On August 9, 2021 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP),reported that corporate updates for the second quarter of 2021, ended June 30, 2021 (Press release, Ziopharm, AUG 9, 2021, View Source [SID1234586111]). The Company will host a conference call and webcast today at 4:30 pm EDT.

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"Since our last update, the Company has made tremendous progress across multiple fronts," said Heidi Hagen, Interim Chief Executive Officer. "The Company has built significant positive momentum in our TCR-T Library clinical trial enabling activities and in our efforts to strengthen the balance sheet. This quarterly update reflects the Company doing exactly what we said we would do earlier in the year and this is very exciting to see. The appointment of a permanent CEO is also something we look forward to in the very near future. The strong science underlying our work continues to provide us with confidence as we move forward. On behalf of the Board and management team, I want to thank all our dedicated researchers, scientists, partners, and the entire organization for their commitment to our mission."

Significant Progress in Company’s TCR-T Library Clinical Program

Since the last quarterly update, the Company achieved several key milestones in its TCR-T Program. Importantly, the Company remains on track to begin dosing patients in its Phase I/II TCR-T Library trial during the second half of 2021, and now anticipates dosing the first patient during the fourth quarter of this year. The trial initially targets six individual solid tumor indications: cholangiocarcinoma, pancreatic cancer, ovarian cancer, endometrial cancer, colorectal cancer, and lung cancer, which were selected due to the frequency of KRAS and / or TP53 mutations. The Company intends on expanding into additional indications in the future.

The clinical trial will open for enrollment upon establishing clinical manufacturing readiness. During 2020, the Company successfully transferred the manufacturing process for its TCR-T cell products to KBI Biopharma, a previously undisclosed contract manufacturing organization with cGMP cell therapy manufacturing facilities in The Woodlands, TX. TCR-T batch data generated at both KBI and the Company’s own laboratory were the basis of the Chemistry, Manufacturing and Controls portion of the Investigational New Drug Application (IND) filed earlier this year. KBI is now working to complete the process qualification and aseptic process validation to facilitate clinical manufacturing.

Additionally, the Company has been implementing a strategy to build in-house cGMP clinical production capabilities at the Company’s facility in Houston, TX. This is being done to provide greater flexibility and control of this important aspect of clinical development and the Company is moving forward rapidly to establish these manufacturing capabilities. The commissioning of the Company’s clinical production unit (CPU), as well as aseptic process validation, were completed this past quarter, which are meaningful steps in establishing the CPU’s capabilities. The team is completing process qualification, which will support the opening of the facility to manufacture TCR-T cells for the clinical trial. The ability to use both its in-house facility and KBI’s facility for manufacturing will provide the Company a strong degree of risk mitigation and greater capacity as the Company scales up clinical trial activities.

The Company continues to qualify TCRs in its Library and plans to amend the IND during the second half of 2021 to include these additional TCRs. The Company expects that the supplemental TCRs will expand the potential utility, applicable patient population, and addressable commercial market for the Library, and may include additional KRAS and / or TP53 mutations or other genetic hotspots associated with solid tumors such as EGFR.

During the second quarter, the Company presented a poster at the annual American Association of Cancer Research (AACR) (Free AACR Whitepaper) meeting, entitled "Hotspot mutations in KRAS targeted by TCR-T cells genetically modified with the Sleeping Beauty transposon/transposase system". The poster highlighted preclinical work regarding the Company’s TCR-T program and demonstrated that multiple TCRs with unique specificities targeting recurrent p53 and KRAS substitutions in frequent HLA haplotypes could be stably expressed using Sleeping Beauty transposition to re-direct peripheral blood T-cells towards tumor cells. The Company plans on providing additional preclinical data further demonstrating the strong science behind the program later this year at a scientific conference.

"Our TCR-T program continues to build momentum scientifically and clinically, and we remain poised to begin dosing during the fourth quarter of this year. We have begun pre-screening patients and based on initial data from investigators, we are very optimistic that we will consistently find mutation / HLA combinations within the targeted patient populations that match TCRs in our Library," said Raffaele Baffa, MD, Ph.D., Chief Medical Officer. "I thank the team for their tremendous efforts as they work to ensure success."

