On September 18, 2019 NANOBIOTIX (Euronext : NANO – ISIN : FR0011341205 – the ‘‘Company’’), reported encouraging findings from the Company’s phase I study evaluating first-in-class radioenhancer NBTXR3 activated by stereotactic body radiation (SBRT) (Press release, Nanobiotix, SEP 18, 2019, View Source [SID1234539628]). These data were presented at the ASTRO 2019 Annual Meeting by investigator Dr. Enrique Chajon.

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Hafnium oxide nanoparticles activated by SBRT for the treatment of hepatocellular carcinoma and liver metastasis: a phase I/II trial

Enrique Chajon1, Marc Pracht1, Yann Rolland1, Thierry de Baere2, France Nguyen2, Jérôme Durand-Labrunie2, Jean-Pierre Bronowicki3, Véronique Vendrely4, Antonio Sa Cunha5, Anne-Sophie Baumann6, Valérie Croisé-Laurent3, Emanuel Rio7, Samuel Le Sourd1, Pierre Gustin2, Patricia Said8, Christophe Perret7, Didier Peiffert5, Eric Deutsch2

1Radiation Oncology, Centre Eugene – Marquis, Rennes, FR, 2Radiation oncology, Institut Gustave Roussy, Villejuif, FR, 3Hepatology and Gastroenterology, Hôpital de Brabois, Vandoeuvre Les Nancy, FR, 4Radiotherapy, Groupe Hospitalier Sud – Hôpital Haut-Lévêque, Pessac, FR, 5Centre Hépato-Biliaire Paul Brousse, Villejuif, FR, 6Radiotherapy, Institut de Cancérologie de Lorraine, Nancy, FR, 7Radiotherapy, Institut de cancérologie de l’Ouest, Nantes, FR, 8Nanobiotix, SA, Paris, Fr

The population for this study included (i) hepatocellular carcinoma (HCC) patients with a minimum life expectancy of three (3) months that were unsuitable for local surgery or local treatment and (ii) liver metastasis (mets) patients with a minimum life expectancy of six (6) months whose tumors are unresectable. The total number of HCC patients was 11, the total numbers of mets patients was 6.

The HCC patient population has high unmet medical needs as they have underlying liver dysfunction and concomitant malignancies that limit treatment options. Stereotactic body radiation therapy (SBRT) is a well-tolerated alternative therapy, however the dose is limited by hepatic function which is widely impaired in this group. Therefore, these patients need a way to increase the dose delivered to the tumor while sparing surrounding healthy tissues without additional strain on the liver. Moreover, given the short life expectancy of these patients, the need is particularly urgent.

Given that NBTXR3 is inert unless exposed to radiotherapy, is not metabolized by the liver, and can be administered regardless of liver and renal function, the first-in-class treatment offers an opportunity to increase

the efficacy of SBRT while maintaining tolerable dose levels for the patient. In this study, the safety profile of NBTXR3 activated by SBRT was similar to SBRT alone with no dose-limiting toxicity (DLT). Additionally, NBTXR3 also proved to remain within the injected tumor with no leakage to surrounding tissue.

To date, 62.5% of evaluable HCC patients have shown a complete response while the rest have shown partial response. These results show that NBTXR3 activated by SBRT may present as a valuable option in patients with HCC not amenable to curative local treatment or with unresectable liver metastases.

In liver mets, 50% of patients have shown partial response and 50% show stable disease.

Given that the safety profile was very good and there were no DLTs, the protocol was amended to include a fifth dose escalation level of 42%. Three (3) patients have already been recruited and recruitment is expected to be complete in Q4. Results for all patients are expected in Q1 2020.

These results are significant for the Company as they increase the scope of patients NBTXR3 may treat beyond Soft Tissue Sarcoma and Head and Neck cancer. The Company remains focused on its head and neck plan but will now accelerate integration of HCC into its go-to-market strategy.

