On January 9, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) ("Infinity") reported a $20 million non-dilutive asset-backed financing with BVF Partners L.P. ("BVF"), Infinity’s largest shareholder (Press release, Infinity Pharmaceuticals, JAN 9, 2020, View Source [SID1234552924]). This investment by BVF entails no equity to be issued by Infinity and has its sole recourse in potential royalty payments due on future sales of patidegib, a hedgehog pathway inhibitor discovered by Infinity and licensed to PellePharm in 2013. Infinity has the right to repay the $20 million plus interest to repurchase the right to future patidegib royalties during the next three years under certain conditions.

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"This non-dilutive financing is representative of our very collaborative relationship with BVF and their tremendous support as a value-added investor. Leveraging our financial interest in patidegib royalties enables us to preserve significant upside for all of Infinity’s shareholders," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Importantly, with this financing, we now have over $60 million cash on hand to fund all of our current IPI-549 trials to key data readouts throughout 2020 and into 2H 2021. These data readouts include studies of IPI-549 in a randomized, controlled Phase 2 study in bladder cancer and in front line settings with novel triple therapy combinations, including in indications for which we have seen clinical activity, as part of a thoughtful clinical development strategy designed to reveal the potentially transformative impact of reprogramming macrophages with IPI-549."

Mark Lampert, President of BVF, Inc., commented, "In light of our longstanding association with Infinity, the enormous potential of IPI-549 to help cancer patients, which is not currently reflected in the company’s stock price, and our admiration for management’s dilution sensitivity in advance of data, we wanted to help Infinity raise capital without equity dilution. BVF’s large existing ownership stake in the company was fundamental in aligning our interests with the company to preserve IPI-549 upside for all shareholders, and we believe this innovative financing structure accomplishes the objective in a win-win manner."

Within this extended cash runway into 2H 2021, Infinity expects to generate data on approximately 525 patients from the following trials:

MARIO-275, our global randomized Phase 2 study in collaboration with Bristol-Myers Squibb (BMS), evaluating IPI-549 in combination with Opdivo in patients with advanced urothelial cancer.
MARIO-3, our Phase 2 study in collaboration with Roche/Genentech evaluating IPI-549 in combination with Tecentriq and Abraxane as a front-line treatment in patients with triple negative breast cancer (TNBC) and in combination with Tecentriq and Avastin as a front-line treatment for patients with renal cell cancer (RCC).
MARIO-1, our Phase 1/1b study in collaboration with BMS evaluating IPI-549 in combination with Opdivo in patients with advanced solid tumors.
Arcus Biosciences’ Phase 1 collaboration study evaluating IPI-549 in a novel, checkpoint inhibitor free regimen that includes their dual adenosine receptor inhibitor, AB928, and Doxil in patients with relapsed/refractory TNBC.
In addition to the initial $20 million payment, Infinity is eligible to receive from BVF an additional $5 million payment upon positive data from PellePharm’s Phase 3 trial in patients with Gorlin Syndrome. PellePharm announced the completion of enrollment in a Phase 3 trial of a topical formulation of patidegib in patients with Gorlin Syndrome in December 2019. FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for a topical formulation of patidegib in patients with Gorlin Syndrome in November 2017. Infinity retains rights to all patidegib milestone payments from PellePharm of up to $9 million in regulatory and first commercial sale milestones and $37.5 million in sales threshold milestones.

Infinity has the option to repurchase the rights to future patidegib royalties by paying BVF an amount equal to the principal amount received by Infinity plus interest at any time when the 20-day volume weighted average price per share of Infinity’s common stock exceeds $5.00 during the next three years.

Furthermore, Infinity retains its approximately 1% equity interest in PellePharm. PellePharm has previously announced that LEO Pharmaceuticals has the right to acquire PellePharm following Phase 3 data for total potential consideration of $690 million.

If, during the period ending three years from the date of the agreement or earlier in the event Infinity has exercised its repurchase option for future patidegib royalties from BVF, Infinity completes future equity financings above a specified share quantity threshold and below a specified price threshold, then Infinity has agreed to provide BVF with 50% warrant coverage at a 50% premium to the price at which such shares in excess of the share quantity threshold were sold.

The terms and conditions of the transaction are described in more detail in a Form 8-K filed by Infinity with the Securities and Exchange Commission on January 9, 2020.

