On December 9, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") reported that it will be participating in the Targeted Radiopharmaceuticals Summit being held virtually December 8th – 10th (Press release, Actinium Pharmaceuticals, DEC 9, 2020, View Source [SID1234572527]). During the event, Dale Ludwig, Ph.D., Actinium’s Chief Scientific and Technology Officer will participate in a panel titled "Beyond PSMA – How Do We Realistically Identify New Targets to Take on with Radioligand Therapy."

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"Targeted radiotherapy is witnessing a renaissance driven by strong clinical data emerging in multiple indications. This has resulted in acquisitions, new company formation and investments in new targets to pursue with radiotherapy," said Sandesh Seth, Actinium’s Chairman and CEO. He continued, "Actinium is proud to have not only established ourselves as a leader in the targeted radiotherapy field and is the only company pursuing well validated targets in hematology with late stage clinical programs. Both our Iomab-B and Actimab-A programs have yielded promising clinical data in the relapsed/refractory Acute Myeloid Leukemia (AML) setting that was prominently highlighted at ASH (Free ASH Whitepaper) demonstrating the potential of our ARCs for targeted conditioning in the case of Iomab-B and as a backbone for therapeutic combinations with Actimab-A. We intend to build upon our leadership position in targeted radiotherapy by leveraging our AWE technology platform including our gold standard linker, our robust IP portfolio and know-how, our clinical and supply chain expertise and our recently enhanced research infrastructure and capabilities. Our team is enthused to be spreading awareness about our activities with this exciting and valuable approach to drug development at this important industry conference."

Panel Details
Title: Beyond PSMA – How Do We Realistically Identify New Targets to Take on with Radioligand Therapy
Date: December 10, 2020
Time: 10:20 ET

Dr. Dale Ludwig, Actinium’s Chief Scientific and Technology Officer, said, "The targeted radiotherapy field has produced several successful drug candidates of late, however, they have been focused on a select number of targets and indications. It is critical that innovation continues to truly capitalize on the potential of targeted radiation. I look forward to highlighting Actinium’s efforts to leverage our AWE platform and clinical development experience to lead the field in its next evolution through the development of novel targeted radiotherapies for patients with unmet needs not addressed by current standards of care."

Members of Actinium’s executive and R&D teams will be in attendance at the virtual meeting. To schedule a meeting with Actinium please email [email protected].

This year, Actinium announced the expansion of its R&D capabilities with a new research facility to capitalization on the increased interest in targeted radiotherapy and to enhance its development of next generation Antibody Radiation Conjugates (ARC) candidates, ARC therapeutic combination strategies, and supporting AWE platform research collaborations. Actinium’s R&D efforts will employ a multidisciplinary approach leveraging its team’s expertise and experience in cancer cell biology, radiochemistry, radiation sciences, immunology and oncology drug development. The proprietary AWE technology platform, protected by over one hundred and thirty patents, is the foundation for Actinium’s R&D and exploits multiple different radioisotope payloads including the potent alpha-emitter, Actinium-225. Actinium is a leader in the field of targeted radiotherapy with several hundred patients treated to date with its ARCs, patent protection to 2037 and beyond for its drug candidates, patent protected "gold standard" linker technology, an established clinical supply chain at over 30 leading hospitals and a research collaboration with Astellas.

On December 9, 2020 NANOBIOTIX (Paris:NANO) (Euronext: NANO – ISIN : FR0011341205 – the ‘‘Company’’), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported the filing of an amended registration on form F-1 in connection with its intention to issue and sell, subject to market and other conditions, 6,500,000 ordinary shares of the Company in an initial public offering of American Depositary Shares ("ADSs"), each representing the right to receive one ordinary share, in the United States (the "U.S. Offering") and a concurrent offering of ordinary shares in certain jurisdictions outside the United States to certain investors (the "European Offering" and together with the U.S. Offering, the "Global Offering") (Press release, Nanobiotix, DEC 9, 2020, View Source [SID1234572546]). The offering price per ADS is expected to be between $13.50 and $14.50, or between €11.15 and €11.97 per ordinary share (assuming an exchange rate of €1.00 = $1.2109, the exchange rate published by the European Central Bank on December 9, 2020).

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Assuming an offering price of $14.00 per ADS in the U.S. Offering and €11.56 per ordinary share in the European Offering, which are the midpoints of the respective price ranges, the Company expects to receive net proceeds of approximately $79.6 million (€65.8 million) from the Global Offering. The Company intends to grant the underwriters a 30-day option to purchase, at the same price, additional ADSs and/or ordinary shares in an aggregate amount of up to 15% of the total number of ADSs and ordinary shares proposed to be sold in the Global Offering. If such option is exercised in full, the expected net proceeds to the Company will increase to approximately $92.3 million (€76.2 million).

