On May 12, 2020 Sutro Biopharma, Inc. (Nasdaq: STRO), a clinical stage drug discovery, development and manufacturing company focused on deploying its proprietary integrated cell-free protein synthesis platform, XpressCF, to create a broad variety of optimally designed, next-generation protein therapeutics initially for cancer and autoimmune disorders, reported the pricing of an underwritten public offering of 11,000,000 shares of its common stock at a price to the public of $7.75 per share (Press release, Sutro Biopharma, MAY 12, 2020, View Source [SID1234557630]). The gross proceeds from this offering are expected to be approximately $85.3 million, before deducting underwriting discounts and commissions and other offering expenses payable by Sutro. Sutro has also granted the underwriters a 30-day option to purchase up to an additional 1,650,000 shares of common stock in connection with the public offering. All of the shares of common stock are being offered by Sutro. The offering is expected to close on or about May 14, 2020, subject to the satisfaction of customary closing conditions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cowen, Piper Sandler and Wells Fargo Securities are acting as joint book-running managers in the offering.

Sutro intends to use the net proceeds from the proposed offering, together with its existing cash, cash equivalents and marketable securities, to fund the continued clinical development of STRO-001 and STRO-002 and the remainder to fund the further development of its technology platform, including manufacturing, to broaden its pipeline of product candidates, and for working capital and general corporate purposes.

The shares are being offered by Sutro pursuant to a registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to this offering have been filed with the SEC. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering, and when available, the final prospectus supplement, may be obtained from: Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at [email protected]; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at [email protected]; or Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 500 West 33rd Street, New York, New York 10001, by telephone at (800) 326-5897, or by email at [email protected]. Electronic copies of the preliminary prospectus supplement and accompanying prospectus will also be available on the website of the SEC at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Sutro, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On May 12, 2020 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported financial results for the first quarter ended Mar. 31 and provided operational updates, including measures taken by the company in response to the COVID-19 pandemic (Press release, Turning Point Therapeutics, MAY 12, 2020, View Source [SID1234564374]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In reflecting on the past quarter of events, I want to acknowledge and thank the dedicated health care providers worldwide who have been treating patients during this unprecedented time," said Athena Countouriotis, M.D., president and chief executive officer. "As we have adapted to manage our business, we are appreciative of their efforts for the health and well-being of patients.

"Despite a very challenging environment in the first quarter, I am pleased with our recent Fast Track designation for repotrectinib based on Phase 1 TRIDENT-1 ROS1+ TKI Naïve NSCLC data which included an update to the duration of response, duration of treatment, and new progression free survival data; and the progress our team made advancing our programs, including our global registrational Phase 2 clinical study of repotrectinib. Site activations continued, with enrollment progressing across our four ongoing clinical trials.

"As it relates to COVID-19, we took action during the first quarter to implement multiple steps in response to the pandemic, including remote site activation and data monitoring; enabling patients to have routine tests conducted closer to home and allowing sites to evaluate certain patients remotely, in compliance with their local procedures. We are also evaluating additional sites for participation in our TRIDENT-1 study.

"Despite these interventions, we have more recently started to experience delays in trial site initiations and patient enrollment within the Phase 2 TRIDENT-1 study. We are also closely watching the progress in our other studies and believe the extent of the impact on our pipeline will depend on the continued duration and severity of the pandemic.

"We continue to anticipate providing data updates from our registrational Phase 2 TRIDENT-1 and TPX-0022 trials in the second half of 2020. For TRIDENT-1, we are currently monitoring the overall study conduct and data collection to be in a position to potentially provide this update in the third quarter. We anticipate the update will include preliminary efficacy and safety data from approximately 30 to 40 patients across multiple Phase 2 cohorts, including registrational and exploratory cohorts. For TPX-0022 we anticipate our update will be primarily a safety update as well as any early efficacy signals as we continue through our dose escalation cohorts."

First quarter and recent highlights include:

The company announced today that repotrectinib has been granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of ROS1-positive advanced non-small cell lung cancer (NSCLC) patients who have not been previously treated with a ROS1 tyrosine kinase inhibitor (TKI). In January, repotrectinib was granted Fast Track designation for the treatment of ROS1-positive advanced NSCLC patients who had been previously treated with one prior line of platinum-based chemotherapy and one prior line of a ROS1 TKI, a setting where there are currently no approved targeted therapies.

