On May 17, 2021 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, reported financial results and recent corporate highlights for the first quarter ended March 31, 2021 (Press release, Fortress Biotech, MAY 17, 2021, View Source [SID1234580122]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and our partner companies had an exciting start to the year, including the addition and commercial launch of two dermatology products, bringing our total number of marketed products to seven. Moreover, we continued to achieve significant milestones in the advancement of multiple key development programs. Notably, in February, our partner company, Cyprium Therapeutics ("Cyprium"), and Sentynl Therapeutics ("Sentynl"), a wholly owned subsidiary of the Zydus Group, signed a Development and Asset Purchase Agreement for CUTX-101 for the treatment of Menkes disease. This agreement, which included an $8 million upfront payment for the ongoing development of CUTX-101, in addition to regulatory and sales milestone payments plus royalties, allows us to potentially maximize the value of this important asset as Cyprium continues to advance CUTX-101 toward a rolling submission of a New Drug Application ("NDA") later this year."

Dr. Rosenwald continued, "Our portfolio continues to grow with more than 25 product candidates across our partner companies, including 17 clinical programs, of which four are pivotal programs. We expect to have a multitude of regulatory and clinical inflection points throughout the remainder of 2021, including the availability of clinical data from cosibelimab, CAEL-101 and MB-106. Importantly, our diversified business model is supported by a strong balance sheet, as we ended the first quarter with $291.5 million in consolidated cash, cash equivalents and restricted cash. Our operational catalysts and financial strength have us well-positioned for success and we remain focused on creating long-term shareholder value."

Recent Corporate Highlights1:

Marketed Dermatology Products

Our seven dermatology products are marketed by our partner company, Journey Medical Corporation ("Journey").
Our products generated net revenues of $10.7 million for the first quarter of 2021, compared to first quarter 2020 net revenues of $11.9 million. While product demand increased in the first quarter of 2021 compared to the first quarter of 2020, the decrease in net revenue in the first quarter of 2021 is primarily attributable to increased coupon expense costs related to standard insurance deductible resets. We expect year-over-year annual revenue growth in 2021 to exceed the 28% growth Journey achieved in 2020.
In April 2021, Journey acquired and recently launched its seventh prescription dermatology product, QBREXZA.
On April 1, 2021, Journey entered into an agreement with East West Bank ("EWB") in which EWB will provide a $7.5 million working capital line of credit.
On March 31, 2021, Journey completed its first close in connection with its Cumulative Convertible Class A Preferred Stock Offering. In connection with the first close, Journey issued an aggregate of 501,480 Journey Preferred A shares at a price of $25.00 per share, and after deducting commissions, fees, and expenses, for a total of $11.2 million in net proceeds.
We plan on launching one additional prescription product in the second half of this year.
CUTX-101 (Copper Histidinate for Menkes disease)

In February 2021, our partner company, Cyprium, and Sentynl signed a Development and Asset Purchase Agreement for CUTX-101 for the treatment of Menkes disease. Under the terms of the agreement, Cyprium received $8 million upfront to fund the development of CUTX-101 and could receive up to $12 million in regulatory milestone payments through NDA approval, and is eligible to receive sales milestones plus royalties. Royalties start from mid-single digits, scaling up to 25% on sales exceeding $100 million annually. Cyprium will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101. Cyprium is responsible for the development of CUTX-101 through approval of the NDA by the FDA, and Sentynl will be responsible for commercialization of CUTX-101, as well as progressing newborn screening activities.
We intend to begin the rolling submission of the NDA for CUTX-101 to the FDA in the second half of 2021.
CUTX-101 is currently in development at our partner company, Cyprium Therapeutics, Inc.
CAEL-101 (Light Chain Fibril-reactive Monoclonal Antibody for AL Amyloidosis)

Caelum Biosciences, Inc. ("Caelum") has two on-going Phase 3 studies of CAEL-101 for AL amyloidosis.
Caelum formed a collaboration with Alexion Pharmaceuticals, Inc. in 2019, which includes an option to acquire Caelum. AstraZeneca announced the execution of a definitive agreement to purchase Alexion Pharmaceuticals, Inc. In the event of the closing of such transaction, the timeline for a potential exercise of the option to purchase Caelum will be accelerated to six months following the date of acquisition closing.
In May 2021, we announced that CAEL-101 clinical data were selected for two presentations at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress ("EHA2021") taking place in June. The abstracts can be viewed online through the EHA (Free EHA Whitepaper)2021 website: here and here.
CAEL-101 is currently in development at Caelum Biosciences, Inc., a company founded by Fortress in 2017 and in which Fortress maintains a minority position.
Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