All securities to be sold in the Global Offering will be offered by the Company. The ADSs have been approved for listing on the Nasdaq Global Select Market under the ticker symbol "NBTX." The Company’s ordinary shares are listed on the regulated market of Euronext Paris under the ticker symbol "NANO."

The Company plans to use the net proceeds of the Global Offering to advance the overall development of NBTXR3, prioritizing the treatment of locally advanced head and neck cancers, including approximately $58.1 million to advance its clinical trial of NBTXR3 in the United States and Europe for the treatment of locally advanced head and neck cancers through an interim analysis of efficacy data, and approximately $20.6 million to advance the development of its other clinical and pre-clinical programs. The Company expects to use the remainder of the net proceeds, if any, from the Global Offering for working capital funding and other general corporate purposes.

The Company expects that the net proceeds from the Global Offering, together with its cash and cash equivalents of €42.4 million as of September 30, 2020, will be sufficient to fund its operating expenses and capital expenditure requirements through the end of 2022.

Jefferies LLC is acting as global coordinator and joint book-running manager for the Global Offering, and Evercore Group, L.L.C. and UBS Securities LLC are acting as joint book-running managers for the U.S. Offering. Jefferies International Limited and Gilbert Dupont are acting as managers for the European Offering.

The final offering price per ADS in U.S. dollars and the corresponding offering price per ordinary share in euros, as well as the final number of ADSs and ordinary shares to be sold in the Global Offering, will be determined by the Company’s executive board following a bookbuilding process commencing immediately. The offering price per ADS and per ordinary share will be at least equal to the volume weighted average price of the Company’s ordinary shares on the regulated market of Euronext in Paris over the last three trading days preceding the start of the offering (i.e., December 7, 8 and 9, 2020), subject to a maximum discount of 10%.

On an indicative basis, the completion of the Global Offering would result in a dilution of approximately 25% of the Company’s outstanding share capital on a non-diluted basis (excluding the exercise by the underwriters of the option to purchase additional ordinary shares) and approximately 28% of the Company’s outstanding share capital on a non-diluted basis (in the event that the underwriters exercise in full their option to purchase additional ordinary shares (including in the form of ADSs)).

The ADSs and/or ordinary shares will be issued through a capital increase without shareholders’ preferential subscription rights by way of a public offering excluding offerings referred to in Article L. 411-2 1° of the French Monetary and Financial Code (Code monétaire et financier) and under the provisions of Article L.225-136 of the French Commercial Code (Code de commerce) and pursuant to the 2nd and 7th resolutions of the Company’s extraordinary general shareholders’ meeting held on November 30, 2020. The European Offering will be open only to qualified investors as such term is defined in article 2(e) of the regulation (EU) 2017/1129 of the European Parliament and of the Council of June 14, 2017.

The Company plans to announce the result of the Global Offering as soon as practicable after pricing thereof in a subsequent press release.

The existing liquidity contract between the Company and Gilbert Dupont is suspended until the end of the stabilization period.

The securities referred to in this press release will be offered only by means of a prospectus. When available, copies of the preliminary prospectus relating to and describing the terms of the Global Offering may be obtained from Jefferies LLC, 520 Madison Avenue New York, NY 10022, or by telephone at 877-547-6340 or 877-821-7388, or by email at [email protected]; or from Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, or by telephone at 888-474-0200, or by email at [email protected]; or from UBS Securities LLC, Attention: Prospectus Department, 1285 Avenue of the Americas, New York, New York 10019, or by telephone at 888-827-7275, or by email at [email protected].

A registration statement on Form F-1 relating to the securities referred to herein has been filed with the U.S. Securities and Exchange Commission ("SEC") but has not yet become effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration statement becomes effective. The registration statement can be accessed by the public on the website of the SEC.

On December 9, 2020 Novartis reported new Kisqali (ribociclib) data demonstrating consistent efficacy benefit with Kisqali plus endocrine therapy across the main intrinsic subtypes of hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer (Press release, Novartis, DEC 9, 2020, View Source [SID1234572512]). The largest biomarker analysis of efficacy in intrinsic subtypes evaluated whether there was a correlation between these subtypes and efficacy outcomes in patients treated with Kisqali across the three Phase III MONALEESA trials1. The findings will be presented in an oral presentation at the 2020 San Antonio Breast Cancer Virtual Symposium.

