Poseida Therapeutics Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

On August 8, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported the closing of its previously announced underwritten public offering of 23,000,000 shares of its common stock at a public offering price of $3.50 per share, including 3,000,000 shares sold pursuant to the full exercise of the underwriters’ option to purchase additional shares (Press release, Poseida Therapeutics, AUG 8, 2022, View Source [SID1234617821]). All of the shares were sold by Poseida. Including the option exercise, the aggregate gross proceeds to Poseida from the offering, before deducting underwriting discounts and commissions and other offering expenses, were $80.5 million.

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Piper Sandler and William Blair & Company, L.L.C. acted as joint book-running managers for the offering. Cantor Fitzgerald & Co. and BTIG, LLC also acted as book-running managers for the offering.

The offering was made pursuant to a shelf registration statement on Form S-3, including a base prospectus, previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). The offering was made only by means of a written prospectus and prospectus supplement that formed a part of the registration statement. A copy of the final prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available for free on the website of the SEC at www.sec.gov. Copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained by contacting Piper Sandler, Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by email at [email protected]; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687 or by email 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 the registration or qualification under the securities laws of any such state or other jurisdiction.

VolitionRx Limited Schedules Second Quarter 2022 Earnings Conference Call and Business Update

On August 8, 2022 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported it will host a conference call on Thursday August 11, at 08.30 a.m. Eastern Time to discuss its financial and operating results for the second quarter 2022, in addition to providing a business update (Press release, VolitionRX, AUG 8, 2022, View Source [SID1234617820]).

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Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, Chief Financial Officer, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on important events which have taken place in the second quarter of 2022 and upcoming milestones.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until August 25, 2022. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13732149.

Rocket Pharmaceuticals Reports Second Quarter 2022 Financial Results and Highlights Recent Progress

On August 8, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, reported its financial results for the quarter ending June 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 8, 2022, View Source [SID1234617819]).

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"Rocket continues on an excellent trajectory following a strong and highly productive quarter that featured positive results across all four clinical programs, concrete steps taken toward manufacturing readiness and leadership, and filing preparedness for our first two gene therapies," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "Positive safety data from our Phase 1 study for Danon Disease demonstrated RP-A501 was well-tolerated in both pediatric patients. We now look forward to presenting early efficacy data from the pediatric cohort with three to six months of follow-up in late Q3. If early signals of efficacy and ongoing tolerability in the pediatric cohort are demonstrated along with evidence of longer-term safety and efficacy in the adult cohort, we expect to begin Phase 2 pivotal trial planning activities in Q4, including FDA alignment on study design and endpoints."

Dr. Shah continued, "In parallel, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal trial in Danon Disease. To further strengthen our manufacturing and commercial capabilities, we appointed Mayo Pujols, one of the most seasoned cell and gene therapy technical operations and manufacturing leaders in the industry, as our Chief Technical Officer."

"This quarter, we also shared positive top-line data from our pivotal Phase 2 trial for severe LAD-I showing that RP-L201 was well-tolerated and associated with 100% overall survival at one year," said Dr. Shah. "We have initiated work towards regulatory filings planned for the first half of 2023. In addition, based on achievement of the primary endpoint in our pivotal Phase 2 study for Fanconi Anemia, we have initiated FDA dialogue around BLA planning activities."

"These steps deliver on our effort to best leverage our strong cash position to create value as we embark on transitioning from a clinical to a commercial-stage company. Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024," concluded Dr. Shah. "Taken together, our positive data updates and steady progress this quarter continue to motivate us to push the boundaries of science and deliver on our mission to seek gene therapy cures for patients and families facing devastating, life-threatening diseases."

