On November 4, 2020 IO Biotech, a clinical-stage biopharmaceutical company developing novel, immune modulating anti-cancer therapies based on its proprietary T-win technology, reported that the first patient has received the first dose in a Phase 2 study (HN1901) investigating activity and safety of peptide-based immunotherapy in the preoperative setting for in-patients with squamous cell carcinoma of the head and neck (SCCHN) (Press release, IO Biotech, NOV 4, 2020, View Source [SID1234570272]). The study is being conducted in collaboration with Cliniques universitaires Saint-Luc in Brussels and is designed as an umbrella trial enabling the testing of several of IO Biotech’s compounds after initial testing of IO102 in a monotherapy setting.

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"We are very happy to start this interesting study that will help us to improve our knowledge in immunotherapy for head and neck cancer patients," said Professor Jean-Pascal Machiels, MD, PhD.

"We are excited to start this collaboration led by Professor Jean-Pascal Machiels from Institut Roi albert II Cancer Center of Cliniques universitaires Saint-Luc in Brussels," said Mai-Britt Zocca, PhD, Chief Executive Officer and founder at IO Biotech. "This study will enable IO Biotech to further validate our technology in a new indication, SCCHN, and broaden our scope to a third indication within solid tumors beyond non-small cell lung cancer (NSCLC) and melanoma."

IO Biotech’s lead candidate, IO102, is an Indoleamine 2,3-dioxygenase (IDO) derived immune modulating therapy with a dual mode of action-killing both cancer cells and immune-suppressive cells. IO Biotech’s IDO-derived immune modulating therapies have previously demonstrated both a favorable safety profile and promising anti-tumor activity in its first human clinical trial of heavily pre-treated patients with NSCLC.

About the Study
HN1901 is an open-label, randomized, Phase 2, window of opportunity trial investigating immunological changes in tumor and antitumor activity of vaccine-based immunotherapy in the pre-operative setting of patients with squamous cell carcinoma of the head and neck (SCCHN). This clinical trial is planned to sequentially test IO Biotech’s proprietary immune modulatory vaccines as monotherapy and as multi antigene vaccines at the Cliniques universitaires Saint-Luc in Brussels.

About SCCHN
Squamous cell carcinoma of the head and neck (SCCHN) is one of the most common tumor types worldwide and accounts for more than 550,000 new cases and 380,000 deaths per year. The five-year survival rate for patients suffering from locally advanced head and neck cancer is estimated between 40 and 60 percent depending on the anatomical location of the primary tumor.
IO Biotech is currently conducting studies with T-Win compounds in combination with Pembrolizumab and Nivolumab, both of which are approved for SCCHN.

About T-win Technology Platform
IO Biotech’s proprietary T-win technology platform enables identification of compounds with a dual mechanism of action targeting and directly killing immunosuppressive cells and tumor cells while indirectly activating other T-effectors, leading to strong anti-tumor responses. The company’s compounds are administered as "off-the-shelf" subcutaneous injections, distinguishing these treatments from many immuno-oncology therapies.

On November 4, 2020 Inhibikase Therapeutics reported that it has nearly halved its shares offering ahead of a now lowered IPO (Press release, Inhibikase Therapeutics, NOV 4, 2020, View Source [SID1234570261]).

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The Atlanta-based biotech had first planned to raise $25 million by offering 2.3 million shares at the $10 to $12 range, but, while keeping the range, it has slashed its offering by 40% down to 1.4 million.

Most of its drugs are preclinical, with two INDs filed last February for its leading clinical med, IkT-148009, which is targeting Parkinson’s disease as well as several other conditions.

It works as a first-in-class small molecule designed as a once-daily oral med that targets an underlying biological mechanism that leads to Parkinson’s, with "the goal of halting disease progression and reversing functional loss," according to the biotech.

IkT-148009 is designed to block the activation of Abl kinase, a clinically validated drug target, to halt and reverse the loss of dopamine-secreting neurons in the brain and GI tract by restoring neuroprotective mechanisms.

It expects to commence the first dosing of Parkinson’s patients shortly after the conclusion of the offering, according to Renaissance Capital.

It also has several other earlier-stage pipeline meds in blood cancer and progressive multifocal leukoencephalopathy.