Closing of Venture Debt Financing with Silicon Valley Bank

The Company reported that it has closed a venture debt facility with Silicon Valley Bank in the aggregate amount of $50 million. The Company will draw down an initial $25 million tranche from this facility immediately. A second $25 million tranche is available contingent on the achievement of certain clinical milestones and other conditions.

The initial $25 million tranche extends the Company’s cash runway into the fourth quarter of 2022, well beyond the time required to generate and assess the initial clinical data from the Company’s Phase I/II TCR-T Library trial.

Holger Weis, member of the Board of Directors and Chair of the Audit Committee, commented, "We have developed a very strong relationship with Silicon Valley Bank and are happy to have its partnership and support on this funding. This debt vehicle will be used in a prudent and judicious manner and extends the Company’s cash runway significantly. This is a clear demonstration that the Company is being guided by strong consideration of shareholder impact and a desire to support expanding shareholder value through our capital strategy and tactics."

Update on Phase I CD19 RPM CAR-T Trial Being Conducted by Eden BioCell in Taiwan

As previously disclosed, in March 2021, Eden BioCell, the Company’s Joint Venture in Taiwan with TriArm Therapeutics, began treating patients in a clinical trial with the Company’s investigational CD19 RPM CAR-T cell therapy, under the Phase I IND cleared by the Taiwan Food and Drug Administration in December 2020. The Company today provided an update on this program.

Two patients have been treated in the trial. The lead investigator at National Taiwan University in Taipei has reported no serious adverse safety events in either of these patients. Laboratory results continue to support, as previously published, that non-viral Sleeping Beauty gene transfer is effective in genetically modifying autologous T-cells. Patients were infused two days after gene transfer, thus shortening the turnaround time and providing a clear advantage over viral methods.

However, based on laboratory data generated from the first two patients between March and May 2021, the TriArm/Eden BioCell team concluded, in concert with the lead investigator and with the support from the team at Ziopharm, that further process development work is required. This additional work is intended to optimize and refine the manufacturing process in order to manufacture cells more consistently in the desired clinical dose range seeking to be studied.

Per the terms of the Joint Venture agreement, the TriArm/Eden BioCell team will work towards the necessary process development improvements before infusing additional patients. The length of time to do so is unknown and maybe require up to 12 months. The ongoing COVID-19 outbreak in Taiwan presents added uncertainty, as the operational activities in the manufacturing facility are currently limited due to employee restrictions related to the pandemic. These restrictions are impacting clinical trials broadly in Taiwan.

Additionally, consistent with its strategic focus on TCR, the Company is seeking and considering broader partnerships to enable further development of the investigational CD19 RPM CAR-T cell therapy. Several parties have expressed interest in such a partnership, including TriArm Therapeutics. The Company will carefully consider all options regarding the future of the Joint Venture, the technology, and the global development pathway, in order to maximize shareholder value.

The Company noted the distinctions between the CAR-T program and the TCR-T program. "As we have previously described, the TCR and CAR-T processes are intrinsically different and follow very separate process development pathways," commented Dr. Baffa. "While both involve Sleeping Beauty gene transfer, the constructs, and manufacturing processes are very distinct. The unique clinical presentations and challenges associated with the treatment of blood cancers and solid tumors also reflect differences and we believe the recent findings from CAR-T do not read through to the TCR-T program."

Update on Search for Permanent Chief Executive Officer

The Company expects to make an announcement regarding its permanent CEO position in the near future.

Robert Postma, member of the Board of Directors, said, "We have seen and considered a number of very strong and qualified candidates during the search, and the Board is in the final stages of selecting our new CEO."

Cash Position

As of June 30, 2021, the Company had approximately $76.7 million of cash and cash equivalents. This amount is unaudited and preliminary, and does not present all information necessary for an understanding of our financial condition as of June 30, 2021, which will be presented in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021.

Additionally, a prepayment of approximately $1.8 million remains for work to be conducted by the Company at MD Anderson under the Company’s research and development agreements.