Key Pipeline and Operational Updates

Danon, FA, LAD-I and PKD trials remain on track. All 2022 milestones remain on track, including pediatric efficacy data readout from the Phase 1 Danon Disease trial in late Q3, updated results for FA and preliminary Phase 1 data readout for PKD in Q4. The originally planned Q3 topline readout for FA was achieved earlier than anticipated in Q2 when the trial met its primary endpoint.
Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included new initial safety data from the low-dose (6.7 x 1013GC/kg; n=2) pediatric cohort as of April 30, 2022, cut-off date. Results demonstrated RP-A501 was well-tolerated in both patients. The patients were observed to have normal-range platelets, diminished complement activation and no complement-related adverse events. Early efficacy and safety data with three to six months of follow up from the pediatric patient cohort of the Phase 1 trial are expected in late Q3 2022; longer-term safety and efficacy for adults will also be presented. Pending health authority interactions, Phase 2 trial planning activities are expected to begin in Q4 of 2022.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L102 for Fanconi Anemia (FA). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included updated data from the initial nine of 12 FA patients who received RP-L102 as of the April 4, 2022, cut-off date. Five of nine evaluable patients had increased resistance to mitomycin-C in bone marrow-derived colony forming cells, ranging from 21% to 42% at 12 to 18 months, increasing to 51% to 94% at 18 to 21 months. The primary endpoint has been achieved, based on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints and concomitant evidence of genetic correction and clinical stabilization. The safety profile of RP-L102 appears favorable with no signs of dysplasia, clonal dominance or oncogenic integrations; as previously reported, one patient experienced a Grade 2 transient infusion related reaction, which resolved. Based on these results, the Company has initiated FDA dialogue in anticipation of BLA filing activities.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all nine patients as of the March 9, 2022, cut-off date and overall survival data for the seven patients with at least 12 months after infusion. All patients demonstrated sustained CD18 restoration and expression on more than 10% of neutrophils (range: 20%-87%, median: 56%), as well as a statistically significant reduction in the rate of all-cause hospitalizations and severe infections, relative to pre-treatment. At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort was 100% based on the Kaplan-Meier estimate. RP-L201 was well-tolerated with no drug product-related serious adverse events as of the cut-off date. Based on the data presented at ASGCT (Free ASGCT Whitepaper), Rocket has initiated discussions with health authorities on filing plans for RP-L201 for the treatment of severe LAD-I and anticipates filings in the first half of 2023.
Announced positive clinical data from ongoing Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included interim data from two adult PKD patients with severe and transfusion dependent anemia who were treated with RP-L301 as of the April 13, 2022, cut-off date. At 18 months post-infusion, both patients had sustained transgene expression, normalized hemoglobin, improved hemolysis, no red blood cell transfusion requirements post-engraftment and improved quality of life, both reported anecdotally and as documented via formal quality of life assessments. RP-L301 appears favorable with no drug product-related serious adverse events through 18 months post-infusion. Transient transaminase elevation was seen in both patients post-therapy/conditioning, with no clinical stigmata of liver injury and subsequent resolution without clinical sequelae. Enrollment in the pediatric cohort is ongoing, and additional Phase 1 data are expected in Q4 2022.
Achieved in-house AAV cGMP manufacturing readiness. The Company’s state-of-the-art, 103,720 ft2 manufacturing facility in Cranbury, New Jersey has been scaled up to manufacture AAV drug product for a planned Phase 2 pivotal study in Danon Disease. The facility also houses lab space for research and development and quality.
Appointed Chief Technical Officer and expanded leadership team. In July 2022, Mayo Pujols joined the Company as its first Chief Technical Officer and Executive Vice President. Mr. Pujols brings nearly three decades of experience from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing. He most recently served as Chief Executive Officer of Andelyn Biosciences, a leading gene therapy contract development and manufacturing organization (CDMO), and prior to Andelyn was the Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals. Mr. Pujols has also served in key technical operations and manufacturing roles at Celgene, Merck, Advaxis, MedImmune and Schering-Plough. In his new role, Mr. Pujols leads the technical operations function and chemistry, manufacturing and controls (CMC) for all lentiviral programs. Additionally, he leads the Company’s state-of-the-art adeno-associated virus (AAV) manufacturing facility.
Upcoming Investor Conferences