On November 4, 2020 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported that it will present initial data from its Phase 1 UNIVERSAL trial of ALLO-715, an anti-BCMA AlloCAR T therapy, in relapsed/refractory multiple myeloma in an oral presentation at the 62nd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), taking place virtually December 5 – 8, 2020 (Press release, Allogene, NOV 4, 2020, View Source [SID1234570145]). Preclinical findings from investigations of ALLO-316, an AlloCAR T targeting CD70 in acute myeloid leukemia and ALLO-605, a BCMA-directed TurboCAR TTM cell therapy in multiple myeloma, will also be presented in poster sessions.

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"We’re looking forward to presenting initial clinical data for our first anti-BCMA AlloCAR T therapy, ALLO-715, which we believe will provide insights into how we might optimize lymphodepletion and cell dose to reach its potential for patients in need of readily available treatments options," said Rafael Amado, M.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene. "These findings will help inform trial design for our BCMA platform as we look to advance ALLO-715, alone and in combination with a gamma secretase inhibitor, as well as ALLO-605, our first TurboCAR T clinical candidate."

In the initial dose escalation phase of the UNIVERSAL trial, patients received lymphodepletion (LD) followed by ALLO-715 at one of three dose levels (DL) in a 3+3 dose escalation design. At the time of the July 2020 abstract data cutoff, two LD regimens were being evaluated:

FCA: Fludarabine 90 mg/m2, Cyclophosphamide 900 mg/m2, and ALLO-647 39 mg divided over three days; and
CA: Cyclophosphamide 900 mg/m2 and ALLO-647 39 mg divided over three days.
The ASH (Free ASH Whitepaper) abstract includes preliminary data on the first 15 patients evaluable for efficacy and treated with escalating doses of ALLO-715 as well as lower dose (39mg) ALLO-647. Patients were in advanced stage of disease with a median of five prior lines of therapy. The trial did not permit bridging therapy.
In 15 evaluable patients, higher dose of ALLO-715 (DL3) achieved greater activity with 60% (3/5) patients responding (95% CI 14.7, 94.7). Of the three patients who received DL3 FCA, two responded (1 stringent complete response (sCR) and 1 very good partial response (VGPR)) and both were minimum residual disease (MRD) negative by local MRD testing.

LD Regimen and
Cell Dose
FCA CA Overall DL3
(95% CI)
(N=5)
DL1
40 x 106
CAR+ cells
(N=3) DL2
160 x 106
CAR+ cells
(N=4) DL3
320 x 106
CAR+ cells
(N=3) DL2
160 x 106
CAR+ cells
(N=3) DL3
320 x 106
CAR+ cells
(N=2)
ORR, n (%) 0 (0%) 2 (50%) 2 (66%) 0 (0%) 1(50%) 3/5 (60%)
(14.7, 94.7)
At the time of the data cutoff, 17 of the patients were evaluable for safety. No neurotoxicity or graft-vs-host disease (GvHD) was observed. Cytokine release syndrome (CRS) was reported in four patients (24%) with three Grade 1 and one Grade 2. All CRS was resolved without tocilizumab or corticosteroids. The most common Grade ≥ 3 adverse events were anemia (41.2%), neutropenia (41.2%), lymphopenia (29.4%), and thrombocytopenia (29.4%).

Four (23.5%) instances of Grade ≥ 3 infections were observed. Three of these were Grade 3 and resolved with treatment. The fourth was a Grade 5 event of suspected fungal pneumonia that occurred on day eight post-ALLO-715 infusion. The suspected fungal pneumonia was diagnosed on the day after cell infusion in this patient with advanced and rapidly progressing disease who had failed multiple lines of therapy. This event occurred in the CA cohort, and it was assessed by the investigator as related to progressive disease and the CA conditioning.

The oral presentation will include data on approximately 20 patients evaluable for efficacy across ALLO-715 cell dose cohorts and lower dose (39mg) of ALLO-647, as well as patients evaluable for efficacy who were treated with a higher dose of ALLO-715 and higher doses of ALLO-647. The Phase 1 UNIVERSAL study continues to enroll patients at these higher doses in an effort to optimize the therapy.