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This broad ad hoc exploratory analysis showed that Kisqali plus endocrine therapy consistently provided significant progression-free survival (PFS) benefit across three of four subtypes of HR+/HER2- metastatic breast cancer (LumA HR=0.63; p<.001, LumB HR=0.52; p<.001, HER2-enriched HR=0.39; p<.001, Basal-like HR=1.15; p=.7672)1. The largest PFS benefit was seen in patients with the HER2-enriched subtype – a non-luminal subtype associated with endocrine resistance and poor prognosis1. In contrast, benefit was not observed with palbociclib in the HER2-enriched subtype in a retrospective analysis of the PALOMA-2 trial presented at the 2017 San Antonio Breast Cancer Symposium 7.

"The significant benefit seen with ribociclib in the endocrine-resistant HER2-enriched subtype is a unique and important finding, differentiating it from the other CDK4/6 inhibitors. The underlying hypothesis is that ribociclib induces hormone-sensitivity in this group of tumors beyond inhibition of the cell-cycle," said Aleix Prat, Head, Department of Medical Oncology, Hospital Clinic, Barcelona, Spain. "The body of preclinical data showing that ribociclib has the ability to more selectively target and inhibit CDK4, which is a key driver of disease progression in breast cancer, may help us understand why ribociclib provides consistent benefit, especially in patients with more aggressive disease."

The four intrinsic subtypes of breast cancer (Luminal A, Luminal B, HER2-enriched and basal-like) have revealed critical differences in terms of incidence, survival and response to treatment8-12. Additionally, the insights provided by intrinsic subtypes complement and expand upon the information provided by standard clinical parameters and pathological markers. The observed benefit with Kisqali provides reassurance about it as a treatment choice for the majority of people with metastatic breast cancer without the need for additional testing.

Preclinical CDK4/6 Study
Additional data to be presented at SABCS include a poster presentation of a preclinical analysis in which cellular models were used to examine the effects of CDK4/6 inhibitors on either CDK4 or CDK6. The preclinical in vivo study confirmed previously published biochemical in vitro and proliferation data, demonstrating that Kisqali selectively inhibits CDK4, whereas palbociclib has similar activity against both CDK4 and CDK6 in cells3. Kisqali inhibited CDK4 at 11-fold and 9-fold lower drug concentrations than CDK6, whereas palbociclib inhibited CDK4 at 2-fold lower drug concentrations than CDK6 in both cell lines3.

"The data presented at SABCS show that Kisqali offers a superior benefit for metastatic breast cancer patients, even in those with the HER2-enriched subtype, who face a very poor prognosis. These data build on previous findings showing Kisqali provides a benefit regardless of type of metastases, endocrine partner or menopausal status. The totality of evidence to date gives us incredible confidence that Kisqali is unique among CDK4/6 inhibitors, and we believe doctors should consider Kisqali for their patients," said Susanne Schaffert, PhD, President of Novartis Oncology.

About Kisqali (ribociclib)
Kisqali is the CDK4/6 inhibitor with the largest body of first-line clinical trial evidence demonstrating consistent and sustained efficacy compared to endocrine therapy alone. Kisqali is the only CDK4/6 inhibitor to achieve statistically significant OS in two Phase III trials with two distinct patient populations4-6. The substantial OS benefit and improved quality of life observed in MONALEESA-7 support the ESMO (Free ESMO Whitepaper) Magnitude of Clinical Benefit Scale (MCBS) perfect five out of five rating for Kisqali plus endocrine therapy in premenopausal HR+/HER2- MBC13. Kisqali also received an ESMO (Free ESMO Whitepaper)-MCBS score of four out of five, the highest score achieved by any CDK 4/6 inhibitor in combination with fulvestrant, for first-line postmenopausal patients based on the statistically significant OS benefit observed in MONALEESA-3 and maintained quality of life13. Overall survival follow-up is ongoing for the Phase III MONALEESA-2 trial.

Kisqali was initially approved by the US Food and Drug Administration (FDA) in March 2017 and by the European Commission (EC) in August 2017, as initial endocrine-based therapy for postmenopausal women with HR+/HER2- locally advanced or metastatic breast cancer in combination with an aromatase inhibitor based on findings from the pivotal MONALEESA-2 trial. Kisqali in combination with an aromatase inhibitor was approved for the treatment of pre-, peri- or postmenopausal women as initial endocrine based therapy, and also indicated for use in combination with fulvestrant as both first- or second-line therapy in postmenopausal women by the FDA in July 2018 and by the EC in December 2018. Regulatory filings are underway with other health authorities worldwide.