Fast Track Designation granted in ROS1+ TKI-naive patients. In the Phase 1 portion of TRIDENT-1, utilizing the 22 July 2019 data cut-off date with a median follow-up of 20.1 months (range: 5.3 to 24.9+), repotrectinib demonstrated a confirmed overall response rate (ORR) by blinded independent central review (BICR) of 91 percent (N=11, 95% CI: 59–100) in patients with ROS1+ advanced NSCLC who are ROS1 TKI-naïve. Repotrectinib demonstrated a median duration of response (DOR) of 23.1 months (95% CI: 5.6–NR) (based on Kaplan-Meier estimation). The probability of patients with a DOR ≥ 9 months, ≥ 12 months and ≥ 18 months was 78 percent, 65 percent, and 65 percent, respectively. Also, repotrectinib showed a median progression-free survival (PFS) of 24.6 months (95% CI: 7.2–NR). With an additional 8.5 months of follow-up as of 6 April 2020, 4 of the 5 responding patients remained in a PR (partial response) per physician assessment data since the 22 July 2019 data cutoff and the duration of treatment ranged from 9.2 to 34.2+ months with 7 of the total 11 (64%) patients remaining on repotrectinib.
All 7 (64%) remain on treatment for more than 17 months,
6 (55%) on treatment for more than 24 months, and
3 (27%) on treatment for more than 30 months at the time of the analysis

Repotrectinib has demonstrated CNS activity among patients with ROS1+ advanced NSCLC who are ROS1 TKI-naïve, with an intracranial objective response rate (IC-ORR) of 100% (3 of 3 patients, 95% CI: 29–100) with durations of response, as of the 22 July 2019 data cut-off, of 14.8+, 17.6+ and 23.1 months. All three of these patients remain on treatment, as of 6 April 2020, for 26.0+, 28.5+ and 34.2+ months.

Ongoing site activations and enrollment in the Phase 2 registrational portion of the TRIDENT-1 study of repotrectinib, with approximately 50 percent of planned sites now active in 11 countries. The study is now planned at up to 120 global sites with enrollment of approximately 320 ROS1-positive advanced non-small cell lung cancer (NSCLC) and NTRK-positive advanced solid tumor patients.

Ongoing progress in the Phase 1/2 open-label study to assess repotrectinib in pediatric patients with ALK-, NTRK- or ROS1-positive advanced solid tumors; the Phase 1 study of TPX-0022, Turning Point’s MET/CSF1R/SRC inhibitor; and Phase 1/2 study of TPX-0046, Turning Point’s RET/SRC inhibitor. Both the TPX-0022 and TPX-0046 trials continue dose escalation based on real time pharmacokinetic data evaluation and enrollment includes both TKI-naïve and -pretreated patients across both studies.

Acceptance of multiple abstracts for poster presentations at the ASCO (Free ASCO Whitepaper) and AACR (Free AACR Whitepaper) virtual annual meetings. The ASCO (Free ASCO Whitepaper) presentation is expected to highlight preclinical data for TPX-0046 and the AACR (Free AACR Whitepaper) presentations are planned to highlight preclinical repotrectinib combination data and TPX-0131 preclinical data.

Recent publication in the AACR (Free AACR Whitepaper) journal Clinical Cancer Research of repotrectinib preclinical data and patient case studies from the Phase 1 portion of TRIDENT-1. The publication highlighted potent antitumor activity of repotrectinib in treatment-naïve and solvent-front mutation ROS1-rearranged NSCLC. In addition, repotrectinib demonstrated significant reduction of brain lesions in an intracranial tumor model.
First Quarter Financial Update
Operating expenses for the first quarter totaled $62.6 million, which included $38.4 million in non-cash stock-based compensation expense. The non-cash stock-based compensation expenses included a one-time charge of $31.4 million associated with previously disclosed modifications to the vesting of existing stock options, pursuant to the transition agreement with the company’s scientific founder. Excluding this one-time charge, non-GAAP operating expenses in the first quarter totaled $31.2 million compared to GAAP operating expenses of $14.1 million in the first quarter of 2019 and $23.1 million in the fourth quarter of 2019. Primary drivers of the year-over-year increase were investments made to develop repotrectinib, TPX-0022 and TPX-0046, as well as personnel expenses.

Cash, cash equivalents and marketable securities at Mar. 31 totaled $380.8 million, a decrease of $28.4 million from Dec. 31, 2019. The company projects its cash position funds current operations into 2022.

Upcoming Milestones
Key milestones anticipated through 2020 include:

Presenting preclinical data for TPX-0046, the company’s novel RET/SRC inhibitor, as a poster presentation during the ASCO (Free ASCO Whitepaper) virtual annual meeting on May 29.

Presenting preclinical repotrectinib combination data and preclinical data for TPX-0131, the company’s novel ALK inhibitor, during the AACR (Free AACR Whitepaper) virtual annual meeting in late June.

Early interim data from initial patients in the TRIDENT-1 Phase 2 study during the second half of the year. The company anticipates this update will include preliminary efficacy and safety data from approximately 30 to 40 patients across multiple Phase 2 cohorts, including registrational and exploratory cohorts.