The registration-enabling study in metastatic cutaneous squamous cell carcinoma is fully enrolled and we are on track to report top-line results by year-end. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, we believe cosibelimab has the potential to be a market disruptive product in the $25 billion and growing PD-(L)1 class.
A Phase 3 registration-enabling trial is planned to begin in first-line metastatic non-small cell lung cancer in mid-2021.
Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. ("Checkpoint").
MB-107 and MB-207 (Lentiviral Gene Therapies for X-linked Severe Combined Immunodeficiency)

In February 2021, we announced encouraging MB-107 and MB-207 clinical updates from our investigator-IND X-linked severe combined immunodeficiency ("XSCID") trials, as well as additional consistent safety and efficacy data. On January 28, 2021, the FDA removed a CMC hold on the MB-107 Phase 2 clinical trial Investigational New Drug ("IND") application after reviewing a comprehensive CMC package that was submitted in late December 2020. We expect to enroll the first patient in this pivotal multicenter trial in the second quarter of 2021 and we are targeting top-line data from the trial in the second half of 2022. We also expect to file an IND in the second quarter of 2021 for our pivotal multicenter Phase 2 clinical trial of MB-207.
MB-107 and MB-207 are currently in development at our partner company, Mustang Bio, Inc. ("Mustang Bio").
MB-106 (CD20-targeted CAR T Cell Therapy)

In May 2021, we announced that the FDA approved Mustang Bio’s IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted CAR T for relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL").
Also in May 2021, we announced that CD20-targeted CAR T data were selected for presentation at EHA (Free EHA Whitepaper)2021 scheduled to take place in June. Dr. Mazyar Shadman of Fred Hutchinson Cancer Research Center will present updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2021 website here.
MB-106 is currently in development at our partner company, Mustang Bio.
Dotinurad (Urate Transporter (URAT1) Inhibitor)

In May 2021, we announced an exclusive license agreement with Fuji Yakuhin Co. Ltd. to develop Dotinurad in North America and Europe. Dotinurad is a potential best-in-class urate transporter (URAT1) inhibitor for gout and possibly other hyperuricemic indications including chronic kidney disease and heart failure. Dotinurad (URECE tablet) was approved in Japan in 2020 as a once-daily oral therapy for gout and hyperuricemia. Dotinurad was efficacious and well-tolerated in more than 500 Japanese patients treated for up to 58 weeks in Phase 3 clinical trials.
Dotinurad is currently in development at our partner company, FBIO Acquisition Corp. VIII.
IV Tramadol

In October 2020, Avenue Therapeutics ("Avenue") announced that it had received a Complete Response Letter ("CRL") from the FDA regarding Avenue’s NDA for IV tramadol. The FDA held a Type A meeting with Avenue in November 2020 to discuss the issues outlined in the CRL. On February 12, 2021, Avenue resubmitted its NDA to the FDA for IV tramadol. The NDA resubmission followed the receipt of the official minutes from Avenue’s Type A meeting with the FDA. The NDA resubmission included revised language relating to the proposed product label and a report relating to terminal sterilization validation. On February 26, 2021, Avenue received an acknowledgment letter from the FDA stating that Avenue’s resubmission of its NDA is a complete, class 1 response to the CRL, and a Prescription Drug User Free Act ("PDUFA") goal date was set for April 12, 2021. On April 13, 2021, Avenue announced that the FDA was still reviewing its NDA for IV tramadol and had not provided a decision regarding the NDA. As of May 1, 2021, Avenue had not received approval from the FDA for IV tramadol. Accordingly, under the Stock Purchase and Merger Agreement ("SPMA"), InvaGen retains an option to consummate the second stage closing until October 31, 2021 (after which Avenue can choose to terminate the SPMA), and also retains the option to terminate the SPMA.
IV tramadol is currently in development at our partner company, Avenue.
General Corporate

In February 2021, Fortress partner company, Aevitas Therapeutics, appointed Markus Peters, Ph.D., M.Sc., as Chief Executive Officer.
Financial Results:

To assist our stockholders in understanding our company, we have prepared non-GAAP financial results for the three months ended March 31, 2021 and 2020. These results exclude the operations of our three public partner companies: Avenue, Checkpoint, and Mustang Bio. The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which are comprised of our commercial-stage business, our privately held development-stage entities, as well as our business development and finance functions.