Novartis is continuing to reimagine cancer by investigating Kisqali in early breast cancer. The NATALEE study is a Phase III clinical trial of Kisqali with endocrine therapy in the adjuvant treatment of HR+/HER2- early breast cancer being conducted in collaboration with Translational Research In Oncology (TRIO).

Kisqali was developed by the Novartis Institutes for BioMedical Research (NIBR) under a research collaboration with Astex Pharmaceuticals.

Important Safety Information from the Kisqali EU SmPC
Kisqali (ribociclib) is a prescription medicine approved in combination with an aromatase inhibitor as initial endocrine – based therapy in women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer or fulvestrant as initial endocrine – based therapy or following disease progression on endocrine therapy in postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. It is not known if Kisqali is safe and effective in children or adolescents. Kisqali can cause a heart problem known as QT prolongation. This condition can cause an abnormal heartbeat and may lead to death. Kisqali is not indicated for concomitant use with tamoxifen due to an increased risk of QT prolongation. Patients should tell their health care provider right away if they have a change in their heartbeat (a fast or irregular heartbeat), or if they feel dizzy or faint. Kisqali can cause serious liver problems. Patients should tell their health care provider right away if they get any of the following signs and symptoms of liver problems: yellowing of the skin or the whites of the eyes (jaundice), dark or brown (tea-colored) urine, feeling very tired, loss of appetite, pain on the upper right side of the stomach area (abdomen), and bleeding or bruising more easily than normal. Low white blood cell counts are very common when taking Kisqali and may result in infections that may be severe. Patients should tell their health care provider right away if they have signs and symptoms of low white blood cell counts or infections such as fever and chills. Before taking Kisqali, patients should tell their health care provider if they are pregnant, or plan to become pregnant as Kisqali can harm an unborn baby. Females who are able to become pregnant and who take Kisqali should use highly effective birth control during treatment and for at least 3 weeks after the last dose of Kisqali. Do not breastfeed during treatment with Kisqali and for at least 3 weeks after the last dose of Kisqali. Patients should tell their health care provider about all of the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements since they may interact with Kisqali. Patients should avoid grapefruit or grapefruit juice while taking Kisqali. The most common side effects (incidence >=20%) include infections, white blood cell count decreases, headache, cough, nausea, tiredness, diarrhea, vomiting, constipation, hair loss and rash. The most common Grade 3/4 side effects (incidence >5%) were infections, low neutrophils, low leukocytes, low red blood cells, abnormal liver function tests, low lymphocytes, low phosphate levels and vomiting. Abnormalities were observed in hematology and clinical chemistry laboratory tests.

About Novartis in Advanced Breast Cancer
Novartis tackles breast cancer with superior science, collaboration and a passion for transforming patient care. We’ve taken a bold approach to our research by including patient populations often neglected in clinical trials, identifying new pathways or mutations that may play a role in disease progression and developing therapies that not only maintain, but also improve, quality of life for patients. Our priority over the past 30 years and today is to deliver treatments proven to improve and extend lives for those diagnosed with advanced breast cancer.

On December 9, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, reported that it will be providing an update on the Phase 3 clinical study of omidubicel, commercial readiness plan and pipeline at its virtual Pipeline Deep Dive event (Press release, Gamida Cell, DEC 9, 2020, View Source [SID1234572528]).

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"Our goal with omidubicel is to revolutionize the field of bone marrow transplantation and bring a potentially curative cell therapy option to thousands of patients who are in need of a bone marrow transplant, but lack a suitable stem cell donor. These results bring us one step closer towards that goal," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "What’s more, transplantation with omidubicel has been shown to result in more rapid neutrophil engraftment, a decrease in the amount of time patients spend in hospital, and a reduction in infections. These are very meaningful outcomes for patients and may also lessen the financial costs of certain aspects of the transplant."

Gamida Cell previously reported top-line data for omidubicel. In October, the company reported that the omidubicel phase 3 study achieved its secondary endpoints, analyzed in all randomized patients (intent-to-treat). In May, Gamida Cell reported that the study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a bone marrow transplant.

These pivotal data form the basis of a Biologics License Application (BLA) that Gamida Cell expects to initiate on a rolling basis before the end of this year. Gamida Cell is preparing to be launch ready in anticipation of potential FDA approval as early as the fourth quarter of 2021, subject to ongoing FDA discussions on manufacturing, quality and other matters.

