On November 3, 2021 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, and The Royal Marsden NHS Foundation Trust, one of the United Kingdom’s (UK) leading cancer research organizations, reported that they have agreed to enter into a partnership to establish an in-house liquid biopsy testing service using Guardant Health’s industry-leading proprietary digital sequencing platform (Press release, Guardant Health, NOV 3, 2021, View Source [SID1234594275]).

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The service is expected to become operational at the end of 2022 and will be available for clinical research and clinical care. This will be the first UK hospital for Guardant Health to house a dedicated liquid biopsy testing facility for cancer diagnostics.

"We are delighted to announce this partnership with Guardant Health, establishing a state-of-the-art liquid biopsy testing facility. In a clinical diagnostic setting, it will provide many of our patients with more rapid access to bespoke diagnostic testing, leading to earlier, faster, and more accurate diagnosis, along with targeted treatment selection and monitoring," said Professor Michael Hubank, Director of Clinical Genomics (Research) at The Royal Marsden NHS Foundation Trust. "The opening of this service will also allow us to significantly increase our capacity for research using liquid biopsies. Bringing expanded capacity for genomic testing, the facility will help us identify more people with cancer for clinical trials based on targeted treatments, improving outcomes for patients across the UK and beyond."

It is estimated that there were around 375,000 new cancer cases in the UK from 2016-20181, with approximately 166,000 cancer deaths in the UK from 2016-2018.1 Guardant Health’s tests are used by oncologists around the world to guide treatment decisions across solid tumor cancers, and by pharmaceutical companies and academic researchers in clinical trials to accelerate precision medicine drug development. Guardant Health has achieved CE Mark and U.S. Food and Drug Administration approval for its Guardant360 CDx assay for tumor mutation profiling, also known as comprehensive genomic profiling (CGP), in patients with any solid cancerous tumor.

"We are pleased to announce this partnership with The Royal Marsden NHS Foundation Trust, which will enable clinicians to quickly and accurately detect relevant tumor alterations with a blood test to guide personalized treatment plans and provide researchers with diagnostic tools to support clinical research and innovation," said Helmy Eltoukhy, Guardant Health Co-CEO. "This partnership further advances our goal of ensuring all patients with cancer have access to the latest innovations to inform their treatment management to enable the best possible outcomes."

On November 3, 2021 ANI Pharmaceuticals, Inc. (NASDAQ: ANIP) ("ANI") reported that it intends to offer and sell shares of its common stock in an underwritten public offering (Press release, ANI Pharmaceuticals, NOV 3, 2021, View Source [SID1234594274]). All of the shares to be sold in the offering will be offered by ANI. The offering is subject to market 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. In addition, ANI intends to grant the underwriters a 30-day option to purchase up to an additional 15% of shares of its common stock offered in the public offering.

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Guggenheim Securities is acting as book-running manager.

ANI anticipates using the net proceeds from the offering to fund its Cortrophin commercialization efforts, including, but not limited to, sales and marketing and consulting expenses related thereto, and for general corporate purposes. We may also use a portion of the net proceeds to in-license, acquire or invest in additional businesses, technologies, products or assets.

The securities described above are being offered by ANI pursuant to a shelf registration statement on Form S-3 (File No. 333-239771) which was initially filed by the Company with the Securities and Exchange Commission (the "SEC") on July 9, 2020, and was declared effective by the SEC on July 17, 2020.

The securities will be offered only by means of a prospectus supplement and accompanying prospectus relating to the offering that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the final prospectus supplement, when available, and accompanying prospectus relating to the offering may be obtained from Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, 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 November 3, 2021 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported updated clinical data from a Phase 1 study of tidutamab, an SSTR2 x CD3 bispecific antibody, in patients with neuroendocrine tumors (NETs) (Press release, Xencor, NOV 3, 2021, View Source [SID1234594273]). The data will be presented during the North American Neuroendocrine Tumor Society’s 2021 Multidisciplinary NET Medical Virtual Symposium (NANETS).