On January 9, 2020 OncoCell MDx, a privately held immunogenomics company with a patented liquid biopsy platform, reported it has changed its name to Immunis.AI (Press release, OncoCell MDx, JAN 9, 2020, View Source [SID1234552940]). The name was chosen to better reflect the power and breadth of application of the company’s novel Intelligentia platform, which assesses and stratifies risk of disease on an individualized basis, earlier in the process, at the cellular level. Intelligentia combines the power of the immune defense system, RNAseq technology and machine learning for the development of proprietary disease-specific algorithms to detect and grade disease with a simple blood test. The company will present as Immunis.AI at the 2020 Biotech Showcase, being held January 13-15, 2020 in San Francisco, California.

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Date: Tuesday, January 14, 2020

Time: 10:30 AM

Track: Franciscan D (Ballroom Level)

Venue: Hilton San Francisco Union Square, 333 O’Farrell Street, San Francisco, CA 94102

"Our name change reflects the expanded potential of the technology as well as a new stage in the company’s growth and development," said Mark McDonough, President and CEO of Immunis.AI. "Our Intelligentia platform is unique in leveraging the immune system’s surveillance apparatus to overcome the limitations of cfDNA and circulating tumor cells to improve early stage disease detection, at the point of immune-escape, when there is the best opportunity for cure. We’re continuing to develop the platform in multiple cancer types, where there is a significant opportunity to change the diagnostic paradigms, and we’re now also focusing development in other needed disease states. We’re looking forward to sharing details of our expanded platform and focus areas at Biotech Showcase next week."

Over the past year, Immunis.AI has built out a new leadership team, convening top executives in life sciences with years of experience leading companies towards commercialization and acquisition. In September 2019, the company announced a series B funding of $22.2 million to help advance their novel platform towards commercialization. The company’s novel technologies were created at Harvard University by co-founder, Professor Amin I. Kassis, in 2012. Through 2019, the company advanced its internal discovery studies, intellectual property portfolio, and patient databases. In 2020, the company plans to launch its first noninvasive diagnostic test for early prostate cancer detection, and to further advance development of tests in other diseases.

To schedule a meeting with Mark McDonough during the conference, or for additional details, please contact [email protected].

On January 9, 2020 Danaher Corporation (NYSE: DHR) reported that President and Chief Executive Officer, Thomas P. Joyce, Jr., will be presenting at the J.P. Morgan Healthcare Conference in San Francisco, California on Tuesday, January 14, 2020 at 8:00 a.m. PT (Press release, Danaher, JAN 9, 2020, View Source [SID1234552957]). The audio will be simultaneously webcast on and the presentation will be archived on www.danaher.com.

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On January 9, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported that key objectives and anticipated milestones for 2020 and provided an overview of recent progress (Press release, Synlogic, JAN 9, 2020, View Source [SID1234552906]).

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"In 2020 we look forward to advancing our platform and our growing development stage pipeline of Synthetic BioticTM medicines – advancing our phenylketonuria program and additional oral metabolic programs that build on the critical capabilities we have established in development and manufacturing, as well as advancing our first clinical program in oncology," said Aoife Brennan, M.B. Ch.B., Synlogic’s president and chief executive officer. "We believe that Synthetic Biotic medicines have potential across multiple diseases and conditions. Our expanded collaboration with Ginkgo Bioworks, strong balance-sheet and solid momentum out of our discovery and development efforts position us well to execute on our near-term clinical plans."

