities, thereby setting the stage for a potential uplist to a national exchange, concomitant with the U.S. FDA study and significant progress in China."