The $25 million drawdown of the Silicon Valley Bank debt facility is not included in the above figure as it was closed subsequent to June 30, 2021.
Conference Call and Webcast

Ziopharm will host a conference call and webcast for the investment community today, August 9, 2021, at 4:30 p.m. EDT. The conference call can be accessed by dialing 877-451-6152 (U.S. and Canada) or 201-389-0879 (International). The passcode for the conference call is 13721210. A live webcast may be accessed using the link here, or by visiting the "Investors" section of the Ziopharm website at www.ziopharm.com. The call will be recorded and available for replay on the Company’s website for approximately 90 days after the call.

On August 9, 2021 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported second quarter and first half 2021 financial results (Press release, Precigen, AUG 9, 2021, View Source [SID1234586110]).

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"We have made significant progress in the first half of 2021 and are well on our way to meet or exceed the goals we set at the beginning of the year. We are excited about the advancement of our portfolio and look forward to providing further clinical updates and data readouts at a planned R&D call on November 4th as well as at medical congresses in the fourth quarter of the year," said Helen Sabzevari, PhD, President and CEO of Precigen. "We see 2021 as a pivotal year for the UltraCAR-T, ActoBiotics and AdenoVerse platforms with significant new clinical data on our most advanced therapeutic candidates from these core therapeutic platforms."

Business Highlights:

R&D Update Call

Precigen will host an R&D call on November 4th that will be dedicated to reviewing progress made in advancing the Company’s clinical pipeline, including the latest data for several of our key programs.
PRGN-3005 UltraCAR-T

PRGN-3005 UltraCAR-T is a first-in-class investigational therapy under evaluation in a Phase 1/1b clinical trial for the treatment of patients with advanced, recurrent platinum resistant ovarian cancer. Study subjects receive the PRGN-3005 infusion either via intraperitoneal (IP) (Arm A) or intravenous (IV) (Arm B) infusion.
Preliminary Phase 1 data previously reported from the two lowest dose levels of the IP arm showed a favorable safety profile with no dose-limiting toxicities (DLTs), neurotoxicity or cytokine release syndromes (CRS); encouraging expansion and persistence without lymphodepletion; and clinical activity as evidenced by regression in total target tumor burden.
The dose escalation phase of both the IP and IV arms of the trial is ongoing concurrently. Enrollment in dose level 4 of the IP arm and dose level 3 of the IV arm is ongoing.
The Company anticipates the presentation of interim data from the Phase 1 trial in the fourth quarter of 2021.
PRGN-3006 UltraCAR-T

PRGN-3006 UltraCAR-T is a first-in-class investigational therapy under evaluation in a Phase 1/1b clinical trial for the treatment of patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS). Study subjects receive the PRGN-3006 infusion either without prior lymphodepletion (Cohort 1) or following lymphodepleting chemotherapy (Cohort 2). PRGN-3006 UltraCAR-T has been granted Orphan Drug Designation in patients with AML by the US FDA.
Preliminary Phase 1 data previously reported for the two lowest dose levels in Cohort 1 and the lowest dose level in Cohort 2 showed a favorable safety profile with no DLTs or neurotoxicity; encouraging expansion and persistence of PRGN-3006 UltraCAR-T in both cohorts; and clinical activity as evidenced by reduction in AML tumor blast levels. One of the patients­ treated with PRGN-3006 at the lowest dose level with lymphodepletion (Cohort 1), with approximately nine million UltraCAR-T cells, achieved complete remission with incomplete hematologic recovery (CRi) per European Leukemia Net (ELN) criteria and subsequently received allogeneic hematopoietic stem cell transplant (HSCT).
The dose escalation phase of both the lymphodepletion and non-lymphodepletion cohorts of the Phase 1 trial is ongoing concurrently. Enrollment in dose level 4 of the non-lymphodepletion cohort and dose level 3 of the lymphodepletion cohort is ongoing.
The Company anticipates the presentation of interim data from the Phase 1 trial in the fourth quarter of 2021.
PRGN-2009 AdenoVerse Immunotherapy