As of March 31, 2021, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $291.5 million, compared to $235.0 million as of December 31, 2020, an increase of $56.5 million during the quarter.
On a GAAP basis, Fortress’ net revenue totaled $11.6 million for the first quarter of 2021, which included $10.7 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $12.9 million for the first quarter of 2020, which included $11.9 million in net revenue generated from our marketed dermatology products.
On a GAAP basis, consolidated research and development expenses including license acquisitions were $20.2 million for the first quarter of 2021, compared to $15.1 million for the first quarter of 2020. On a non-GAAP basis, research and development expenses including license acquisitions were $4.1 million for the first quarter of 2021, compared to $2.3 million for first quarter of 2020.
On a GAAP basis, consolidated selling, general and administrative expenses were $17.5 million for the first quarter of 2021, compared to $15.5 million for the first quarter of 2020. On a non-GAAP basis, consolidated selling, general and administrative expenses were $13.0 million, of which $6.2 million is attributed to Journey, for the first quarter of 2021, compared to $11.6 million, of which $5.6 million is attributed to Journey, for the first quarter of 2020.
On a GAAP basis, consolidated net loss attributable to common stockholders was $8.8 million, or $0.11 per share, for the first quarter of 2021, compared to consolidated net loss attributable to common stockholders of $12.4 million, or $0.19 per share for the first quarter of 2020.
Fortress’ non-GAAP loss attributable to common stockholders was $7.3 million, or $0.09 per share, for the first quarter of 2021, compared to Fortress’ non-GAAP loss attributable to common stockholders of $4.2 million, or $0.07 per share, for the first quarter of 2020.

1. Includes Research and development expense and Research and development – licenses acquired expense for the quarter ended March 31, 2021 and 2020, respectively.
2. Excludes $0.1 million for Fortress MSA for the quarter ended March 31, 2021; excludes $0.1 million for Fortress MSA for the quarter ended March 31, 2020.
3. Excludes $0.1 million of Fortress MSA expense and $0.6 million Fortress financing fee for the quarter ended March 31, 2021; and $0.1 million of Fortress MSA expense for the quarter ended March 31, 2020.

4. Excludes $0.1 million of Fortress MSA expense and $1.2 million Fortress financing fee for the quarter ended March 31, 2021; and $0.1 million of Fortress MSA expense and $0.1 million Fortress financing fee for the quarter ended March 31, 2020.

On May 17, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported the appointment of industry veteran Salim Yazji, M.D., as Chief Medical Officer (Press release, Oncternal Therapeutics, MAY 17, 2021, View Source [SID1234580146]).

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"Salim brings a wealth of relevant experience to Oncternal during this exciting time as we begin to advance our pipeline toward the registrational phase," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We believe that his extensive background in oncology drug development and strategy, experience with global regulatory authorities, and proven leadership ability make him an ideal fit for Oncternal as we advance our lead clinical product candidate, cirmtuzumab, along with our ROR1 targeted CAR-T program and clinical TK216 product candidate and make key strategic decisions about next steps."

Dr. Yazji stated, "I am excited to be joining Oncternal as the Company’s novel pipeline matures and the organization pivots towards key decisions in its clinical programs in both hematological and solid tumors. I look forward to working with the focused team at Oncternal to advance regulatory strategy and execute upon the Company’s current and future clinical development programs."

Dr. Yazji has held positions of increasing responsibility in both large and entrepreneurial organizations. He founded Elpida Therapeutics in January 2019 and co-founded Ajuta Therapeutics in October 2019, where he served as Chief Executive Officer until February 2021. He has also served on the Board of Directors of Versatope Therapeutics since April 2019. From March 2018 to January 2019, he served as Chief Medical Officer of PMV Pharma, and from November 2016 to February 2018, he served as Executive Vice President and Chief Medical Officer of Calimmune, which was acquired by CSL Behring in August 2017. Prior to that, he served as Vice President & Global Head of Oncology at Baxter International from 2013 to 2015 and its spinoff Baxalta from 2015 until it was acquired by Shire Plc in July 2016. From 2009 to 2013, he held global positions of increasing responsibility within Novartis where he led multiple oncology registrational clinical trials, most recently as Senior Global Clinical Leader. Prior to 2009, he held positions with Exelixis, PDL BioPharma, and Johnson & Johnson.