***

About NBTXR3

NBTXR3 is a first-in-class product designed to destroy tumors through physical cell death when activated by radiotherapy. NBTXR3 has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide standards of radiation care. The physical mode of action of NBTXR3 makes it applicable across solid tumors such as lung, prostate, liver, glioblastoma, and breast cancers.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity oropharynx in elderly and frail patients unable to receive chemotherapy or cetuximab with very limited therapeutic options. Promising results have been observed in the phase I/II trial regarding the local control of the tumors. In the United States, based on the discussions with the Food and Drug Administration that occurred in the first half of 2019, the Company plans to begin the clinical trial authorization process in the second half of 2019 and commence a phase II/III clinical trial in locally advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. In the United States, the Company received approval from the Food and Drug Administration to launch a clinical trial of NBTXR3 activated by radiotherapy in combination with antiPD-1 antibodies in lung, and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung cancer).

The other ongoing NBTXR3 trials are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma. Further, the company has a large-scale, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center (9 new phase I/II clinical trials in the United States) to evaluate NBTXR3 across head and neck, pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers.

On September 18, 2019 Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement (C5) and/or leukotriene (LTB4) systems are implicated, reported that Clive Richardson, Chief Executive Officer, will participate and host investor meetings at the following conference (Press release, Akari Therapeutics, SEP 18, 2019, View Source [SID1234539608]):

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Ladenburg Thalmann 2019 Healthcare Conference

Date: Tuesday, September 24, 2019

Place: Sofitel New York Hotel, New York City

Presentation Time: 10:30 a.m. ET

A live audio webcast of the conference presentation can be accessed by visiting ‘Events’ in the Investor Relations section on the Company’s website at www.akaritx.com. An archived replay of the webcast will be available for 60 days on the Company’s website after the conference.

On September 18, 2019 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI or the Company), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that it has commenced an underwritten public offering of its common shares (Press release, VBI Vaccines, SEP 18, 2019, View Source [SID1234539629]). VBI also intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the number of common shares offered in the public offering. 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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Raymond James & Associates, Inc. and Oppenheimer & Co. Inc. are acting as joint book-running managers, and National Securities Corporation, a wholly owned subsidiary of National Holdings, Inc. (NASDAQ:NHLD), is acting as lead manager for the underwritten public offering.

VBI intends to use the net proceeds from the offering to progress its pipeline programs including the completion of the global CONSTANT Phase 3 lot-to-lot consistency study, regulatory submissions, and pre-commercialization activities for Sci-B-Vac, a trivalent hepatitis B vaccine, and for the continued clinical development of VBI-1901, a vaccine immunotherapeutic candidate for recurrent glioblastoma (GBM); VBI-2601, an immunotherapeutic candidate for chronic hepatitis B infection; and VBI-1501, a cytomegalovirus (CMV) vaccine candidate. The net proceeds will also be used for general corporate purposes, including working capital and capital expenditures.

A shelf registration statement relating to the common shares was previously filed with the Securities and Exchange Commission (SEC) and declared effective on July 30, 2018. A preliminary prospectus supplement and accompanying prospectus relating to the underwritten public offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement (when available) and accompanying prospectus may be obtained from Raymond James & Associates, Inc., Attention: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida 33716, by telephone at (800) 248-8863, by e-mail at [email protected], or from Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY 10004 or by e-mail at [email protected].

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 registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement

On September 18, 2019 BeyondSpring Inc. ("BeyondSpring" or the "Company") (NASDAQ:BYSI), a global biopharmaceutical company focused on developing transforming immuno-oncology cancer therapies, reported its financial results for the three months ended June 30, 2019, and provided an update on its operations (Press release, BeyondSpring Pharmaceuticals, SEP 18, 2019, View Source [SID1234539609]).

"During the second quarter, we continued to demonstrate Plinabulin’s favorable drug profile for the treatment of NSCLC and CIN," said Lan Huang, Ph.D., Chairman, Chief Executive Officer and Co-Founder of BeyondSpring. "In addition to results from our CIN studies, which provided strong rationale for the use of Plinabulin alone and in combination with G-CSF for the prevention of intermediate-risk and high-risk CIN, the Data Safety Monitoring Board advised Study 103 to continue after the first interim analysis."