PRGN-2009 is a first-in-class, off-the-shelf (OTS) investigational immunotherapy utilizing the AdenoVerse platform that has been designed to activate the immune system to recognize and target HPV-positive solid tumors. PRGN-2009 is currently under evaluation in a Phase 1/2 clinical trial as a monotherapy or in combination with bintrafusp alfa (M7824) in patients with HPV-associated cancers. The trial is being conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI).
Preliminary Phase 1 data previously reported for the monotherapy arm of the Phase 1 trial showed that the treatment was well-tolerated with no DLTs. Furthermore, preliminary correlative analysis from the patients treated at the lowest dose in the monotherapy arm showed an increase in HPV-specific T-cell response in 100% (3 of 3) of patients and an increase in the magnitude of immune response with repeated PRGN-2009 administrations.
As previously announced, enrollment in the Phase 1 monotherapy dose escalation arm is complete, with all patients (n=6) receiving multiple doses of PRGN-2009, as many as thirteen doses to date. Subsequently, the monotherapy arm of the Phase 2 trial, which evaluates PRGN-2009 as neoadjuvant therapy for newly diagnosed oropharyngeal or sinonasal squamous cell cancer patients as a first line treatment was initiated. Enrollment in the Phase 2 monotherapy arm is ongoing with four patients enrolled to date. Enrollment in the Phase 1 combination arm is also ongoing with six patients enrolled to date.
A trial-in-progress update on the PRGN-2009 study was provided at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 annual meeting as a poster presentation by Charalampos S. Floudas, MD, DMSc, MS, Assistant Research Physician, Genitourinary Malignancies Branch at the Center for Cancer Research at the NCI.
The Company anticipates the presentation of interim Phase 1 data in the fourth quarter of 2021.
PRGN-2012 AdenoVerse Immunotherapy

PRGN-2012 is a first-in-class, investigational OTS AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with HPV 6 or HPV 11 for treatment of recurrent respiratory papillomatosis (RRP). A Phase 1 clinical trial of PRGN-2012 in adult patients with RRP is ongoing. The Phase 1 trial is designed to follow 3+3 dose escalation of PRGN-2012 as an adjuvant immunotherapy following standard-of-care surgical removal of visible papillomas. Patients receive up to four injections of PRGN-2012. The study is designed to enroll 3 to 6 subjects at each dose level followed by an expansion cohort with 12 patients treated at the maximum tolerated dose. The trial is being conducted under a CRADA with the NCI. PRGN-2012 has been granted Orphan Drug Designation in patients with RRP by the US FDA.
In January 2021,the Company announced US FDA clearance of the IND to initiate the PRGN-2012 Phase 1 trial.
In March 2021, the first patient was dosed and, subsequently, enrollment was completed in the 3+3 dose escalation portion of the Phase 1 trial. Enrollment then was initiated and the first patient dosed in the expansion cohort of the Phase 1 study at the maximum study dose, exceeding the Company’s goal this year.
AG019 ActoBiotics

AG019 ActoBiotics is a first-in-class, orally administered, investigational therapy designed to address the underlying cause of Type 1 diabetes (T1D) and is currently under evaluation in a Phase 1b/2a clinical trial for the treatment of early-onset T1D. The study is assessing safety and tolerability of AG019 administered as monotherapy or in combination with teplizumab.
Enrollment and dosing is complete in the Phase 1b and Phase 2a portions of the study.
Positive topline data from the AG019 Phase 1b/2a clinical trial were reported at the Federation of Clinical Immunology Societies (FOCIS) 2021 Virtual Annual Meeting by Kevan Herold, MD, CNH Long Professor of Immunobiology and of Medicine (Endocrinology) at the Yale School of Medicine, meeting the Company’s goal to present AG019 Phase 1b/2a data this year. The primary endpoints of safety and tolerability for the Phase 1b AG019 monotherapy and the Phase 2a AG019 combination therapy were met. No serious adverse events (SAEs) were reported. AG019, as a monotherapy and in combination with teplizumab, showed stabilization or increase of C-peptide levels, a biomarker for T1D disease progression, and induced antigen-specific tolerance in conjunction with the reduction of disease-specific T cell responses. Results indicated the potential of the oral AG019 monotherapy to preserve insulin production in recent-onset T1D through its capacity to reduce autoreactive T cells and increase the frequency of memory Tregs to induce antigen-specific immune modulation.
Additional data from the AG019 Phase 1b/2a clinical trial will be presented on October 1, 2021 at 12:00 PM CET as an oral presentation at the European Association for the Study of Diabetes (EASD) 57th Annual Meeting. The abstract entitled, "AG019 ActoBiotics as monotherapy or in association with teplizumab in recent-onset type 1 diabetes was safe and demonstrated encouraging metabolic and immunological effects" will be presented by Chantal Mathieu, MD, PhD, Professor of Medicine at the Katholieke Universiteit Leuven, Belgium.
A clinical trial assessing the efficacy of prolonged treatment of oral AG019 is planned.
Second Quarter and First Half 2021 Financial Highlights