Early interim data from initial patients treated with TPX-0022 during the second half of the year.

Submitting the IND for TPX-0131 by early 2021.
Webcast and Conference Call
Turning Point will webcast its Quarterly Update Conference Call today, May 12 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Countouriotis will host the call, which will be accessible through the "Investors" section of tptherapeutics.com or by dialing (877) 388-2118 (in the United States) or (470) 495-9489 (outside the U.S.) using conference ID 6135289. A replay will be available through the "Investors" section of www.tptherapeutics.com.

On May 12, 2020 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported its financial results for the first quarter 2020 and business highlights, including an update on its progress with fadraciclib, Cyclacel’s novel CDK inhibitor (Press release, Cyclacel, MAY 12, 2020, View Source [SID1234557599]). The Company’s net loss applicable to common shareholders for the three months ended March 31, 2020 was $1.3 million. As of March 31, 2020, cash and cash equivalents totaled $8.9 million. Following net proceeds of $18.4 million from an equity financing in April 2020, pro forma cash and cash equivalents total $27.3 million. Based on current spending, the Company estimates it has sufficient resources to fund planned operations, including research and development, to the end of 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The global pandemic is creating uncertainty in every business sector and it is clear that we need novel, science-based solutions to emerge from the crisis," said Spiro Rombotis, President and Chief Executive Officer. "While our priorities are ensuring patient safety and addressing our social responsibility, we remain committed to our business strategy of building an innovative pipeline addressing the rising problem of cancer resistance. Fadraciclib is establishing a leadership position among MCL1 suppressing compounds in clinical development. We are encouraged by observations of deep response and prolonged stable disease with tumor shrinkage in both intravenous schedules tested this far. Importantly, initial clinical data with oral fadraciclib show concordance with intravenous pharmacokinetics. After strengthening our balance sheet, we will now turn our attention to executing a precision medicine strategy to evaluate fadraciclib in patients with solid tumors and achieve our other clinical milestones through late 2022."

Key Corporate Highlights

·In light of the pandemic caused by the novel coronavirus and to ensure the health and wellbeing of our employees, patients and the communities we serve, we have redesigned our work flow and business processes in line with current standards and government recommendations. In addition, we are working hard to provide uninterrupted clinical supplies and maintain the integrity of our clinical research. At present, we have not experienced recruitment delays, and our clinical investigators continue to screen and enroll patients. As the future course of the pandemic is uncertain, we will continue to closely monitor developments.

·CYC065-01 Phase 1 part 2 single agent i.v. – We have previously reported that a heavily pretreated patient with MCL1 amplified endometrial cancer achieved a radiographically confirmed partial response (PR) after a month and a half on fadraciclib at 213mg. This patient continues on therapy and reduction in her target tumor lesions is 79% after nine months. An additional patient with cyclin E amplified ovarian cancer achieved stable disease with 29% tumor shrinkage after approximately four months at 213mg. Based on data thus far, we are designing a Phase 1/2 precision medicine study to further evaluate fadraciclib as monotherapy and in combinations in patients with advanced solid tumors.

þ 200 Connell Drive, Suite 1500, Berkeley Heights, NJ 07922, USA Tel +1 908 517 7330 Fax +1 866 271 3466

¨ 1 James Lindsay Place, Dundee, DD1 5JJ, UK Tel +44 1382 206 062 Fax +44 1382 206 067

www.cyclacel.com – [email protected]

·CYC065-01 Phase 1 part 3 single agent p.o. – Initial data from an oral capsule formulation of fadraciclib given once daily to three patients with advanced solid tumors demonstrated a predictable pharmacokinetic profile closely overlapping the intravenous form with encouraging exposure levels.

·CYC065-03 Phase 1 fadraciclib i.v. and venetoclax p.o. in AML/MDS – We have dosed 11 heavily pretreated patients with relapsed/refractory (R/R) AML in five dose levels up to 200 mg/m2 of fadraciclib in combination with the venetoclax. Evidence of anticancer activity has been observed in multiple patients with blast reductions in peripheral blood. Preclinical data in AML suggest that targeting both MCL1 and BCL2 may be more beneficial than inhibiting either protein alone.

·CYC065-02 Phase 1 fadraciclib i.v. and venetoclax p.o. in CLL – We have dosed 5 patients with R/R CLL in four dose levels up to 150 mg/m2 of fadraciclib in combination with venetoclax. Evidence of anticancer activity has been observed in two patients who achieved MRD negativity on the combination. Preclinical data suggest that targeting both BCL2 and MCL1 in CLL may be more beneficial than single agent treatment in this setting as well.