BTIG Biotechnology Conference – August 8-9, 2022
Citi 17th Annual BioPharma Conference – September 7-8, 2022
Morgan Stanley 20th Annual Global Healthcare Conference – September 12-14, 2022
Second Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2022, were $321.4 million.
R&D expenses. Research and development expenses were $41.4 million for the three months ended June 30, 2022, compared to $24.5 million for the three months ended June 30, 2021. The increase in research and development expense was primarily driven by an increase in manufacturing and development costs, an increase in compensation and benefits expense due to increased R&D headcount and an increase in laboratory supplies.
G&A expenses. General and administrative expenses were $12.9 million for the three months ended June 30, 2022, compared to $9.5 million for the three months ended June 30, 2021. The increase in general and administrative expenses was primarily driven by an increase in commercial preparation expenses, an increase in compensation and benefits expense due to increased G&A headcount, and an increase in legal expenses.
Net loss. Net loss was $54.4 million or $0.83 per share (basic and diluted) for the three months ended June 30, 2022, compared to $34.5 million or $0.55 per share (basic and diluted) for the three months ended June 30, 2021.
Shares outstanding. 65,837,894 shares of common stock were outstanding as of June 30, 2022.
Financial Guidance

Cash position. As of June 30, 2022, the Company had cash, cash equivalents and investments of $321.4 million. As of June 30, 2022, the Company sold 1.3 million shares of common stock for net proceeds of $17.3 million under its at-the-market facility. With the at-the-market facility proceeds, the Company expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2024, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our four clinical programs as well as future pipeline programs.

Arcus Biosciences Appoints Dimitry Nuyten, M.D., Ph.D. as Chief Medical Officer

On August Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported that Dimitry S.A. Nuyten, M.D., Ph.D has been appointed chief medical officer (CMO) effective August 1, 2022 (Press release, Arcus Biosciences, AUG 8, 2022, View Source [SID1234617818]). In his role as CMO, Dr. Nuyten will oversee Arcus’s clinical development organization that includes nearly 200 employees and six clinical-stage programs targeting TIGIT, the adenosine axis (CD73 and dual A2a/A2b), HIF-2a and PD-1. Dr. Nuyten will oversee the advancement of four registrational Phase 3 trials that are ongoing or expected to start by year end for the anti-TIGIT antibody domvanalimab in novel combinations across multiple cancers.

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"Dr. Nuyten’s attributes and experiences are well matched to Arcus’s rapid evolution as we enter 2023 with four ongoing registrational trials for domvanalimab-based combinations and a portfolio of Phase 2 and early signal-seeking trials investigating combinations of our six clinical molecules," said Terry Rosen, Ph.D., chief executive officer of Arcus. "His proven abilities to grow and lead large cross-functional teams, interface with commercial and regulatory organizations and navigate complex portfolio management will complement our exceptional development organization and facilitate the continued growth of Arcus. In Dr. Nuyten’s new role, he will also be working closely with our global partners, including Gilead Sciences, to optimize and implement our clinical development strategy."

As part of his role as CMO, Dr. Nuyten will serve as a member of Arcus’s executive committee and co-chair of Arcus’s and Gilead’s Joint Steering Committee. He will be responsible for our clinical organization, including clinical development and operations, clinical pharmacology and biometrics. Prior to joining Arcus, Dr. Nuyten served as senior vice president and CMO of Nektar Therapeutics where he led a 200-person development organization which included clinical development, safety, clinical operations, medical affairs, clinical and non-clinical pharmacology, biostats, data management and programming. Prior to Nektar, he served as CMO of Aduro Biotech and served as the immuno-oncology development leader and vice president of global product development for oncology at Pfizer, where he led the late-stage development of Bavencio and early-stage clinical programs for oncology and immune-oncology. Prior to Pfizer, Dr. Nuyten was group medical director at BristolMyers Squibb. He holds a Ph.D. in cancer biology from the University of Amsterdam Medical School in The Netherlands, is Board Certified in radiation oncology and certified as a physician in The Netherlands by the University of Groningen Medical School. Over the course of his career, Dr. Nuyten has authored numerous peer-reviewed papers, is co-inventor on multiple patents and has been recognized with prestigious awards, including as a two-time recipient of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Merit Award.

"My career has been dedicated to understanding and exploiting the biologic drivers of cancer to develop new treatments that have the potential to improve outcomes or even cure cancer," said Dimitry Nuyten, M.D., Ph.D, incoming chief medical officer at Arcus Biosciences. "I was attracted to Arcus by the breadth and diversity of its portfolio of molecules and clinical programs and the corresponding opportunity to substantially impact the way that many important cancers are treated. I am thrilled to join the company and excited to work with the talented and patient-centric Arcus team to translate an innovative pipeline into clinically meaningful therapies for a broad array of cancers with high unmet medical need."