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"The Phase 1 study of tidutamab in patients with neuroendocrine tumors informed our view that an XmAb CD3 bispecific antibody is generally well tolerated in solid tumors, with a low incidence and severity of CRS, and can induce meaningful biological activity in a challenging disease setting," said Allen Yang, M.D., Ph.D., senior vice president and chief medical officer at Xencor. "Importantly, we identified a recommended dose for continued study, and a Phase 1b/2 study has been opened to evaluate tidutamab as a potential treatment option for patients with Merkel cell carcinoma and small cell lung cancer, SSTR2-expressing tumor types known to be responsive to immunotherapy."

A poster with data from the study is available in the NANETS Virtual Poster Hall, and it will be made available under Archived Scientific Presentations on the Events & Presentations page in the Investors section of www.xencor.com. In addition to the poster, these results will be presented during the Clinical Abstracts session, which begins at 1:25 p.m. ET on Saturday, November 6, 2021.

Key Highlights

The primary objectives of the Phase 1 study were to determine the safety and tolerability profile of tidutamab in patients with advanced, well-differentiated NETs of pancreatic, gastrointestinal, lung and undetermined origin, and to identify the maximum tolerated dose and/or recommended dosing regimen for continued study, which was determined to be a 0.3 mcg/kg priming dose followed by 1.0 mcg/kg on subsequent dosing days.

At data cut-off in August 2021, 41 patients with neuroendocrine tumors, with the initial lesion location in the pancreas (46%), intestine (22%), lung (20%), and other GEP-NET or unknown (12%), received doses of tidutamab ranging from 0.1 to 2.0 mcg/kg. Dosing in the study included a lower priming dose, followed by a higher repeated dose on subsequent dosing days. The 20 patients in the expansion cohort received the recommended dosing regimen. Patients had a median age of 64 years and a median of four prior lines of systemic therapies. Fifty percent of patients received prior peptide receptor radionuclide therapy.

Tidutamab was generally well tolerated at the 0.3/1.0 mcg/kg dose identified for the expansion portion of the study. All 41 patients treated were included in the safety analysis. The most common treatment-related Grade 3 or Grade 4 adverse events across all doses were lymphopenia (29%), gamma-glutamyl transferase increases (20%), transaminase increases (20%) and vomiting (17%). In addition, Grade 3 esophageal dysmotility was observed in 7% of patients. CRS of any grade was observed in 41% of patients. The majority of CRS was limited to Grade 1 and Grade 2 and to the first two doses. Grade 3 CRS was observed in two patients (5%) upon the first dose. All patients experiencing CRS fully recovered.

Analysis of peripheral blood biomarkers indicated that tidutamab induced acute and sustained T-cell activation at the recommended dose for expansion. CD8-positive effector T cells showed a dose-dependent increase in proliferation (Ki67) and activation (PD-1) markers that began within 48 hours of the first dose and persisted at least seven weeks, as measured at cycle 2, day 22.

The best overall response was stable disease, with a disease control rate of 27%. Higher tumor PD-L1 expression was associated with a shorter time on study, and the patients with prolonged stable disease were most likely to have low or absent PD-L1 expression, suggesting a potential beneficial effect from the combination of tidutamab with PD-L1 inhibitors.

About Tidutamab

Tidutamab is a tumor-targeted bispecific antibody that contains both an SSTR2 binding domain and a T-cell binding domain (CD3). An XmAb bispecific Fc domain serves as the scaffold for the two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on tidutamab. SSTR2 (somatostatin receptor 2) is an antigen highly expressed on some solid tumors, and engagement of CD3 by tidutamab activates T cells for highly potent and targeted killing of SSTR2-expressing tumor cells. Tidutamab is being evaluated in an ongoing Phase 1b/2 study, which is enrolling patients with Merkel cell carcinoma and small cell lung cancer, SSTR2-expressing tumor types known to be responsive to immunotherapy.