The ASH (Free ASH Whitepaper) abstracts are now available at www.hematology.org. Allogene will also host a conference call on December 5th following the virtual presentation. Details on the ASH (Free ASH Whitepaper) presentations are as follows:

Allogene Oral Presentation

Session: 653. Myeloma/Amyloidosis: Therapy, Excluding Transplantation; CAR T Therapies for Myeloma: Novel Approaches and Longer-Term Follow Up Data
Abstract #129
Title: Universal: An Allogeneic First-in-Human Study of the Anti-BCMA ALLO-715 and the Anti-CD52 ALLO-647 in Relapsed/Refractory Multiple Myeloma
Presenter: Sham Mailankody, M.D., Memorial Sloan Kettering Cancer Center
Session Date & Time: Saturday, December 5, 2020; 9:30 a.m. – 11 a.m. PT

Allogene Poster Presentations

Session: 616. Acute Myeloid Leukemia: Novel Therapy, Excluding Transplantation: Poster II
Abstract #1972
Title: Investigation of ALLO-316: A Fratricide-Resistant Allogeneic CAR T Targeting CD70 As a Potential Therapy for the Treatment of AML
Presenter: Nguyen Tan, Allogene Therapeutics
Session Date & Time: Sunday, December 6, 2020; 7 a.m. – 3:30 p.m. PT

Session: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: Poster III
Abstract #3258
Title: Preclinical Evaluation of ALLO-605, an Allogeneic BCMA TurboCAR TTM Cell Therapy for the Treatment of Multiple Myeloma
Presenter: Cesar Sommer, Ph.D., Allogene Therapeutics
Session Date & Time: Monday, December 7, 2020; 7 a.m. – 3 p.m. PT

On November 4, 2020 PRA Health Sciences, Inc. ("PRA," "we," "us" or the "Company") (NASDAQ: PRAH) reported financial results for the quarter ended September 30, 2020 (Press release, PRA Health Sciences, NOV 4, 2020, View Source [SID1234569973]).

"I am very appreciative of the effort and focus of all of our employees despite the difficult health circumstances in the world today. As a result of this commitment, I am very pleased to report revenue and earnings that were significantly better than the guidance we provided in August and an increase to our 2020 guidance. I am also happy to report another quarter of record level gross and net new business awards" said Colin Shannon, PRA’s President and Chief Executive Officer. "Although we continue to experience challenges with the pandemic, we believe we will finish 2020 strong and are well-positioned for 2021."

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Net new business for our Clinical Research segment for the three months ended September 30, 2020 excluding reimbursement revenue was $737.9 million, representing a net book-to-bill ratio of 1.28 for the period. Net new business for our Clinical Research segment for the three months ended September 30, 2020 including reimbursement revenue was $986.0 million, representing a net book-to-bill of 1.35 for the period. Consistent with the prior quarters, we did not experience any material COVID-19 related cancellations during the third quarter. Net new business, excluding reimbursement revenue, contributed to an ending backlog at September 30, 2020 of $5.1 billion, an increase of 11.0% year over year and 3.7% sequentially.

For the three months ended September 30, 2020, revenue was $796.3 million, which represents an increase of 2.0%, or $15.6 million, compared to the three months ended September 30, 2019 at actual foreign exchange rates. On a constant currency basis, revenue increased $10.3 million, an increase of 1.3% compared to the third quarter of 2019. By segment, the Clinical Research segment generated revenues of $732.1 million, while the Data Solutions segment generated revenues of $64.2 million. The increase in revenue for the quarter was attributable to increased billable hours offset by a decrease in the reimbursable portion of revenue, which was impacted by the continued disruption from the COVID-19 pandemic.
Direct costs, exclusive of depreciation and amortization, were $412.1 million during the three months ended September 30, 2020 compared to $389.3 million for the three months ended September 30, 2019 at actual foreign exchange rates. On a constant currency basis, direct costs increased $21.4 million compared to the third quarter of 2019. The increase in direct costs continues to be driven by increased labor costs in our Clinical Research segment and increased data costs in our Data Solutions segment. Direct costs were 51.7% of revenue during the third quarter of 2020 compared to 49.9% of revenue during the third quarter of 2019.

Selling, general and administrative expenses were $115.4 million during the three months ended September 30, 2020 compared to $95.5 million for the three months ended September 30, 2019. Selling, general and administrative costs were 14.5% of revenue during the third quarter of 2020 compared to 12.2% of revenue during the third quarter of 2019.

GAAP net income was $91.3 million for the three months ended September 30, 2020, or $1.41 per share on a diluted basis, compared to GAAP net income of $83.0 million for the three months ended September 30, 2019, or $1.25 per share on a diluted basis.