The live event will be available here. More information about the Phase 3 study of omidubicel and the other updates included in this release can be found in the Pipeline Deep Dive presentation on the Gamida Cell website immediately following the event.

Details of Phase 3 Endpoints

As previously reported, Gamida Cell achieved positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001). Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.

Today, Gamida Cell announced the details of achieving all three of the prespecified secondary endpoints of the study, analyzed in all randomized patients (intent-to-treat). These secondary endpoints were the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant. All three secondary endpoints demonstrated statistical significance in an intent-to-treat analysis.

Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 28 percent for the comparator (p = 0.028).
Infection rates were significantly reduced for patients randomized to omidubicel. The cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel was 37 percent, compared to 57 percent for the comparator (p = 0.027).
Total days in hospital were reduced in patients randomized to omidubicel. The median number of days alive and out of hospital for patients randomized to omidubicel was 60.5 days, compared to 48.0 days for the comparator (p = 0.005).
Additionally, Gamida Cell reported that the exploratory endpoints in the study demonstrated a reduction in the cumulative incidence of viral infections.

The international, multi-center, randomized Phase 3 study for omidubicel was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant.

The company anticipates reporting the full data set in a peer-reviewed setting in the first half of 2021.

Commercial Readiness

The company discussed the market potential for omidubicel and launch plans. These included quantifying the market opportunity and keys aspects for a successful launch.

As it prepares for the potential commercial launch of omidubicel, the company also announced plans for the Gamida Cell Assist program, which has been designed to focus on patient access and support of every individual and their caregiver at each step of the transplant process. Once the program is launched, the Gamida Cell Assist case management team would provide a consistent, single point of contact for patients and health care professionals. This team would work with the transplant center to track each individual patient’s omidubicel therapy and provide real-time updates on the status of the therapy. Gamida Cell Assist is also designed to provide additional services, including coverage and reimbursement support, and patient and caregiver support, which may include financial, travel, and lodging assistance.

"At Gamida Cell we are inspired to cure, with the goal of pioneering new standards of care for patients with blood cancers and serious blood diseases," said Michele Korfin, chief operating and chief commercial officer of Gamida Cell. "The transplant process can be challenging and complex for the patient, caregivers and the entire transplant care team. As we prepare for commercialization, we have developed Gamida Cell Assist to serve as a comprehensive support program to focus on assuring a positive patient experience with omidubicel. We are committed to supporting patients and their caregivers during every step of their journey and enabling what matters most, a successful clinical outcome that makes a meaningful difference for patients."

Update on Natural Killer Cell Therapy GDA-201

In an oral presentation at the recent American Society of Hematology (ASH) (Free ASH Whitepaper) 62nd Annual Meeting, it was shown that GDA-201 was well tolerated and no dose limiting toxicities were observed in the Phase 1 clinical study. GDA-201 demonstrated significant clinical activity in patients with non-Hodgkin lymphoma, with 13 complete responses and one partial response observed in 19 patients, for a response rate of 74 percent. Full details of the presentation can be found in the press release.

Phase 2 Study of Omidubicel in Patients with Severe Aplastic Anemia

In a poster presentation at ASH (Free ASH Whitepaper), it was shown that patients with severe aplastic anemia treated with omidubicel achieved sustained early engraftment. These data, which were presented on December 5 by Mohamed Samour, M.D., Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, are the first evidence that omidubicel can result in rapid engraftment and can achieve sustained hematopoiesis in patients who are at high risk for graft failure with conventional umbilical cord blood transplant.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.12 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the U.S. Food and Drug Administration or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.3 For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy has not been established by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic Platform

Gamida Cell’s proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types — including stem cells and natural killer (NK) cells — with appropriate growth factors to maintain the cells’ original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

On December 9, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00 per share (Press release, Rocket Pharmaceuticals, DEC 9, 2020, View Source [SID1234572547]). The gross proceeds to Rocket from the offering are expected to be approximately $260 million, before deducting the underwriting discounts and commissions and other offering expenses. Rocket has granted the underwriters a 30-day option to purchase up to an additional 696,428 shares of its common stock.

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All the shares in the offering are to be sold by Rocket. The offering is expected to close on or about December 14, 2020, subject to satisfaction of customary closing conditions. Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes. J.P. Morgan, BofA Securities, SVB Leerink and Piper Sandler are acting as the joint bookrunning managers for the public offering.

The public offering is being made by Rocket pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC. A final prospectus supplement relating to and describing the terms of this offering will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, from BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at [email protected], from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at [email protected], or from Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected]. You may also obtain these documents free of charge by visiting the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these 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.