Dr. Yazji obtained his MD from the Pavlov School of Medicine, University of St. Petersburg, St. Petersburg, Russia, and completed his post-graduate training at the University of Texas M.D. Anderson Cancer Center, Park Plaza Hospital, Houston and the Almozov Hospital, St. Petersburg, Russia.

Equity Inducement Grant

On May 17, 2021, Oncternal granted Dr. Yazji options to purchase a total of 425,000 shares of Oncternal common stock, including options to purchase 70,600 shares of Oncternal common stock granted as an inducement award under Oncternal’s 2021 Employment Inducement Incentive Award Plan, which provides for the granting of equity awards to new employees of Oncternal as an inducement to join the Company. The options have a 10-year term and an exercise price equal to the closing price of Oncternal’s common stock on May 17, 2021. The options vest over a four-year period, with 25% of the options vesting on the first anniversary of Dr. Yazji’s employment start date, and the rest vesting in equal monthly installments over three years thereafter. The inducement award was approved by Oncternal’s compensation committee, comprised entirely of independent directors, as required by Nasdaq Rule 5635(c)(4), and was granted as an inducement material to Dr. Yazji entering into employment with Oncternal in accordance with Nasdaq Rule 5635(c)(4).

On May 17, 2021 Kiromic reported the submission of a novel Investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA) for a Phase 1 clinical trial that has the potential to be a universal T-Cell therapy for any solid malignancy that expresses the biomarker PD-L1, with higher efficacy, higher safety, as well as lower manufacturing and distribution costs (Press release, Kiromic, MAY 17, 2021, View Source;Gamma-Delta-T-cell-Therapy [SID1234580162]).

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The clinical trial title: ALEXIS-PRO-1: A Phase 1, Open-label, Dose Escalation Study of KB-PD1, an Allogeneic Gamma Delta T-Cell Expressing a Chimeric PD1 Switch Receptor, in Subjects with PD‑L1 Positive Metastatic or Progressive Locally Advanced Solid Malignancies.

The FDA feedback is expected within 30 days from this IND submission.

Kiromic’s preclinical studies show rapid and complete tumor elimination with no toxicity as per results presented at the American Association Cancer Research 2021 poster LB148.

Unlike in the case of autologous CAR-T cell therapy products, which are derived from pretreated cancer patients, Kiromic’s proprietary PD1 Gamma-delta switch receptor therapy is derived from healthy donors.

Key features of the IND:

Product Name

KB-PD1

Product Type

Chimeric PD1 T-Cell live cell therapy

Cancer Type

All solid tumors

Targeting

Chimeric PD-L1

Patient Type

Patients with solid tumor positive for PD‑L1

Projected No. of Patients

30

Dosing

Dose escalation

Primary Completion of Trial

18 months

First-in-human dosing

3Q 2021 pending FDA authorization

First data from Trial

4Q 2021

How Our KB-PD1 Live T-Cell Therapy Improves CAR-T Market:

Marketed and traditional CAR-T

Kiromic KB-PD1

Malignancies
(Cancer Type)

Hematologic

Solid Tumors

Live Cell Origin

Autologous
Live Cells from pre-treatment patients

Allogeneic
Live Cells from healthy donors

Live Cell Cloning
(Manufacturing)

Single batch
Single dose

Single batch
Multi dose
(aka. Off-The-Shelf)

Mass Manufacturing
on-demand
a single patient

— Will be manufactured like a traditional drug
— Kiromic proprietary manufacturing
— Kiromic proprietary cryopreservation processing techniques

Kiromic was able to respect the expectations and meet the timeline 45 days before the end of the second quarter. Kiromic now expects that it will be able to deliver a first in human patient dosing by the end of Q3 2021. Kiromic, an organization that is driven to achieve scientific breakthroughs, dedicated significant resources to the IND submission in an effort to achieve the optimal clinical trial design.

Historically, checkpoint inhibitors have been successful in solid malignancies, but KB-PD1 takes it one step further by not just blocking the PD1/PD-L1 interaction ("cutting the brakes") like a checkpoint inhibitor does, but rather, KB-PD1 rewires the brake into an accelerator.