Arcus Ongoing and Announced Clinical Studies

Trial Name

Arms

Setting

Status

NCT No.

Lung Cancer

ARC-7

zim vs. dom + zim vs. etruma + dom + zim

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Randomized Phase 2

NCT04262856

PACIFIC-8

(AZ)

dom + durva vs. durva

Curative-Intent Stage 3 NSCLC

Ongoing Registrational Phase 3

NCT05211895

ARC-10

dom + zim vs. zim vs. chemo

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Registrational Phase 3

NCT04736173

STAR-121

(GILD)

dom + zim + chemo vs pembro + chemo

1L NSCLC (PD-L1 all-comers)

Planned Registrational Phase 3

TBD

EDGE-Lung

dom + zim +/- quemli

1L/2L NSCLC (lung cancer platform study)

In Planning Phase 2

TBD

Lung Platform (GILD)

dom + zim +/- etruma or sacituzumab govitecan (Trodelvy) or other combos

1L/2L NSCLC (lung cancer platform study)

In Planning Phase 2

TBD

Gastrointestinal Cancers

ARC-9

etruma + zim + mFOLFOX vs. SOC

2L/3L/3L+ CRC

Ongoing

Randomized Phase 2

NCT04660812

ARC-21

dom + zim ± chemo

1L/2L Upper GI Malignancies

Ongoing

Phase 2

NCT05329766

STAR-221

dom + zim + chemo vs. nivo + chemo

GI Malignancies

Planned Registrational Phase 3

TBD

Pancreatic Cancer

ARC-8

quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac

1L, 2L PDAC

Ongoing Randomized Phase 1/1b

NCT04104672

Prostate Cancer

ARC-6

etruma + zim + SOC vs. SOC (Adding sacituzumab govitecan (Trodelvy) combination cohorts)

2L/3L CRPC

Ongoing Randomized Phase 2

NCT04381832

Various

ARC-12

AB308 + zim

Advanced Malignancies

Ongoing

Phase 1/1b

NCT04772989

ARC-14

AB521

Healthy Volunteers

Ongoing

NCT05117554

ARC-20

AB521

Cancer Patients / ccRCC

Planned Phase 1/1b

TBD

dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; nivo: nivolumab; pembro: pembrolizumab; quemli: quemliclustat; SOC: standard of care; zim: zimberelimab; ccRCC: clear-cell renal cell carcinoma

About the Gilead Collaboration

In May 2020, Gilead and Arcus entered into a 10-year collaboration that provided Gilead immediate rights to zimberelimab and the right to opt into all other Arcus programs arising during the collaboration term. In November 2021, Gilead and Arcus amended the collaboration in connection with Gilead’s option exercise for three of Arcus’s then-clinical stage programs. For all other programs that are in clinical development or new programs that enter clinical development thereafter, the opt-in payments are $150 million per program. Gilead’s option, on a program-by-program basis, expires after a specified period of time following the achievement of a development milestone for such program and Arcus’s delivery to Gilead of the requisite qualifying data package. Concurrent with the May 2020 collaboration agreement, Gilead and Arcus entered into a stock purchase agreement under which Gilead made a $200 million equity investment in Arcus. That stock purchase agreement was amended and restated in February 2021 in connection with Gilead’s increased equity stake in Arcus from 13% to 19.5%, with an additional $220 million investment.

Gilead and Arcus are co-developing and equally share global development costs for five clinical candidates, including domvanalimab, an Fc-silent anti-TIGIT antibody, etrumadenant, a dual adenosine A2a/A2b receptor antagonist, quemliclustat, a small molecule inhibitor of CD73, and zimberelimab, an anti-PD1 antibody.

Guardant Health to Participate in the UBS Genomics 2.0 and MedTech Innovations Summit Conference

On August 8, 2022 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported it will be participating in the upcoming UBS Genomics 2.0 and MedTech Innovations Summit Conference in Dana Point, CA (Press release, Guardant Health, AUG 8, 2022, View Source [SID1234617817]).

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Guardant Health’s management is scheduled to participate in a fireside chat on Wednesday, August 10 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. Interested parties may access a live and archived webcast of the presentation on the "Investors" section of the company website at: www.guardanthealth.com.