Net cash used in operating activities of $24.2 million during the six months ended June 30, 2021 compared to $41.5 million during the six months ended June 30, 2020;
Cash, cash equivalents, short-term and long-term investments totaled $200.4 million as of June 30, 2021; and
Total revenues of $33.6 million and $58.1 million during the three and six months ended June 30, 2021, respectively, compared to $30.4 million and $60.3 million during the three and six months ended June 30, 2020, respectively.
Second Quarter 2021 Financial Results Compared to Prior Year Period
Research and development expenses increased $4.2 million, or 44%, over the quarter ended June 30, 2020. Contract research organization costs and lab supplies increased $3.8 million with the advancement of the Company’s clinical and preclinical programs. Selling, general and administrative ("SG&A") expenses increased $2.1 million, or 12%. The majority of the SG&A increase was due to an increase in professional fees. Net loss from continuing operations was $20.1 million, or $(0.10) per basic share, of which $8.2 million was for non-cash charges in 2021 compared to net loss from continuing operations of $15.7 million, or $(0.10) per basic share, of which $8.7 million was for non-cash charges in 2020.

Total revenues increased $3.2 million, or 10%, over the quarter ended June 30, 2020. Collaboration and licensing revenues decreased $4.0 million primarily due to a decrease in the recognition of previously deferred revenue in the current period resulting from fewer services being performed pursuant to the Company’s collaboration agreements. Product and service revenues generated by Trans Ova and Exemplar increased $7.2 million primarily due to higher customer demand for Trans Ova’s products and services as a result of stronger beef and dairy industries in the current year and a change in pricing structure with certain customers, as well as increased services provided by Exemplar to new and existing customers. Gross margin on products and services improved as a result of the increased revenues, the change in pricing structure for certain customers, and operational efficiencies that have been gained through reductions in workforce and improved inventory management.

First Half 2021 Financial Results Compared to Prior Year Period
Research and development expenses increased $3.4 million, or 16%, over the six months ended June 30, 2020. Contract research organization costs and lab supplies increased $3.8 million with the advancement of the Company’s clinical and preclinical programs. SG&A expenses were comparable period over period due to offsetting changes. Salaries, benefits, and other personnel costs decreased $1.6 million in 2021 primarily due to a reduced headcount as the Company scaled down its corporate functions to support a more streamlined organization and reduced stock compensation costs for previously granted awards that became fully vested in early 2021. These decreases were partially offset by an increase in professional fees. Net loss from continuing operations was $41.9 million, or $(0.21) per basic share, of which $17.9 million was for non-cash charges in 2021 compared to net loss from continuing operations of $36.6 million, or $(0.23) per basic share, of which $16.4 million was for non-cash charges in 2020.

Total revenues decreased $2.2 million, or 4%, from the six months ended June 30, 2020. Collaboration and licensing revenues decreased $14.7 million as the Company accelerated the recognition of previously deferred revenue in the prior period upon the mutual termination of one of its collaboration agreements in February 2020. Product and service revenues generated by Trans Ova and Exemplar increased $12.6 million primarily due to higher customer demand for Trans Ova’s products and services as a result of stronger beef and dairy industries in the current year and a change in pricing structure with certain customers, as well as increased services provided by Exemplar to new and existing customers. Gross margin on products and services improved as a result of the increased revenues, the change in pricing structure for certain customers, and operational efficiencies that have been gained through reductions in workforce and improved inventory management.

On August 9, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that it is offering to sell $200 million of its common stock and, in lieu of common stock, to offer and sell to certain investors pre-funded warrants to purchase shares of its common stock in an underwritten public offering. As part of this offering, BioCryst intends to grant the underwriters a 30-day option to purchase additional shares of common stock in an amount equal to up to 15% of the securities offered in the public offering. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

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J.P. Morgan, Evercore ISI and Piper Sandler are acting as joint book-running managers for the offering.