·CYC682-11 Phase 1 part 2 sapacitabine p.o. and venetoclax p.o. – We have enrolled 12 patients in two dose cohorts in our DNA Damage Response (DDR) program evaluating an oral combination of sapacitabine and venetoclax in patients with R/R AML/MDS. Sapacitabine is a nucleoside analogue that is active in AML and MDS R/R to prior therapy such as cytarabine or hypomethylating agents. Preclinical data demonstrated synergy of sapacitabine with BCL2 inhibition, which may offer an effective, oral treatment regimen for patients who have failed front-line therapy.

·CYC140-01 Phase 1 CYC140 i.v. – We have enrolled 5 patients in our first-in-human, dose escalation study evaluating CYC140 in patients with advanced leukemias. CYC140 is a small molecule, selective polo-like-kinase 1 (PLK1) inhibitor that has demonstrated potent and selective target inhibition and high activity in xenograft models of human cancers.

·COVID-19 Collaboration – We entered into an agreement with The University of Edinburgh to evaluate the potential of our CDK inhibitors, fadraciclib and seliciclib, for reducing runaway inflammation and subsequent lung injury in patients with COVID-19 disease.

More information on our clinical trials can be found at www.clinicaltrials.gov.

Key Business Objectives

·Report updated fadraciclib Phase 1 safety and efficacy data with frequent i.v. dosing schedule in patients with advanced solid cancers;
·Report initial safety and PK data from Phase 1 study of fadraciclib oral formulation;
·Treat first patient in fadraciclib Phase 1/2 precision medicine study;
·Report initial data from fadraciclib-venetoclax Phase 1 study in R/R AML/MDS & CLL;
·Report initial data from sapacitabine-venetoclax Phase 1 study in R/R AML/MDS;
·Report initial data from CYC140 Phase 1 first-in-human study in R/R leukemias; and
·Report data from Phase 1b/2 sapacitabine-olaparib IST in BRCA mutant metastatic breast cancer when reported by the investigators.

Financial Highlights

As of March 31, 2020, cash and cash equivalents totaled $8.9 million, compared to $11.9 million as of December 31, 2019. The decrease of $3.0 million was primarily due to net cash used in operating activities of $2.8 million and $0.1 million of net cash used in financing activities. There were no revenues for each of the three months ended March 31, 2020 and 2019.

Research and development expenses were $1.1 million for the three months ended March 31, 2020 as compared to $1.0 million for the same period in 2019. Research and development expenses relating to transcriptional regulation increased by almost $0.3 million for the three months ended March 31, 2020 as we continue to progress the clinical evaluation of fadraciclib.

General and administrative expenses for the three months ended March 31, 2020 were $1.3 million, compared to $1.2 million for the same period of the previous year.

Total other income, net, for the three months ended March 31, 2020 was $0.9 million, compared to $0.1 million for the same period of the previous year. The increase of $0.8 million for the three months ended March 31, 2020 is primarily related to income received under an Asset Purchase Agreement with Thermo Fisher Scientific Inc.

United Kingdom research & development tax credits were $0.3 million for each of the three months ended March 31, 2020 and 2019.

Net loss for the three months ended March 31, 2020 was $1.2 million, compared to $1.8 million for the same period in 2019.

The Company raised net proceeds of approximately $18.4 million from an equity financing in April 2020.

The Company estimates that cash resources of $8.9 million as of March 31, 2020 together with the $18.4 million net proceeds from the April 2020 financing will fund currently planned programs through 2022.

Conference call information:

US/Canada call: (877) 493-9121 / international call: (973) 582-2750

US/Canada archive: (800) 585-8367 / international archive: (404) 537-3406

Code for live and archived conference call is 4198767.

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On May 12, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will present at the Bank of America 2020 Health Care Conference on Thursday, May 14 at 4:20 p.m. ET (Press release, Sangamo Therapeutics, MAY 12, 2020, View Source [SID1234557615]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The audio presentation will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

On May 12, 2020 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company"), a clinical-stage biotechnology company focused on patient-specific immunotherapies for the treatment of cancer and infectious diseases, reported financial results for the quarter ended March 31, 2020 (Press release, BioNTech, MAY 12, 2020, View Source [SID1234557638]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"BioNTech has demonstrated significant progress to date in 2020. We advanced our oncology pipeline, announced the closing of our acquisition of Neon Therapeutics in the U.S., and signed several new value-adding partnerships," said Ugur Sahin, BioNTech’s CEO. "Most notably, we have rapidly initiated a global clinical development program in Europe and the U.S. for multiple COVID-19 vaccine candidates."