"In the near term, we expect to continue to generate additional data for Plinabulin in both NSCLC and CIN indications to support NDA filings in China followed by the U.S. Our recent financing activities position us well to achieve these key milestones," added Dr. Huang.

Select Second-Quarter 2019 and Recent Highlights

NSCLC

Study 103

In September 2019, BeyondSpring announced that a poster describing the Company’s novel design for Study 103, titled "DUBLIN-3, a Phase (Ph) III Trial Comparing the Plinabulin (P) / Docetaxel (D) Combination with D Alone in Stage IIIB / IV NSCLC," was presented at the International Association for the Study of Lung Cancer World Conference on Lung Cancer in Barcelona, Spain.

CIN

Study 105 (Intermediate-Risk Chemotherapy)

In August 2019, BeyondSpring announced that an abstract, titled "Quality of Life (QoL) in Advanced NSCLC Patients Treated with Docetaxel and with Either Plinabulin or Pegfilgrastim for the Prevention of Neutropenia," was accepted for presentation at the September 8, 2019 IASLC World Conference on Lung Cancer. The abstract demonstrates that, compared to Neulasta, Plinabulin at 20mg/m2 significantly improves QoL over four treatment cycles. Plinabulin also significantly improved fatigue, pain and insomnia.

At the International Society for Pharmacoeconomics and Outcomes Research 2019 Conference in May 2019, Dr. Douglas Blayney, global Principal Investigator for BeyondSpring’s CIN development program and Professor of Medicine at Stanford University Medical Center, presented data derived from the Phase 2 portion of Study 105 demonstrating that Plinabulin at 20mg/m2 has a similar efficacy profile in reducing docetaxel-induced neutropenia as Neulasta 6mg, while avoiding the patient-reported bone pain typically observed with Neulasta.

Study 106 (High-Risk Chemotherapy)

In June 2019, BeyondSpring announced that two abstracts were accepted for publication in the Proceedings of the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. The data, derived from the Phase 2 portion of Study 106, provided a strong rationale for the Plinabulin-G-CSF combination for the prevention of CIN for improved CIN control. Additionally, the Plinabulin-G-CSF combination nearly eliminated patients’ treatment-related bone pain.

1
BeyondSpring is currently initiating the Phase 3 portion of Study 106.

Mechanism of Action

In April 2019, BeyondSpring presented novel data relevant to predictive biomarkers for patient selection for Plinabulin at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. At the same conference, BeyondSpring presented preclinical data demonstrating Plinabulin’s ability to reduce tumor associated M2 macrophages, which are thought to support tumor cell survival and metastasis, as well as shift the phenotypic balance to one favoring M1 macrophages, which are thought to have anti-cancer properties.

Equity Financings

On July 19, 2019, BeyondSpring closed a public offering, led by Decheng Capital, of 2,058,825 ordinary shares at a public offering price of $17.00 per share. Gross proceeds from the public offering were $35.0 million, before deducting underwriting discounts and commissions and other offering expenses. The Company intends to use the net proceeds of this offering to support continued clinical and pre-clinical development and for general corporate purposes.

In June and July 2019, BeyondSpring’s partially owned Chinese subsidiary, Dalian Wanchunbulin Pharmaceuticals Ltd. ("Wanchunbulin") entered into definitive agreements for the sale of its equity interests ("Equity Purchase Agreements") to certain investors led by Efung Capital. Under the Equity Purchase Agreements, Wanchunbulin expects to sell 4.76% of the equity of Wanchunbulin for aggregate cash consideration of RMB 100 million, or approximately $14.5 million, before deducting offering expenses, to finance clinical and pre-clinical development and for general corporate purposes. To date, the Company has received RMB 70 million, or approximately $10.1 million, from this equity financing.

In May 2019, BeyondSpring entered into an Open Market Sale AgreementSM with Jefferies LLC to sell ordinary shares of the Company, with aggregate gross proceeds of up to $30.0 million, from time to time, through an at-the-market ("ATM") facility. In July 2019, this facility was suspended in anticipation of the public offering completed on July 19, 2019. As at the ATM’s suspension date, the Company had received aggregate gross proceeds of $13.0 million on 620,753 ordinary shares already sold through this facility.