EBITDA was $148.3 million for the three months ended September 30, 2020, representing an increase of 15.3% compared to the three months ended September 30, 2019. Adjusted EBITDA was $132.3 million for the three months ended September 30, 2020, representing a decrease of 4.0% compared to the three months ended September 30, 2019.

Adjusted net income was $84.1 million for the three months ended September 30, 2020, representing a decrease of 3.6% compared to the three months ended September 30, 2019. Adjusted net income per diluted share was $1.30 for the three months ended September 30, 2020, representing a decrease of 1.5% compared to the three months ended September 30, 2019.
Nine Months Ended September 30, 2020 Financial Highlights

For the nine months ended September 30, 2020, revenue was $2,309.9 million, which represents an increase of 1.9%, or $43.9 million, compared to the nine months ended September 30, 2019 at actual foreign exchange rates. On a constant currency basis, revenue increased $48.5 million, representing an increase of 2.1% compared to the nine months ended September 30, 2019.

Reported GAAP income from operations was $231.0 million, reported GAAP net income was $145.8 million and reported GAAP net income per diluted share was $2.26 for the nine months ended September 30, 2020.

Adjusted Net Income was $206.7 million for the nine months ended September 30, 2020, a decrease of 14.7% compared to the same period in 2019. Adjusted Net Income per diluted share was $3.20 for the nine months ended September 30, 2020, down 12.1% compared to the same period in 2019.
Guidance

The full extent of the COVID-19 pandemic and its impact on our fourth quarter operations continues to remain uncertain. Specifically, the duration of the pandemic, the geographic location of specific outbreaks, and the length and scope of travel restrictions and business closures imposed by the governments of impacted countries could impact our financial results in the fourth quarter of 2020. These uncertainties could impact the assumptions used in the Company’s 2020 guidance if they result in business interruptions, such as the closure of our Phase I clinics, a slowdown in recruitment activities, or limited access to sites worldwide. However, the Company has used its best efforts to estimate the impact of COVID-19 on its business and the resulting impact on financial performance for the remainder of the year.

For full year 2020, the Company expects to achieve total revenues between $3.120 billion and $3.150 billion, GAAP net income per diluted share between $3.23 and $3.35 and adjusted net income per diluted share between $4.61 and $4.71, with an expected effective income tax rate of 23%.

Our actual effective tax rate may differ from our estimate due to, among other things, changes in the geographic allocation of our pre-tax income as well as changes in interpretations, analysis, and additional guidance that may be issued by regulatory agencies.

Our guidance assumes a EURO rate of 1.15 and a GBP rate of 1.30. All other foreign currency exchange rates are as of September 30, 2020.

A reconciliation of our non-GAAP measures, EBITDA, adjusted EBITDA, adjusted net income, adjusted net income per diluted share and our full year guidance to the corresponding GAAP measures is included in this press release.

Conference Call Details

PRA will host a conference call at 9:00 a.m. ET on November 5, 2020, to discuss the contents of this release and other relevant topics. To participate, please dial (877) 930-8062 within the United States or (253) 336-7647 outside the United States approximately 10 minutes before the scheduled start of the call. The conference ID for the call is 6797898. The conference call will also be accessible, live via audio broadcast, on the Investor Relations section of the PRA website at investor.prahs.com. A replay of the conference call will be available online at investor.prahs.com. In addition, an audio replay of the call will be available for one week following the call and can be accessed by dialing (855) 859-2056 within the United States or (404) 537-3406 outside the United States. The replay ID is 6797898.

Additional Information

A financial supplement with third quarter 2020 results, which should be read in conjunction with this press release, may be found in the Investor Relations section of our website at investor.prahs.com in a document titled "Q3 2020 Earnings Presentation."

On November 4, 2020 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported financial results for the quarter ended September 30, 2020 (Press release, Allogene, NOV 4, 2020, View Source [SID1234569999]).

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"We continue to see rapid advancement across our pipeline of AlloCAR T candidates and look forward to a number of firsts for Allogene over the coming months, starting with the first look at data from the Phase 1 UNIVERSAL trial of ALLO-715 for the treatment of multiple myeloma. The firsts will continue with the submission of our IND in solid tumors to evaluate ALLO-316 in renal cell carcinoma by the end of the year as well as an IND to evaluate ALLO-715 in combination with nirogacestat," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "In the spirit of leading today and creating tomorrow as we advance allogeneic cell therapies for patients, we look forward to 2021 where we anticipate having five clinical trials underway, including our first pivotal trial."