First Quarter 2020 and Subsequent Updates

BioNTech continues to monitor the effect of the current COVID-19 pandemic situation on its overall operations. As previously announced, the company has put significant measures in place to protect supply chain, operations, employees and the execution of clinical trials. The Company has not seen any impact on our mRNA manufacturing, nor on our CAR-T manufacturing operations. BioNTech has implemented a plan to manage the evolving disruptions on our clinical programs, and as previously detailed, is prioritizing execution of ongoing clinical trials, whereas certain first-in-human (FIH) clinical trial timelines have been affected. BioNTech intends to initiate Phase 2 trials as planned, manage ongoing Phase 1 trials to support completion and optimize ability to initiate and conduct FIH studies. BioNTech will continue to evaluate potential effects and provide updates as appropriate.

Infectious disease

BioNTech has made significant progress in its efforts to develop a potential vaccine to induce immunity and prevent COVID-19 infection in response to the global health threat posed by the disease. During the first quarter, the company assembled a global consortium of partners including Pfizer (worldwide collaboration outside of China) and Fosun Pharma (China). BioNTech’s goal is to make a vaccine available to the public worldwide as quickly as possible.

COVID-19 Vaccine Program

BNT162 – BioNTech’s vaccine program against COVID-19, BNT162, leverages the Company’s proprietary mRNA platform. Currently there are four vaccine candidates, two of the four vaccine candidates include a nucleoside modified mRNA (modRNA), one includes a uridine containing mRNA (uRNA), and the fourth vaccine candidate utilizes self-amplifying mRNA (saRNA). Each mRNA format is combined with a lipid nanoparticle (LNP) formulation. The larger spike sequence is included in two of the vaccine candidates, and the smaller optimized receptor binding domain (RBD) from the spike protein is included in the other two candidates.

BNT162 (Europe) – BioNTech’s Phase 1/2 clinical trial, the first of a COVID-19 vaccine candidate in Europe, has dosed the first cohort of patients. Twelve study participants were dosed with the first BNT162 vaccine candidate as of April 29th. The dose escalation portion of the Phase 1/2 trial will include approximately 200 healthy subjects between the ages of 18 to 55 and will target a dose range of 1 µg to 100 µg, aiming to determine the optimal dose for further studies as well as to evaluate the safety and immunogenicity of the vaccine. Three vaccine candidates that utilize uRNA or modRNA will be administered as two injections. The fourth vaccine candidate, which contains saRNA, will be evaluated after a single dose of vaccine. Subjects with a higher risk of severe COVID-19 disease will be included in the second part of the study. First clinical data from the trial is expected end of June or in July 2020.

BNT162 (U.S) – The first cohort has been dosed in the U.S. in the Phase 1/2 clinical trial for the BNT162 vaccine program. The Phase 1/2 study is designed to determine the safety, immunogenicity and optimal dose level of the four mRNA vaccine candidates. The dose level escalation portion (Stage 1) of the Phase 1/2 trial in the U.S. will enroll up to 360 healthy subjects into two age cohorts (18-55 and 65-85 years of age) and will be seamlessly followed by administering the selected vaccine candidate to several thousands of subjects. The first subjects immunized in Stage 1 of the study will be healthy adults 18-55 years of age.

BioNTech will provide clinical supply of the BNT162 vaccine from its GMP-certified mRNA manufacturing facilities in Europe. BioNTech and Pfizer will work together to scale-up manufacturing capacity at risk to provide worldwide supply in response to the pandemic. BioNTech believes it has the potential to supply millions of vaccine doses by the end of 2020 subject to technical success of the development program and approval by regulatory authorities, and then rapidly scale up capacity to produce hundreds of millions of doses in 2021.

Oncology

BioNTech has also continued to advance its broad oncology pipeline. There are currently ten oncology products in 11 ongoing trials with multiple data readouts expected in 2020. BioNTech intends to initiate four Phase 2 trials (BNT111, BNT113, BNT122) and two additional FIH trials (BNT211, BNT411) in 2020.

FixVac

BNT111 – Data from a Phase 1 trial in advanced melanoma remains on track for publication in late 1H 2020. BioNTech expects to initiate a Phase 2 trial in advanced melanoma with registrational potential for BNT111 in 2H 2020.

BNT 113 – Initiation of a Phase 2 trial in HPV+ head and neck cancer with registrational potential is on track for 2H 2020.

BNT114 – Data update from a Phase 1 trial in triple negative breast cancer (TNBC) is expected in 2H 2020.

Individualized neoantigen specific immunotherapy (iNeST)

BNT122 – BioNTech expects the data update presentation for the Phase 1 trial in multiple solid tumors to be disclosed in June 2020 as part of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II. Safety, immunogenicity and tumor response data will be included. BioNTech expects to provide an enrollment update1 from the Phase 2 trial (IMCODE-001) in first line melanoma in 2H 2020 with an interim data update anticipated in 2021. BNT122 is partnered with Genentech.