In this way, Kiromic is optimistic that any cancer cell that expresses the PD-L1 marker will effectively activate and accelerate the ability of KB-PD1 to traffic through the tumor microenvironment (TME), which thus far has proven to be an imposing barrier to effective T-cell treatments in solid cancers.

IQVIA (CRO, Clinical Research Organization)

View Source

IQVIA is an industry driver in data technology and advanced analytics, designed to produce and develop optimal clinical trial outcomes.

IQVIA will be managing our clinical trail sites CRO (Clinical Research Organization).

Leading global sites are lining up to be the first to dose our KB-PD1 live cell therapies for solid tumors.

Site announcements are coming within weeks.

CEO of Kiromic, Dr. Maurizio Chiriva-Internati, DBSc, PhDs

"I’m thrilled to see our technology go into the clinic, and it was due to the dedication of our team, and our strong preclinical results which helped propel Kiromic to be able to file this IND submission to the FDA on May 14, 2021," stated Dr. Maurizio Chiriva-Internati, DBSc, PhDs, CEO of Kiromic.

"Our people and vendors have been working tirelessly. They are excited and ready to launch the first-in-human dosing by 3Q 2021 pending FDA authorization.

Our KB-PD1 will be the first in the world to deliver this T-cell therapy to solid tumors with key features demanded by the T-cell therapy market. We aim to deliver KB-PD1 which will have:

— Higher efficacy

— Higher safety

— Lower manufacturing costs

— Lower distribution costs."

CFO of Kiromic, Mr. Tony Tontat

"This IND filing is an awaited news for the markets and will open up advantageous financing options to the company as we look to extend cash runway into 2022."

CSIO of Kiromic, Mr. Gianluca Rotino

"This IND filing is supported by strong IP portfolio including new IP in areas such as:

— Gene editing

— Live cell harvesting

— Live cell expansion (manufacturing clones from small batches)

— Live cell logistics (manufacturing, tissue preservation, and transfers)

As we continue to grow our DIAMOND derived targets and our related clinical programs, our IP portfolio is continually being fortified in all major geographies, and we look forward to updating our investors in upcoming presentations and filings.

This is a key milestone, not only for Kiromic, positioning Kiromic as a pioneer in T-Cell therapy for solid tumors, but also for immunotherapy in general.

With the support of Kiromic’s innovations in AI, the clinical trial R&D process will now be faster bringing cellular therapies to patients with more expediency than previously achievable."

CMO of Kiromic, Dr. Scott Dalhbeck

"Patients and clinicians alike have been waiting for a T-Cell therapy which could deliver a safe, effective, and lower cost solution for solid tumors. We are on the brink of making this vision a reality when we dose the first solid tumor patients."

About Metastatic Solid Malignancies

The ALEXIS-PRO-1 clinical trial will enroll patients with metastatic or progressive locally advanced solid malignancies that express the PD-L1 marker on their tumor.

These late-stage patients typically have a survival of only a few months, and so effective treatments that are non-toxic and rapidly effective, are desperately needed.

Since most solid malignancies express PD-L1, the dose escalation phase of the trial will be open to a wide range of tumor types such as prostate, breast, pancreas, lung, colon, renal, bladder, ovarian, and others.

Once the optimal biologic dose (OBD) has been determined, the OBD will then be given to an expanded number of patients for the most promising indications.

Since Kiromic’s preclinical animal studies have shown a fast and brisk anti-tumor response, expectations are high that this product will be able to achieve what has thus far proven to be elusive with late-stage cancer patients – a significant and long-lasting improvement in the quality and quantity of life.

On May 17, 2021 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, reported the receipt of the $50 million second tranche under its convertible credit facility with Deerfield Partners, L.P. and certain of its affiliates (collectively, Deerfield) (Press release, ADC Therapeutics, MAY 17, 2021, View Source [SID1234580107]). Under the terms of the Facility Agreement dated April 24, 2020, Deerfield agreed to provide to the Company up to $115 million in financing consisting of two separate tranches. The first $65 million was received upon completion of the Company’s initial public offering in May 2020, and the second tranche of $50 million has now been received following the recent U.S. Food and Drug Administration (FDA) accelerated approval of ZYNLONTA (loncastuximab tesirine-lpyl).

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"Following the recent approval of ZYNLONTA, we are pleased to further strengthen our balance sheet with the $50 million second tranche draw down," said Chris Martin, Chief Executive Officer of ADC Therapeutics. "These funds will support the continued execution of our launch and advancement of our deep pipeline of next-generation ADCs."

