On December 9, 2020 Oblato, Inc. (the Company), a wholly owned U.S. subsidiary of the Korean biotech company GtreeBNT Co., Ltd., reported it had official discussions with the FDA on a detailed plan for a phase 1/2 clinical trial to start developing a new treatment for Diffuse Intrinsic Pontine Glioma (DIPG), a rare pediatric disease, using its proprietary drug, OKN-007 (Press release, Oblato, DEC 9, 2020, View Source [SID1234572548]).

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Through this FDA meeting, the Company obtained consent from the FDA on important matters, such as patient population, starting dose, approach for dose escalation, and criteria for evaluating disease response. Importantly, there was in depth discussion on how to actively utilize existing historical patient data instead of requiring a comparative group in consideration of the nature of DIPG, a severe rare pediatric glioma and the need for all patients to receive anti-cancer treatment. The FDA also gave detailed advice on pharmacokinetic assessment and standard of care radiation therapy during the clinical study. The detailed response from the FDA indicates their support and expectations for Oblato which is developing a new drug for the treatment of DIPG.

In particular, the FDA has been supportive of biopharmaceutical companies developing treatments for rare pediatric diseases. Furthermore, new drugs for rare pediatric diseases have been approved by the FDA if the efficacy was fully demonstrated even with a small clinical trial. In 2019, Vyondys 53 (golodirsen), a treatment for DMD (Duchenne muscular dystrophy) developed by Sarepta Therapeutics, was approved after just a single phase 1/2 clinical trial. Another example is Brineura (ceriponase alpha) that was developed by Biomarin and approved in 2017 for the treatment of CLN2/Batten disease after a single phase 1/2 clinical study.

"DIPG’s nonclinical studies using OKN-007 have been recognized as supportive to the proposed phase 1/2 trial plan for DIPG and were also fully accepted by the FDA during the meeting. If our treatment shows efficacy in the clinical study for DIPG, we will be able to apply for an NDA after the trial is completed," stated an official from the Company.

The FDA has already granted Oblato the Rare Pediatric Disease Designation for DIPG, and the FDA has officially indicated that Oblato would be eligible for a Rare Pediatric Disease Priority Review Voucher.

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.

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 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that efficacy results of neratinib in HER2-positive early stage breast cancer (eBC) from the Phase III ExteNET trial were presented at the 2020 Virtual San Antonio Breast Cancer Symposium (SABCS) that is currently taking place (Press release, Puma Biotechnology, DEC 9, 2020, View Source [SID1234572545]). The presentation entitled, "Continued efficacy of neratinib in patients with HER2-positive early-stage breast cancer: Final overall survival analysis from the randomized phase 3 ExteNET trial," is being presented at a Spotlight Poster Discussion Session by Frankie Ann Holmes, M.D., FACP, Texas Oncology Houston – US Oncology Research, an investigator of the trial. A copy of this poster presentation is available on the Puma website.

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ExteNET was a multicenter, randomized, double-blind, Phase III trial of 2,840 HER2-positive eBC patients who received neratinib after neoadjuvant and/or adjuvant therapy with chemotherapy and trastuzumab. Patients were stratified by hormone receptor status and randomly assigned to one year of treatment with either oral neratinib 240 mg/day or placebo. The primary endpoint of the trial was invasive disease-free survival (iDFS). Secondary endpoints include overall survival and cumulative incidence of CNS metastases. A descriptive analysis that evaluated CNS disease free survival, which was defined as time from randomization to any CNS recurrence or death from any cause, was performed.

Within the European Union, neratinib is approved in patients with hormone receptor positive (HR+) breast cancer who initiated treatment within one year of completing an adjuvant trastuzumab containing regimen.

The endpoints were analyzed for three groups of clinical interest: (i) the intent to treat (ITT) population; (ii) patients with HR+ breast cancer who initiated treatment within one year of completing an adjuvant trastuzumab containing regimen; and (iii) patients with HR+ breast cancer who initiated treatment within one year of completing an adjuvant trastuzumab containing regimen and who did not achieve a pathological complete response (no pCR) after neoadjuvant treatment and therefore were at a high risk of disease recurrence. Results from the Phase III ExteNET trial were published in the October 5, 2020 issue of Clinical Breast Cancer. The manuscript is accessible online at View Source(20)30258-5/fulltext.

In the ITT population, 127 of 1420 patients (8.9%) in the neratinib group and 137 of 1420 patients (9.6%) in the placebo group died, as of the analysis cut-off date (July 2019). The estimated 8-year overall survival (OS) rates were 90.1% in the neratinib group and 90.2% in the placebo group (stratified HR 0.95; 95% confidence interval [CI] 0.75-1.21; p=0.69). The cumulative incidence of CNS metastases at 5 years was 1.3% (95% CI 0.8-2.1) in the neratinib arm and 1.8% (95% CI 1.2-2.7%) in the placebo arm, while the estimated CNS disease free survival at 5 years was 97.5% in the neratinib group and 96.4% in the placebo group (stratified HR 0.73; 95% CI 0.45-1.17).

In the HR+ /< 1 yr patient population, 53 of 670 patients (7.9%) in the neratinib group and 68 of 664 patients (10.2%) in the placebo group died. The estimated 8-year OS rates were 91.5% in the neratinib group and 89.4% in the placebo group, corresponding to a 2.1% absolute benefit (HR 0.79; 95% CI 0.55‒1.13). The cumulative incidence of CNS metastases at 5 years was 0.7% (95% CI 0.2-1.7) in the neratinib arm and 2.1% (95% CI 1.1-3.5) in the placebo arm, while the estimated CNS disease free survival at 5 years was 98.4% in the neratinib group and 95.7% in the placebo group (stratified HR 0.41; 95% CI 0.18-0.85).