BNT122 – Two Phase 2 clinical trials are expected to be initiated in the adjuvant setting in 2H 2020. The first adjuvant Phase 2 study will evaluate the efficacy and safety of RO7198457 plus atezolizumab compared with atezolizumab alone in patients with Stage 2-3 non-small cell lung cancer (NSCLC) who are circulating tumor DNA (ctDNA) positive following surgical resection and have received standard-of-care adjuvant platinum-doublet chemotherapy.

mRNA intratumoral immunotherapy

BNT131 – Data update from Phase 1/2 trial in solid tumors remains on track for 2H 2020. BNT131 is partnered with Sanofi.

CAR-T cell immunotherapy

BNT211 – Initiation of a Phase 1/2a trial in multiple solid tumors (CLDN6) is now expected in 2H 2020.

Next-generation checkpoint immunomodulators

BNT311 – The expansion cohort has been initiated in the Phase 1/2 trial in multiple solid tumors for BNT311 (PD-L1x4-1BB). BioNTech expects to provide a data update, to include dose-escalation and potentially some limited expansion data from the trial in 2H 2020. BNT311 is partnered with Genmab.

We expect this data update to include an update on the ongoing study, including patient enrollment numbers, with full efficacy and safety data for an interim update expected in the second half of 2021.

Toll-Like Receptor Binding

BNT411 – A Phase 1/2a clinical trial of BNT411 is still expected to be initiated in multiple solid tumors in 2H 2020.

Corporate Development

Recently, BioNTech completed the acquisition of Neon Therapeutics, Inc. in an all-stock transaction. BioNTech is now in the integration phase and expects the new subsidiary, based in Cambridge, Massachusetts, to serve as BioNTech’s U.S. headquarters.

First Quarter 2020 Financial Results

Cash Position: Cash and cash equivalents as of March 31, 2020, were €451.6 million.

Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was €27.7 million for the three months ended March 31, 2020, compared to €26.2 million for the three months ended March 31, 2019. The increase was mainly due to revenues resulting from other sales transactions, i.e. development and manufacturing services sold to third-party customers, retroviral vectors for clinical supply, and sales of peptides.

Research and Development Expenses: Research and development expenses were €65.1 million for the three months ended March 31, 2020, compared to €57.2 million for the three months ended March 31, 2019. The increase was primarily due to an increase in headcount leading to higher wages, benefits and social security expenses as well as an increase in expenses for purchased research services.

General and Administrative Expenses: General and administrative expenses were €15.8 million for the three months ended March 31, 2020, compared to €9.3 million for the three months ended March 31, 2019. This increase was mainly driven by higher legal expenses, an increase in headcount leading to higher wages, benefits and social security expenses as well as higher expenses due to newly concluded insurance premiums.

Net Loss: Net loss was €53.4 million for the three months ended March 31, 2020, compared to a net loss of €40.8 million for the three months ended March 31, 2019.

Shares Outstanding: Shares outstanding as of March 31, 2020 were 226,779,744.

Financial Guidance:

On track with previous guidance of approximately €300 million net cash to be used for operating activities and investments into property, plant and equipment in 2020 base business plan (prior to impact of Neon acquisition and BNT162 program).

Majority of BioNTech development costs for our BNT162 program in 2020 will be funded via Pfizer and Fosun Pharma cost sharing, equity investments and upfront payments.

Also anticipate additional funding to support the manufacturing scale-up for our BNT162 program in 2020.

Interim quarterly financial statements can be found in the 6-K filing as published on the SEC website under www.sec.gov.

Conference Call and Webcast Information

BioNTech SE will host a conference call and webcast today at 08:00 a.m. ET (2:00 p.m. CET) to report its financial results for the quarter ended March 31, 2020 and provide a corporate update.

To participate in the conference call, please dial the following numbers 10-15 minutes prior to the start of the call and provide the Conference ID: 9282359.

Participants may also access the slides and the webcast of the c1nference call via the "Events & Presentations" page of the Investor Relations section of the Company’s website at View Source A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

Financial and Operational Results for the First Quarter Ended March 31, 2020

Key Pipeline Updates

Below is a summary of our clinical product candidates, organized by platform and indications.

Oncology

FixVac. Our FixVac product candidates contain selected combinations of pharmacologically optimized uridine mRNA encoding known cancer-specific shared antigens. They feature our proprietary immunogenic mRNA backbone and proprietary RNA-lipoplex, or RNA-LPX, delivery formulation, designed to enhance stability and translation, target dendritic cells and trigger both innate and adaptive immune responses. FixVac is currently being evaluating in five clinical trials, including:

BNT111 in a Phase 1 trial in advanced melanoma with a data update expected via publication in a medical journal late in the first half of 2020. We expect to initiate a Phase 2 trial with registrational potential for BNT111 in metastatic melanoma in the second half of 2020.