All of the shares to be sold in the offering are being sold by BioCryst, with the proceeds to be used for general corporate purposes, which may include, but are not limited to, worldwide development, manufacturing, regulatory and commercial activities for ORLADEYO; advancement of the worldwide development, manufacturing, regulatory and clinical activities for BCX9930 for complement-mediated diseases; discovery, manufacturing, development and clinical activities for other pipeline molecules in rare diseases; and capital expenditures and other general working capital needs.

This offering is being made pursuant to an effective shelf registration statement on Form S-3 filed by BioCryst with the U.S. Securities and Exchange Commission (SEC) and only by means of a prospectus supplement and related prospectus. A preliminary prospectus supplement relating to the offering will be filed with the SEC and will be available on its website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying prospectus may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by email at [email protected], or by telephone at (866) 803-9204; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, by telephone at (888) 474-0200, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected].

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, these securities, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale is not permitted.

On August 9, 2021 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) for the treatment of eosinophil and mast cell-related diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Allakos, AUG 9, 2021, View Source [SID1234586108]).

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Recent Accomplishments

Presented new data at Digestive Disease Week (DDW) 2021 suggesting eosinophilic gastritis and/or eosinophilic duodenitis (EG/EoD) is significantly underdiagnosed and may be a common cause of moderate-to-severe gastrointestinal symptoms. Forty-five percent (45%, 181/405) of patients with chronic functional gastrointestinal symptoms who underwent upper endoscopy with biopsy met the diagnostic criteria for EG/EoD.
Completed patient enrollment in Phase 3 EG/EoD and Phase 2/3 eosinophilic esophagitis (EoE) clinical trials of lirentelimab (AK002).
Upcoming 2021 Milestones

Topline data from a randomized, double-blind, placebo-controlled Phase 3 study of lirentelimab in patients with EG/EoD expected in the fourth quarter of 2021.
Topline data from a randomized, double-blind, placebo-controlled Phase 2/3 study of lirentelimab in patients with EoE expected in the fourth quarter of 2021.
Initiation of a randomized, double-blind, placebo-controlled Phase 2/3 study of subcutaneous lirentelimab in patients with EG and/or EoD expected in the second half of 2021.
Initiation of a Phase 2 study in a non-eosinophilic gastrointestinal disease in the second half of 2021.
Second Quarter 2021 Financial Results

Research and development expenses were $41.0 million in the second quarter of 2021 as compared to $28.3 million in the same period in 2020, an increase of $12.7 million.

General and administrative expenses were $16.2 million in the second quarter of 2021 as compared to $12.1 million in the same period in 2020, an increase of $4.1 million.

Allakos reported a net loss of $57.2 million in the second quarter of 2021 as compared to $39.3 million in the same period in 2020, an increase of $17.9 million. Net loss per basic and diluted share was $1.07 for the second quarter of 2021 compared to $0.80 in the same period in 2020.

Allakos ended the second quarter of 2021 with $559.7 million in cash, cash equivalents and marketable securities.

On August 9, 2021 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the second quarter ended June 30, 2021 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, AUG 9, 2021, View Source [SID1234586107]).

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"Our excellent start to 2021 continued in the second quarter with strong quarterly growth resulting in record quarterly revenues from royalties and the signing of our eleventh collaboration and license agreement," said Dr. Helen Torley, president and chief executive officer. "Our recently announced collaboration with ViiV Healthcare demonstrates the potential for our ENHANZE technology to meet evolving patients’ needs by creating long-acting drugs that facilitate rapid, large volume subcutaneous injections of small molecule drugs. This collaboration also highlights the potential for ENHANZE in new disease areas like infectious disease. As a result of this new collaboration and our strong results to date, we are pleased to share that we are raising our guidance for revenues and earnings for the year."