Pipeline Highlights

Anti-BCMA AlloCAR T Program
The Company continues to execute on a three-pronged anti-BCMA strategy led by ALLO-715, a Phase 1 candidate for the treatment of relapsed/refractory multiple myeloma (MM).

ALLO-715 UNIVERSAL Phase 1 Trial
Initial data from the ALLO-715 Phase 1 UNIVERSAL trial will be presented at a medical meeting in Q4 2020. The UNIVERSAL trial utilizes ALLO-647 (anti-CD52 mAb) together with the standard fludarabine and cyclophosphamide as the lymphodepletion regimen. The initial dose escalation portion of the UNIVERSAL trial studied ALLO-715 dosed at 40M, 120M and 320M cells following lymphodepletion with 39mg (low dose) of ALLO-647. Fifteen patients were treated in the study at the time of abstract submission.
The virtual presentation will include data on approximately 20 patients evaluable for efficacy across ALLO-715 cell dose cohorts and lower dose (39mg) of ALLO-647, as well as patients evaluable for efficacy who were treated with higher doses of ALLO-715 and higher doses of ALLO-647. The Phase 1 UNIVERSAL study continues to enroll patients at these higher doses in an effort to optimize the therapy.
This study is enrolling patients with relapsed/refractory MM who have been treated with at least three prior lines of therapy, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 antibody (unless contraindicated), and refractory to the last treatment line.
ALLO-715 + nirogacestat
The Company is on track to submit an IND application by the end of 2020 to evaluate ALLO-715 in combination with SpringWorks’ investigational gamma secretase inhibitor, nirogacestat, in patients with relapsed/refractory MM.
ALLO-605 (anti-BCMA TurboCAR)
An IND is expected to be submitted in the first half of 2021 for the Company’s first TurboCAR candidate, ALLO-605, an investigational BCMA-directed AlloCAR T therapy for MM. TurboCAR technology allows cytokine activation signaling to be engineered selectively into CAR T cells. TurboCAR has the potential to improve efficacy, overcome cell exhaustion, and reduce dosing requirements of AlloCAR T therapy.
Anti-CD19 AlloCAR T Program

The Company continues to leverage data from the Phase 1 ALPHA trial of ALLO-501 to inform and optimize trial design and dose selection of ALLO-501A and ALLO-647, a differentiated lymphodepleting agent.
In the first half of 2021, additional data from the Phase 1 ALPHA study of ALLO-501 in relapsed/refractory non-Hodgkin lymphoma (NHL) and initial data from the Phase 1 dose escalation ALPHA2 study of ALLO-501A are planned for presentation. Data from these trials are expected to support trial design for a potentially pivotal Phase 2 trial planned for initiation in 2021.
Solid Tumor AlloCAR T Program

ALLO-316 (anti-CD70)
The Company expects to submit an IND for ALLO-316, its first AlloCAR T candidate for the treatment of solid tumors, by the end of 2020. A study in renal cell carcinoma is expected to initiate in 2021. ALLO-316 targets CD70 and also has potential application in hematologic malignancies.
Corporate Highlights

Manufacturing
Construction continues on the Company’s state-of-the-art cGMP cell manufacturing facility in Newark, California with cGMP manufacturing from this facility expected in 2021.
Business Development
The Company and The University of Texas MD Anderson Cancer Center announced a strategic five-year collaboration aimed at accelerating the development of a broad portfolio of AlloCAR T candidates across hematologic and solid tumors. Under the agreement, MD Anderson and the Company will collaborate on the design and conduct of preclinical and clinical studies with oversight from a joint steering committee.
Third Quarter Financial Results

Research and development expenses were $51.4 million for the third quarter of 2020, which includes $8.8 million of non-cash stock-based compensation expense.
General and administrative expenses were $16.6 million for the third quarter of 2020, which includes $9.0 million of non-cash stock-based compensation expense.
Net loss for the third quarter of 2020 was $66.2 million, or $0.52 per share, including non-cash stock-based compensation expense of $17.8 million.
The Company had $1.0 billion in cash, cash equivalents, and investments as of September 30, 2020.
2020 Financial Guidance

Allogene continues to expect full year GAAP net losses to be between $260 million and $280 million including estimated non-cash stock-based compensation expense of $70 million to $75 million and excluding any impact from potential business development activities.
Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 5:30 a.m. Pacific Time / 8:30 a.m. Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 2637139. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days