In the HR+/ <1 yr, no pCR subgroup of patients (n=295), 8-year OS rates were 91.3% in the neratinib group and 82.2% in the placebo group, corresponding to a 9.1% absolute benefit (HR 0.47; 95% CI 0.23–0.92). In the HR+/ <1 yr, with a pCR (n=38), 8-year OS rates were 93.3% in the neratinib group and 73.7% in the placebo group, corresponding to a 19.6% absolute benefit (HR 0.40; 95% CI 0.06–1.88). The cumulative incidence of CNS metastases at 5 years was 0.8% (95% CI 0.1-4.0) in the neratinib arm and 3.6% (95% CI 1.3-7.8%) in the placebo arm, while the estimated CNS disease free survival at 5 years was 98.4% in the neratinib group and 92.0% in the placebo group (stratified HR 0.24; 95% CI 0.04-0.92).

Dr. Frankie Ann Holmes said, "These descriptive analyses in HR+ patients who received neratinib within one year of completing trastuzumab and did not achieve a pCR post neoadjuvant therapy suggest that neratinib may be associated with improved OS in this high-risk group (HR 0.47, absolute benefit 9.1%). Importantly, neratinib is the first HER2-directed agent to show a trend towards improved CNS outcomes in early stage HER2-positive breast cancer, with consistently fewer CNS events observed in the neratinib arm compared with placebo in all groups evaluated."

Alan H. Auerbach, Chief Executive Officer and President of Puma, added, "Descriptive analyses suggest that neratinib may be associated with longer overall survival in subgroups of clinical interest and in the high-risk patient subgroup with residual disease after neoadjuvant therapy who are at a high risk of disease recurrence. Although there have been many new treatment options for patients with early stage HER2-positive breast cancer, the risk of disease recurrence and more specifically CNS recurrence remains significant and more must be done. These newly published data demonstrate that neratinib provides a clinically meaningful reduction in the risk of recurrence and CNS recurrence and provides a very important option for these high risk patients."

About HER2-Positive Breast Cancer

Up to 20% of patients with breast cancer tumors over-express the HER2 protein (HER2-positive disease) and in the ExteNET study, 57% of patients were found to have tumors that were hormone-receptor positive. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer recurring, up to 25% of patients treated with trastuzumab experience recurrence within 10 years, the majority of which are metastatic recurrences.

On December 9, 2020 Arcus Biosciences, Inc. (NYSE:RCUS), an oncology-focused biopharmaceutical company working to create best-in-class cancer therapies, and WuXi Biologics (2269.HK), a global company with leading open-access biologics technology platforms, reported an expansion of their existing strategic relationship under which the parties will discover anti-CD39 antibodies using WuXi Bio’s proprietary technology (Press release, Arcus Biosciences, DEC 9, 2020, View Source [SID1234572544]). This CD39 collaboration represents the fourth antibody development program on which the two companies have joined forces. Arcus was granted exclusive worldwide rights to anti-CD39 antibodies discovered under the collaboration and will be responsible for all further development and commercialization activities of such anti-CD39 antibodies.

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The ATP-adenosine axis is believed to play a critical role in maintaining an immunosuppressed tumor microenvironment. Inhibition of one or more of the key nodes (CD39, CD73, or adenosine A2a and A2b receptors) along this axis aims to reduce the formation or activity of the highly immunosuppressive adenosine. A potential additional benefit of CD39 inhibition, aside from blocking an important source of adenosine, is the increase in intra-tumoral ATP, an important molecule for the recruitment and activation of dendritic cells. Preclinical experiments indicate that the combination of CD39 inhibition with either CD73 or adenosine receptor inhibition provides robust inhibition of this axis and increased anti-tumor immunity.

"WuXi Biologics is a global leader in the development and manufacture of therapeutic antibodies. Our relationship with WuXi Biologics started in 2017 with a clinic-ready anti-PD1 antibody, zimberelimab, which possesses molecular properties similar to those of marketed anti-PD1 therapies and has shown impressive clinical anti-tumor activity," said Juan Jaen, Ph.D., president and head of research at Arcus Biosciences. "Furthermore, WuXi Biologics has been an excellent manufacturing partner for our anti-TIGIT antibodies, domvanalimab (AB154) and AB308. We are excited to now extend our existing relationship with WuXi Biologics by combining core competencies to discover anti-CD39 antibodies that have the potential to synergize with adenosine-targeted molecules in our existing portfolio of clinical agents. This will allow us to continue to maintain our position as one of the industry’s leading companies in the targeting of the ATP-adenosine axis for the treatment of cancer."

"We’re thrilled to expand our strategic partnership with Arcus Biosciences to further enable this innovative company to bring new biologics solutions using WuXi Biologics’ proprietary integrated platforms. This partnership is a strong testament to our industry-leading capabilities and expertise," said Dr. Chris Chen, CEO of WuXi Biologics. "We’re committed to offering global open-access technology platforms with premier quality standards to support our global partners as they build their innovative ideas into transformative new treatments for patients worldwide."

Financial terms of the agreement were not disclosed, and the development of any anti-CD39 antibodies from the collaboration is not expected to materially impact Arcus’s financial position over the current cash runway.