2020 Goals and Milestones
Clinical Programs

SYNB1618: SYNB1618 is an orally delivered, Synthetic Biotic medicine designed to consume phenylalanine (Phe) in the gastrointestinal (GI) tract for the treatment of phenylketonuria (PKU) in patients regardless of age or disease type.
In the first half of 2020, Synlogic expects to initiate a Phase 2 clinical trial to evaluate the Phe-lowering potential of its solid formulation of SYNB1618 in patients with PKU. In addition, the study is expected to provide valuable information to validate predictive mathematical and preclinical modeling.
Strain development and optimization work is being carried out under Synlogic’s 2019 research and development agreement with Ginkgo Bioworks.
SYNB1891: Synlogic’s first clinical immuno-oncology (IO) program, SYNB1891, is an intratumorally delivered Synthetic Biotic medicine designed to produce a STING agonist and act as a dual innate immune activator for the treatment of refractory solid tumors and lymphoma.
SYNB1891 is being evaluated as a monotherapy in a Phase 1 open-label, multicenter, dose escalation clinical trial (NCT04167137) in patients with refractory solid tumors and lymphoma. Three U.S. sites have been activated to enroll and the first subject has been dosed. Synlogic expects to have data from the monotherapy arm of this study in 2020.
After establishing a maximum tolerated dose (MTD) for SYNB1891, Synlogic will initiate a second arm of the trial in which subjects will receive escalating dose levels of SYNB1891 in combination with a fixed dose of the checkpoint inhibitor, atezolizumab (Tecentriq), to establish a recommended dose for the combination regimen.
Pre-clinical data and early pipeline programs:
Synlogic has advanced two new preclinical programs onto its development pipeline, for the treatment of secondary hyperoxaluria and MSUD, respectively. Strain development and optimization work is being carried out under Synlogic’s 2019 research and development agreement with Ginkgo Bioworks.
Synlogic expects to publish and present data at major scientific and medical meetings throughout the year demonstrating the breadth and potential of its Synthetic Biotic platform.
Corporate

Synlogic ended the third quarter of 2019 with $138.7 million in cash, cash equivalents and short- and long-term investments and expects that this will fund Company operations through 2021 under its current plan.
The Company will continue to explore additional strategic opportunities to maximize the potential of its Synthetic Biotic platform.
2019 Accomplishments and Highlights
Clinical Pipeline

SYNB1618
Presentation of positive data from Phase 1/2a study of SYNB1618 in patients with phenylketonuria (PKU) using the liquid formulation as well as modeling work to estimate target dosing of SYNB1618. SYNB1618 was well tolerated in healthy volunteers and patients with PKU and the data demonstrated equivalent SYNB1618-dependent Phe-consumption from the GI tract in both populations.
Development of a solid oral formulation of SYNB1618 that is stable at room temperature. Synlogic executed a bridging study in healthy volunteers of a new solid oral formulation to identify an MTD that could be used to evaluate lowering of blood Phe levels in PKU patients based on Synlogic’s modeling data. Data from this study will be presented at an appropriate medical meeting in 2020. Synlogic expects to advance SYNB1618 into a Phase 2 study in patients in the first half of 2020.
SYNB1891:
Initiation of a Phase 1 open-label, multicenter, dose escalation trial of its STING-agonist producing bacterial strain, SYNB1891, for the treatment of refractory solid tumors and lymphoma.
Corporate

Establishment of a collaboration with Ginkgo Bioworks that provides expanded synthetic biology capabilities and strengthened Synlogic’s balance sheet. In June 2019, Synlogic and Ginkgo entered into a long-term strategic platform collaboration. Under the agreement Ginkgo invested $80.0 million in Synlogic at a premium to market. Synlogic is using Ginkgo’s cell programming platform for building and testing microbial strains to accelerate progression of early preclinical leads to drug candidates optimized for further clinical development. Under the terms of the agreement, Synlogic paid $30.0 million to Ginkgo for synthetic biology services and has exclusive rights to any Synthetic Biotic medicines that it develops as part of the collaboration and to intellectual property covering such products.
Strengthened Synlogic’s executive leadership with the following appointments:
Richard Riese, M.D. Ph.D., joined Synlogic in September 2019 as chief medical officer assuming responsibilities for all clinical and regulatory functions from current president and CEO, Aoife Brennan. Prior to joining Synlogic, he served as Vice President, Clinical Development at Alnylam Pharmaceuticals where he led clinical development projects in several areas across Alnylam’s rare disease portfolio.
Gregg Beloff was appointed as Synlogic’s interim chief financial officer, in October 2019. Mr. Beloff has more than 20 years of experience in the life sciences industry and brings significant expertise in operational management, strategic planning, corporate and business development, fundraising and mergers and acquisitions.
Patricia Hurter, Ph.D., chief executive officer of Lyndra Therapeutics, was appointed to the Synlogic board of directors in January 2019. Dr. Hurter previously served as Senior Vice President at Vertex. Prior to joining Vertex, Dr. Hurter was Director, Formulation Design and Characterization for Merck.
Established a clinical collaboration with Roche that enables evaluation of SYNB1891, in combination with atezolizumab in the second arm of Synlogic’s ongoing Phase 1 study in patients with advanced solid tumors and lymphoma.
Established GMP manufacturing capabilities and manufactured solid and liquid formulations of clinical trial material for its oral and intra-tumoral programs, respectively.
Advanced collaboration with AbbVie to develop Synthetic Biotic-based treatments for inflammatory bowel disease resulting in payment to Synlogic of a $2.5 million milestone in March 2019.
Published first in human clinical data of a Synthetic Biotic medicine (SYNB1020) as well as preclinical data from the program in Science Translational Medicine.