On November 3, 2021 PAVmed Inc. (Nasdaq: PAVM, PAVMZ), a highly differentiated, multi-product, commercial-stage medical device company, and its major subsidiary Lucid Diagnostics Inc. (Nasdaq: LUCD), a cancer prevention medical diagnostics company reported that the companies will host a joint business update conference call on Tuesday, November 16, 2021, at 4:30 PM EDT (Press release, PAVmed, NOV 3, 2021, View Source [SID1234594272]). During the call, Lishan Aklog, M.D., Chairman and Chief Executive Officer of the companies, will provide a business update including an overview of the Company’s near-term milestones and growth strategy. In addition, Dennis McGrath, the companies’ Chief Financial Officer, will discuss the companies’ third quarter 2021 financial results.

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To access the conference call, U.S.-based listeners should dial 877-407-3982 and international listeners should dial 201-493-6780. All listeners should provide the operator with the conference call name "PAVmed, Inc. Business Update Conference Call" to join. Individuals interested in listening to the live conference call via webcast may do so by visiting the investor relations section of the Company’s website at www.pavmed.com.

Following the conclusion of the conference call, a replay will be available for one week and can be accessed by dialing 844-512-2921 from within the U.S. or 412-317-6671 from outside the U.S. To access the replay, all listeners should provide the following pin number: 1374402. The webcast will be available for replay on the investor relations section of the Company’s website at www.pavmed.com.

On November 3, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported its financial results for the quarter ending September 30, 2021 and updates on the Company’s key pipeline developments, business operations, and upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 3, 2021, View Source [SID1234594271]).

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"We are excited about the strong progress we made in the third quarter as we initiated treatment in our trial of RP-A501 for Danon Disease in the low-dose (6.7e13 vg/kg) pediatric patient cohort," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "I am proud of our team’s collaboration with the FDA, through which we were able to swiftly resolve the clinical hold on the Danon trial and resume this highly important work on behalf of Danon patients. We are gearing up for the remainder of 2021 and look forward to reporting a comprehensive clinical update on our Phase I trial in Danon Disease this month at the AHA Scientific Sessions as well as key updates on all five of our first-in-class gene therapy programs throughout the fourth quarter."

Dr. Shah continued, "We are equally excited about the progress of our LAD-I program, where we completed treatment for all nine patients in the RP-L201 Phase 1/2 clinical trial and presented positive interim data updates on the initial seven patients at the ESGCT Congress in October. Based on the data presented from these seven patients, RP-L201 continues to demonstrate a favorable safety profile and preliminary clinical benefit in patients with severe LAD-I. We will share additional clinical data from the LAD-I trial at the 63rd ASH (Free ASH Whitepaper) Annual Meeting in December, where we will also report clinical updates on our Fanconi Anemia and PKD programs. I am proud of the Rocket team’s unwavering dedication to developing and bringing life-changing curative therapies to patients with rare diseases."

Key Pipeline and Operational Updates

Danon Disease:

Initiated pediatric patient treatment in Phase 1 trial of RP-A501 for the treatment of Danon Disease. Rocket has resumed patient enrollment and initiated treatment in the low-dose (6.7e13 vg/kg) pediatric patient cohort. A comprehensive clinical update is anticipated at the American Heart Association (AHA) Scientific Sessions 2021 being held virtually November 13-15, 2021.
Presented previously disclosed data from ongoing RP-A501 Phase 1 trial in Danon Disease at HFSA. The late-breaking oral presentation at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021 overviewed data from the low-dose (6.7e13 vg/kg) adult cohort which demonstrated RP-A501 was well tolerated and showed progressive and durable clinical benefit.
Leukocyte Adhesion Deficiency-I (LAD-I):