BNT112 in a Phase 1/2 trial in prostate cancer.

BNT113 in a Phase 1 trial in HPV+ head and neck cancers. We are planning to initiate a Phase 2 trial with registrational potential for BNT113 in HPV+ head and neck cancers in the second half of 2020.

BNT114 in a Phase 1 trial in triple negative breast cancer. A data updated from the trail is expected in the second half of 2020.

BNT115 in a Phase 1 trial in ovarian cancer.

BNT116 is also in preclinical development for non-small cell lung cancer.

Individualized neoantigen specific immunotherapy (iNeST). Our iNeST immunotherapies contain unmodified, pharmacologically optimized mRNA encoding up to 20 patient-specific neoantigens and also feature our proprietary RNA-LPX formulation.

We, in collaboration with Genentech, initiated a randomized iNeST Phase 2 trial in first-line metastatic melanoma in combination with pembrolizumab. We and Genentech expect to report a data update from our RO7198457 (BNT122) Phase 1 trial in multiple solid tumors in June 2020, and topline data update from our RO7198457 (BNT122) Phase 2 trial in first-line melanoma in the second half of 2020. We expect this topline data update to include an update on the ongoing study, including patient enrollment numbers, with full efficacy and safety data for an interim update expected in 2021.

We and Genentech plan to initiate two additional clinical trials for RO7198457 (BNT122) in the second half of 2020 in first-line solid cancers in the adjuvant setting, one in combination with atezolizumab and the other as a monotherapy.

mRNA intratumoral immunotherapy. In collaboration with Sanofi, we are conducting a Phase 1 trial of SAR441000 (BNT131), our first mRNA-based intratumoral immunotherapy, as a monotherapy and in combination with cemiplimab in patients with solid tumors. SAR441000 (BNT131) consists of a modified mRNA that encodes the IL-12sc, IL-15sushi, GM-CSF and IFN-a cytokines. SAR441000 (BNT131) is designed to be administered directly into the tumor in order to alter the tumor microenvironment and enhance the immune system’s ability to recognize and fight cancer within the tumor (proximal) as well as in other untreated locations (distal). We expect to report a data update in the second half of 2020.

CLDN6 CAR-T cell immunotherapy. We are developing a proprietary chimeric antigen receptor T cell, or CAR-T, product candidate, BNT211, targeting Claudin-6, or CLDN6, a novel solid tumor-specific antigen. We developed BNT211 utilizing our target discovery engine, and we plan to administer it along with a CARVac "primer" to boost the immune response and promote CAR-T cell persistence. We expect to initiate a Phase 1/2 clinical trial for BNT211 in patients with advanced CLDN6 + solid tumors in the second half of 2020.

Next-generation checkpoint immunomodulators. We are developing, in collaboration with Genmab, novel next-generation bispecific antibodies that are designed for conditional activation of immunostimulatory checkpoint molecules. Our first bispecific candidates are GEN1046 (BNT311), which targets PD-L1 in conjunction with 4-1BB, and GEN1042 (BNT312), which targets CD40 in conjunction with 4-1BB. While 4-1BB is a known immune checkpoint target that is expressed on T cells and natural killer, or NK, cells, prior attempts to target 4-1BB with monoclonal antibodies have been severely limited by liver toxicities. Our 4-1BB targeting product candidates are designed to avoid toxicities by conditionally activating a 4-1BB receptor only together with the binding of either PD-L1 or CD40. We have initiated Phase 1/2a trials of GEN1046 (BNT311) and GEN1042 (BNT312) in solid tumors. The expansion cohort has been initiated in the Phase 1/2 trial in multiple solid tumors for BNT311 (PD-L1x4-1BB). BioNTech expects to provide a data update, to include dose-escalation and potentially some limited expansion data from the trial in 2H 2020.

Targeted Cancer Antibodies. BNT321 is a fully human IgG1 monoclonal antibody targeting sialyl Lewis A (sLea), a novel epitope expressed specifically in pancreatic and other solid tumors. MVT-5873 (BNT321) is currently in Phase 1 clinical development in pancreatic cancer, which we resumed in December 2019 upon the enrollment of the first patient.