On January 9, 2020 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported an update on recent progress and the Company’s 2020 priorities and expected milestones (Press release, Intellia Therapeutics, JAN 9, 2020, View Source [SID1234552925]).

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"2020 will be a significant year for Intellia, as we execute on our full-spectrum strategy. With milestones anticipated across our pipeline, we are making important progress towards the development of curative treatments for severe diseases. In particular, we expect to dose ATTR patients with the first-ever systemically delivered CRISPR/Cas9-based therapy this year, and we are beginning IND-enabling activities for our newly announced development candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy, for treatment of AML," said Intellia President and Chief Executive Officer, John Leonard, M.D. "We are focused on developing a robust platform with modular genome editing capabilities that enable a fast and reproducible path to development. Today’s update reflects this strategy, and it also features the announcement of our third development program, an in vivo knockout approach for HAE. Importantly, this program leverages the infrastructure and insights from NTLA-2001 and underscores our ability to produce a rapid succession of new clinical candidates. We are excited by the strong momentum across our diverse pipeline and look forward to providing updates on our development programs in the upcoming year."

Program Updates and Anticipated 2020 Milestones:

ATTR Program: Intellia remains on track to submit an investigational new drug (IND) application in mid-2020 for its lead in vivo candidate, NTLA-2001, for treatment of

transthyretin amyloidosis (ATTR). NTLA-2001 is anticipated to be the first systemically delivered CRISPR/Cas9 therapy to enter the clinic, and Intellia anticipates dosing the first patients in the second half of 2020. In addition, Intellia completed a 12-month durability study of its lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, maintaining an average reduction of >95% of serum transthyretin (TTR) protein and sustained liver genome editing after a single dose in non-human primates (NHPs). NTLA-2001 is part of a co-development/co-promotion (Co/Co) agreement between Intellia, which is the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron). Intellia and Regeneron have a 75% and 25% share of worldwide development costs and profits, respectively.

AML Program: Intellia reported NTLA-5001 as its first engineered T cell therapy development candidate, utilizing its T cell receptor (TCR)-directed approach to target the Wilms’ Tumor 1 (WT1) intracellular antigen for the treatment of acute myeloid leukemia (AML). Intellia’s WT1-TCR-directed approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patient’s leukemia. This approach employs CRISPR/Cas9 to efficiently knock out and replace the endogenous TCR with a natural, high affinity therapeutic TCR. The resulting cells are capable of specific and potent killing of AML blasts, and have no detectable bone marrow cell toxicity. The Company expects to present preclinical data in support of NTLA-5001 at an upcoming scientific meeting in the first quarter of 2020 and plans to submit an IND application in the first half of 2021. Additional efforts are underway to evaluate the potential use of the WT1-TCR construct to treat other tumor types, including solid tumors.

HAE Program: Today, Intellia announced that the Company is committed to developing a CRISPR/Cas9-based therapy for hereditary angioedema (HAE) as its third development program. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of bradykinin, a protein which leads to swelling. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin. Using its modular LNP-based CRISPR/Cas9 delivery system, Intellia aims to knock out the kallikrein B1 (KLKB1) gene, which is part of a biological pathway that results in release of bradykinin. Knocking out this gene should reduce the undesired bradykinin activity in HAE patients. The Company plans to present preclinical data at an upcoming scientific meeting in the first quarter of 2020. In addition, Intellia is evaluating several potential guide RNAs and expects to nominate a development candidate in the first half of 2020. Intellia’s KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.

Cash Position and Financial Guidance:

Intellia ended the fourth quarter of 2019 with approximately $284.5 million in cash, cash equivalents and marketable securities. Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2019 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements at least through the end of 2021. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.