Pursuant to a related Registration Rights Agreement with Deerfield, the Company will file a registration statement on Form F-3 with the U.S. Securities and Exchange Commission within 15 days. In the event that in the future Deerfield elects to exercise its conversion option with respect to the convertible notes issued under the Facility Agreement, this registration statement will allow Deerfield to sell the resulting shares. ADC Therapeutics will not sell any securities under this registration statement.

On May 17, 2021 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results and provided a business update for the quarter ended March 31, 2021 (Press release, Galectin Therapeutics, MAY 17, 2021, View Source [SID1234580123]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Joel Lewis, Chief Executive Officer and President of Galectin Therapeutics, said, "The start to the new year has been an exciting and productive time for Galectin Therapeutics in both our NAVIGATE Phase 2b/3 clinical trial in NASH cirrhosis as well as the cancer, combination immunotherapy trial being conducted in collaboration with the Providence Cancer Institute in Portland, Oregon. In addition, since the beginning of the year, we have raised fresh capital and brought on Dr. Ben Carson, Sr., a world-renowned physician and former Secretary of Housing and Urban Development, as a special consultant. We very much look forward to working with Dr. Carson.

"In support of our innovative NAVIGATE study," continued Lewis, "we launched NAVIGATEnash.com, a new information resource about NASH cirrhosis for both patients and physicians. NASH cirrhosis is still largely overlooked and remains a major unmet medical need. I’ve been deeply moved by the determination of this community of NASH cirrhosis patients and the physicians who treat them.

"In our cancer, combination immunotherapy trial, results of the Phase 1 trial were published in the highest ranked fully open access peer-reviewed immunology journal. The study suggests combination therapy with belapectin results in a better objective response rate with fewer adverse events than pembrolizumab (KEYTRUDA) alone. Brendan Curti, M.D., the first author of the paper, noted galectin-3 is an important driver of tumor-induced immunosuppression. This clinical study constitutes proof-of-concept that the addition of our galectin-3 inhibitor, belapectin, to a PD-1 inhibitor can benefit cancer patients.

"Of course," concluded Lewis, "I would be remiss if I did not thank our Chairman, Mr. Richard Uihlein, who continues to demonstrate his strong support and provided additional funding to help advance our NAVIGATE trial, and for which we are deeply grateful."

Richard E. Uihlein, Chairman of the Board, added, "This quarter has been an important one for Galectin Therapeutics, and having Dr. Ben Carson on our team is an investment in the future. The progress being made in both our NASH cirrhosis study and our collaboration on cancer immunotherapy demonstrates the important science behind what we are doing. Galectin-3 is involved in a wide range of human diseases, and my recent investment in the company underscores my commitment to develop belapectin to its full potential."

Dr. Ben Carson, Sr., Engaged as Special Consultant

Engaged Ben Carson, Sr., M.D., a world-renowned neurosurgeon and the 17th Secretary of the U.S. Department of Housing and Urban Development, as a special consultant to assist with development of the Company’s galectin-3 inhibitor, belapectin, as a treatment for NASH cirrhosis and in combination with immunotherapy for the treatment of cancers. In particular, Dr. Carson will help increase awareness of Galectin Therapeutics including our ongoing Phase 2b/3 NAVIGATE clinical trial in NASH cirrhosis, our continuing research in combination with cancer immunotherapy, and its potential in addressing other fibrotic diseases. Dr. Carson will also assist in the formation of a scientific advisory committee for the Company, recruit potential members of the committee, and identify potential strategic commercial and/or academic partners for the Company.
Recent Financings

Raised $10 million in a convertible debt financing with Richard E. Uihlein, the Company’s Chairman and largest individual stockholder. The debt is unsecured and bears interest at a rate of 2% compounded annually. Additional interest of 2.5% per quarter will accrue but will only be paid if the debt and interest are converted into shares of the Company’s common stock, at Mr. Uihlein’s option, on or prior to maturity, which is four years from the date of the loan. The conversion price of the debt and interest is fixed at $5.00 per share of common stock.
NAVIGATE Trial Update

Launched NAVIGATEnash.com, a trial website dedicated to educating patients and physicians about liver cirrhosis resulting from non-alcoholic steatohepatitis (NASH) as well as support NAVIGATE, the Company’s innovative, seamless adaptive Phase 2b/3 study in NASH cirrhosis.