Financial Results for the Three Months Ended June 30, 2019

Research and development ("R&D") expenses were $5.2 million for the quarter ended June 30, 2019, compared to $11.0 million for the quarter ended June 30, 2018. The $5.8 million decrease in R&D expenses was largely attributable to a $3.9 million decrease in expense incurred by clinical research organizations and other service fee related to clinical trials, a $1.1 million decrease in manufacturing expenses and a $0.8 million decrease in non-cash share-based compensation.

General and administrative ("G&A") expenses were $2.1 million for the quarter ended June 30, 2019, compared to $1.4 million for the quarter ended June 30, 2018. The $0.7 million increase in G&A expenses was mainly due to non-cash share-based compensation.

Net loss attributable to the Company was $7.4 million for the quarter ended June 30, 2019, compared to $12.2 million for the quarter ended June 30, 2018.

As of June 30, 2019, the Company had a cash balance of $0.7 million. With the net proceeds received from the recent equity financing activities described above, the Company believes it has sufficient cash to support its clinical trials and submit NDAs in China for Plinabulin for the treatment of CIN and NSCLC, and to advance its immuno-oncology pipeline and its ubiquitination protein degradation research platform.

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Anticipated Milestones

The following outlines the Company’s key anticipated upcoming milestones and projected timelines.

Second interim analysis for Study 103 Phase 3 for NSCLC – Q4 2019 / Q1 2020

Final data readout for Study 105 Phase 3 for CIN – Q1 2020

Top line data readout for Study 106 Phase 3 for CIN – Q1 2020

Submit NDAs to China’s National Medical Products Administration for Plinabulin for NSCLC and CIN – Q1 2020

Submit NDAs to the FDA for Plinabulin for NSCLC and CIN – 2020

Conference Call and Webcast Information

BeyondSpring’s management will host a conference call and webcast today at 8:00 a.m. Eastern Time to discuss the financial results and provide a corporate update. The dial-in numbers for the conference call are 1-877-451-6152 (U.S.) or 1-201-389-0879 (international). Please reference conference ID 13694411. A live webcast will be available on BeyondSpring’s website at www.beyondspringpharma.com under "Events & Presentations" in the Investors section. An archived replay of the webcast will be available for 30 days.

On September 18, 2019 Johnson & Johnson (NYSE: JNJ) reported that it will host a conference call for investors at 8:30 a.m. (Eastern Time) on Tuesday, October 15th to review third-quarter results (Press release, Johnson & Johnson, SEP 18, 2019, View Source;johnson-to-host-investor-conference-call-on-third-quarter-results-300921132.html [SID1234539630]). Joseph J. Wolk, Executive Vice President, Chief Financial Officer and Christopher DelOrefice, Vice President, Investor Relations will host the call. The question and answer portion of the call will also include the following executives: Ashley McEvoy, Executive Vice President, Worldwide Chairman, Medical Devices; Thibaut Mongon, Executive Vice President, Worldwide Chairman, Consumer; and Jennifer Taubert, Executive Vice President, Worldwide Chairman, Pharmaceuticals.

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Investors and other interested parties can access the webcast/conference call in the following ways:

The webcast and presentation material are accessible at Johnson & Johnson’s website www.investor.jnj.com. A replay of the webcast will be available approximately three hours after the conference call concludes.
By telephone: for both "listen-only" participants and those financial analysts who wish to take part in the question-and-answer portion of the call, the telephone dial-in number in the U.S. is 877-869-3847. For participants outside the U.S., the dial-in number is 201-689-8261.
A replay of the conference call will be available until approximately 12:00 a.m. on October 23, 2019. The replay dial-in number for U.S. participants is 877-660-6853. For participants outside the U.S., the replay dial-in number is 201-612-7415. The replay conference ID number for all callers is 13694535.
The press release will be available at approximately 6:45 a.m. (Eastern Time) the morning of the conference call.