Small molecule immunomodulators. BNT411 is our novel small molecule TLR7 agonist product candidate. BNT411 is engineered for high potency and high selectivity for the TLR7 receptor to activate both the adaptive and innate immune system. BNT411 will be given as a monotherapy or in combination with chemotherapy and/or checkpoint inhibitors in multiple solid tumors, including colorectal cancer, bladder cancer and small cell lung cancer. We expect to initiate a Phase 1 clinical trial for BNT411 in solid tumors in the second half of 2020.

In addition, we have several other cancer immunotherapy programs in development, including:

RiboMabs: novel classes of mRNA-based therapeutics that are designed to encode antibodies directly in the patient’s body. We expect to initiate Phase 1 clinical trials for our first two RiboMab product candidates, BNT141 and BNT142, both in the first half of 2021.

RiboCytokines: novel classes of mRNA-based therapeutics that are designed to encode cytokines directly in the patient’s body. We expect to initiate Phase 1 clinical trials for our first RiboCytokine product candidates, BNT151 and BNT152/BNT153 (combination), in the first half of 2021.

TCR therapy: T cells with engineered TCRs that are designed to specifically target cancer cells.

Infectious Disease Immunotherapies

We have collaborated with third parties to exploit the immunotherapeutic properties of our mRNA drug class for the treatment and prevention of infectious diseases.

COVID-19 Vaccine Program

In response to the Coronavirus global pandemic, the company assembled a global consortium of partners including Pfizer (worldwide collaboration outside of China) and Fosun Pharma (China). BioNTech’s vaccine program against COVID-19, BNT162, leverages the Company’s proprietary mRNA platform. Currently there are four vaccine candidates, two of the four vaccine candidates include a nucleoside modified mRNA (modRNA), one includes a uridine containing mRNA (uRNA), and the fourth vaccine candidate utilizes self-amplifying mRNA (saRNA). Each mRNA format is combined with a lipid nanoparticle (LNP) formulation. The larger spike sequence is included in two of the vaccine candidates, and the smaller optimized receptor binding domain (RBD) from the spike protein is included in the other two candidates.

BNT162 (Europe) – The first cohort of our Phase 1/2 clinical trial in Europe has been dosed. Twelve study participants were dosed with vaccine candidate BNT162 as of April 29th. The dose escalation portion of the Phase 1/2 trial will include approximately 200 healthy subjects between the ages of 18 to 55 and will target a dose range of 1 µg to 100 µg, aiming to determine the optimal dose for further studies as well as to evaluate the safety and immunogenicity of the vaccine. The three vaccine candidates that utilize uRNA or modRNA wil be administered as two injections. The fourth vaccine candidate, which contains saRNA, will be evaluated after a single dose of vaccine. Subjects with a higher risk of severe COVID-19 disease will be included in the second part of the study. First clinical data from the trial is expected end of June or in July 2020.

BNT162 (U.S) – The first cohort has been dosed in the U.S. in the Phase 1/2 clinical trial for the BNT162 vaccine program. The Phase 1/2 study is designed to determine the safety, immunogenicity and optimal dose level of the four mRNA vaccine candidates. The dose level escalation portion (Stage 1) of the Phase 1/2 trial in the U.S. will enroll up to 360 healthy subjects into two age cohorts (18-55 and 65-85 years of age) and will be seamlessly followed by administering the selected vaccine candidate to several thousands of subjects. The first subjects immunized in Stage 1 of the study will be healthy adults 18-55 years of age.

We will provide clinical supply of the BNT162 vaccine from our GMP-certified mRNA manufacturing facilities in Europe. We and Pfizer will work together to scale-up manufacturing capacity at risk to provide worldwide supply in response to the pandemic. We believe we have the potential to supply millions of vaccine doses by the end of 2020 subject to technical success of the development program and approval by regulatory authorities, and then rapidly scale up capacity to produce hundreds of millions of doses in 2021.

Flu vaccine: In August 2018, we entered into a collaboration with Pfizer to develop mRNA-based immunotherapies for the prevention of influenza, product candidate BNT161. We expect to begin clinical testing in the first half of 2021

Infectious diseases: In October 2018, we entered into a research collaboration with Penn, under which we have the exclusive option to develop and commercialize mRNA immunotherapies for the treatment of up to 10 infectious disease indications. In August 2019, we entered into a letter agreement and investment agreement with the Bill & Melinda Gates Foundation to advance the development of immunotherapies for the prevention and/or treatment of HIV and tuberculosis and up to three additional infectious diseases.

Rare Disease Protein Replacement Therapies

We are collaborating with Genevant in order to capitalize on opportunities for our mRNA technology in rare disease indications potentially featuring expedited paths to market. We are combining our mRNA technology with Genevant’s lipid nanoparticle, or LNP, delivery technology to create up to five mRNA protein replacement therapies for the treatment of rare diseases with high unmet medical needs. We expect our first compound to enter the clinic in the first half of 2021.