Presented positive interim data updates from RP-L201 LAD-I trial at ESGCT. The oral presentation included data from the initial seven patients with severe LAD-I who were treated with RP-L201 in the Phase 1/2 trial. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related serious adverse events. Preliminary efficacy was evident in all seven patients, including two patients with at least 12 months of follow-up. All seven patients demonstrated durable neutrophil CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent with reversal of the severe LAD-I phenotype. Peripheral blood vector copy number (VCN) levels have been stable and in the 0.5 – 2.5 copy per genome range. No patients have had LAD-I related infections requiring hospitalization subsequent to hematopoietic reconstitution post RP-L201. Additional clinical data are anticipated at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Fanconi Anemia (FA):

Presented previously disclosed clinical data from RP-L102 Fanconi Anemia (FA) program at ESGCT. The oral presentation included data for nine pediatric patients treated with RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate, in the ongoing clinical trials. RP-L102 demonstrated a highly favorable safety profile with all subjects being treated without conditioning and with no sign of dysplasia or other concerning features. RP-L102 showed evidence of preliminary engraftment in at least six of nine patients. A clinical update is anticipated at the 63rd ASH (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Pyruvate Kinase Deficiency (PKD):

Presented incremental updates from RP-L301 Pyruvate Kinase Deficiency (PKD) program at ESGCT. The oral presentation included data from two adult patients treated with RP-L301, Rocket’s ex vivo lentiviral gene therapy candidate, in the ongoing Phase 1 trial. The safety profile of RP-L301 appears favorable with no infusion-related serious adverse events at up to 9 months post-infusion. Both patients have normalized hemoglobin, improved hemolysis markers and no red blood cell transfusion requirements post-engraftment, as well as no hospitalizations post-hospital discharge. A clinical update is anticipated at the 63rd ASH (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Published peer-reviewed studies supporting scientific rationale for clinical results observed to date in RP-L301 trial. "Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment" was published in Molecular Therapy: Methods & Clinical Development. The studies demonstrate that in the murine model of PKD, reversion of the phenotype is seen when at least 20% of hematopoietic stem/progenitors are corrected, with comprehensive reversion seen when at least 30% of these progenitors are corrected. This provides meaningful scientific rationale for the clinical results that have been observed to date for the first two patients who have received RP-L301.
Infantile Malignant Osteopetrosis (IMO):

Presented preclinical data supporting ongoing RP-L401 Infantile Malignant Osteopetrosis (IMO) Phase 1 trial at ESGCT. The Phase 1 trial is designed to assess safety and tolerability, as well as preliminary efficacy, of RP-L401, Rocket’s ex vivo lentiviral gene therapy candidate. A clinical update on the Phase 1 trial is anticipated later in the fourth quarter.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (Q4 2021)
LAD-I (RP-L201)
Longer-term Phase 2 data (Q4 2021)
Danon Disease (RP-A501)
Longer-term Phase 1 data (Q4 2021)
PKD (RP-L301)
Longer-term Phase 1 data (Q4 2021)
IMO (RP-L401)
Phase 1 clinical update (Q4 2021)
Third Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2021, were $421.5 million.
R&D expenses. Research and development expenses were $40.0 million for the three months ended September 30, 2021, compared to $21.7 million for the three months ended September 30, 2020, due to increase in manufacturing and development costs, an increase in new research agreements of $7.6 million in non-cash expenses, increases in compensation and benefits due to increased R&D headcount, and an increase in non-cash stock compensation expense.
G&A expenses. General and administrative expenses were $9.7 million for the three months ended September 30, 2021, compared to $5.7 million for the three months ended September 30, 2020, due to an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount and an increase in commercial preparation expenses.
Net loss. Net loss was $50.1 million or $0.79 per share (basic and diluted) for the three months ended September 30, 2021, compared to $29.1 million or $0.53 per share (basic and diluted) for the three months ended September 30, 2020.
Shares outstanding. 64,442,601 shares of common stock were outstanding as of September 30, 2021.
Financial Guidance

Rocket expects its balance in cash, cash equivalents and investments of $421.5 million as of September 30, 2021 to fund its operations into the second half of 2023, 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 five clinical programs.