Galectin Therapeutics continues to actively recruit patients into NAVIGATE, its seamless, adaptive Phase 2b/3 study of belapectin for the prevention of esophageal varices in NASH cirrhosis.
Peer-reviewed publications, Scientific Presentations and Conferences

Published in the Journal for ImmunoTherapy of Cancer (JITC), the highest ranked fully open access immunology journal, a peer-reviewed paper entitled, "Enhancing Clinical and Immunological Effects of anti-PD-1 with Belapectin, a Galectin-3 Inhibitor" (doi:10.1136/jitc-2021-002371), provided further clinical evidence that using belapectin, a potent galectin-3 inhibitor, in combination with pembrolizumab (KEYTRUDA), a PD-1 inhibitor, significantly enhances tumor response to immunotherapy in patients with advanced metastatic melanoma (MM) and head and neck squamous cell carcinoma (HNSCC). The paper describes results from an ongoing Phase 1 clinical study, a collaboration between Galectin Therapeutics and Providence Cancer Institute in Portland, Oregon. An objective response was observed in 50% of MM (7/14) and 33% of HNSCC (2/6) patients. This compares favorably to published response rates on pembrolizumab alone.

At the 4th Global NASH Congress, which took place virtually on April 29, 2021, Pol F. Boudes, M.D., Chief Medical Officer, reviewed Galectin Therapeutics’ scientific and clinical activities in NASH cirrhosis in a presentation entitled, "An innovative clinical development program: belapectin for NASH cirrhosis."
Financial Results

For the three months ended March 31, 2021, the Company reported a net loss applicable to common stockholders of $6.3 million, or ($0.11) per share, compared to a net loss applicable to common stockholders of $3.6 million, or ($0.06) per share for the three months ended March 31, 2020. The increase is largely due an increase in 2021 research and development expenses related to the Company’s NAVIGATE trial.

Research and development expense for the three months ended March 31, 2021, was $4.9 million compared with $2.1 million for the three months ended March 31, 2020. The increase was primarily due to costs related to our NAVIGATE clinical trial and other supportive activities. General and administrative expenses for the three months ended March 31, 2021, were $1.4 million, compared to $1.4 million for the three months ended March 31, 2020.

As of March 31, 2021, the Company had $20.8 million of cash and cash equivalents. On April 16, 2021, the Company received $10,000,000 in proceeds from an unsecured convertible promissory note from its Board Chairman, Richard E. Uihlein. The Company also has a $10 million unsecured line of credit, under which no borrowings have been made to date. The Company believes it has sufficient cash, including availability under the line of credit, to fund currently planned operations and research and development activities through at least September 30, 2022.

The Company expects that it will require more cash to fund operations after September 30, 2022 and believes it will be able to obtain additional financing as needed. Currently, we expect to require an additional approximately $35-$40 million to cover costs of the trial to reach the planned interim analysis estimated to occur in the second half of 2023 along with drug manufacturing and other scientific support activities and general and administrative costs. However, there can be no assurance that we will be successful in obtaining such new financing or, if available, that such financing will be on terms favorable to us.

About Belapectin (GR-MD-02)

Belapectin (GR-MD-02) is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis. A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis, and these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (NAVIGATEnash.com), entitled "A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis" began enrolling patients in June, 2020, and is posted on www.clinicaltrials.gov (NCT04365868). Galectin-3 also has a significant role in cancer, and the Company is supporting a Phase 1 study in combined immunotherapy of belapectin and KEYTRUDA in treatment of advanced melanoma and in head and neck cancer.

About Fatty Liver Disease with Advanced Fibrosis and Cirrhosis

Non-alcoholic steatohepatitis (NASH) has become a common disease of the liver with the rise in obesity and other metabolic diseases. NASH is estimated to affect up to 28 million people in the U.S. It is characterized by the presence of excess fat in the liver along with inflammation and hepatocyte damage (ballooning) in people who consume little or no alcohol. Over time, patients with NASH can develop excessive fibrosis, or scarring of the liver, and ultimately liver cirrhosis. It is estimated that as many as 1 to 2 million individuals in the U.S. will develop cirrhosis as a result of NASH, for which liver transplantation is the only curative treatment available. Approximately 8,890 liver transplants are performed annually in the U.S. There are no drug therapies approved for the treatment of liver fibrosis or cirrhosis.