Recent Partner Highlights:

In July 2021, Janssen received U.S. Food and Drug Administration (FDA) approval for DARZALEX FASPRO (daratumumab and hyaluronidase – fijh) in combination with pomalidomide and dexamethasone for patients with multiple myeloma after first or subsequent relapse, marking the sixth indication for DARZALEX FASPRO in the treatment of multiple myeloma.
In June 2021, Halozyme announced a global collaboration and license agreement with ViiV Healthcare, the global specialist HIV company majority owned by GlaxoSmithKline plc. This license gives ViiV exclusive access to Halozyme’s ENHANZE technology for four specific small and large molecule targets for the treatment and prevention of HIV. Under the terms of the agreement, Halozyme received an upfront payment of $40 million and ViiV is obligated to make potential future payments of up to $175 million in development and commercial milestones per target, subject to achievement of specified development and commercial milestones, including certain specified sales milestones. Halozyme will also be entitled to receive mid-single digit royalties on sales of commercialized medicines using the ENHANZE technology.
In June 2021, Janssen was granted two marketing authorizations by the European Commission (EC) for DARZALEX (daratumumab) SC in two new indications in the European Union. The first authorization was for use in combination with cyclophosphamide, bortezomib and dexamethasone (D-VCd) in newly diagnosed adult patients with systemic light chain (AL) amyloidosis marking the first approved therapy for AL amyloidosis in Europe. The second authorization was for use in combination with pomalidomide and dexamethasone (D-Pd) in adult patients with relapsed or refractory multiple myeloma.
In June 2021, Bristol Myers Squibb enrolled the first patient in a Phase 3 study of nivolumab using ENHANZE technology for patients with advanced or metastatic clear cell renal cell carcinoma.
Recent Corporate Highlights:

During the second quarter, the Company repurchased approximately 1.0 million shares of common stock for $48.8 million at an average price per share of $47.05, achieving its target for share repurchases in 2021 with a total of $125 million at an average price per share of $44.43.
Second Quarter Financial Highlights

Revenue for the second quarter was $136.5 million compared to $55.2 million for the second quarter of 2020. The year-over-year increase was primarily driven by $60.0 million in milestone revenues, including $40 million from ViiV and $20 million from Janssen that was earned upon achievement of a commercial milestone related to subcutaneous DARZALEX, an increase in royalty revenue primarily attributable to subcutaneous DARZALEX and an increase in product sales. Revenue for the quarter included $45.8 million in royalties, an increase of 189% compared to $15.8 million in the prior year period.
Cost of product sales for the second quarter was $23.0 million, compared to $5.7 million for the second quarter of 2020. The year-over-year increase was primarily driven by higher product sales, principally the sales of bulk rHuPH20 to the Company’s partners.
Research and development expenses for the second quarter were $8.1 million, compared to $9.0 million for the second quarter of 2020. The decrease in expenses was due to the discontinuation of some development related activities for PEGPH20 and closure of the Company’s oncology operations, partially offset by an increase in costs to support additional ENHANZE targets.
Selling, general and administrative expenses for the second quarter were $12.3 million, compared to $11.0 million for the second quarter of 2020. The increase was primarily due to an increase in compensation expense, including stock compensation, for personnel to support additional ENHANZE targets entering clinical development.
Operating Income: On a GAAP basis in the second quarter of 2021, operating income was $93.0 million, compared to an operating income of $29.6 million in the second quarter of 2020.
Net Income: On a GAAP basis in the second quarter of 2021, net income was $91.5 million, compared with net income of $25.8 million in the second quarter of 2020. Non-GAAP net income was $97.8 million in the second quarter of 2021, compared with Non-GAAP net income of $33.6 million in the second quarter of 2020.1
Earnings per Share: On a GAAP basis in the second quarter of 2021, diluted earnings per share was $0.62, compared with $0.19 in the second quarter of 2020. On a Non-GAAP basis diluted earnings per share was $0.66, compared with diluted earnings per share of $0.24 in the second quarter of 2020.1
Cash, cash equivalents and marketable securities were $755.3 million on June 30, 2021, compared to $368.0 million on December 31, 2020.
During the second quarter, the Company repurchased 1.0 million shares of common stock for $48.8 million at an average price of $47.05, bringing the total for share repurchases since the announcement of the Company’s three-year share repurchase program to $475.0 million at an average price of $23.27.
Financial Outlook for 2021

Based on the recent signing of a new collaboration and license agreement and strong year-to-date results as well as the latest information from collaboration partners and planned expenditures for the year, the Company is raising guidance for 2021 and now expects:

Revenues of $425 million to $445 million, representing year-over-year growth of 59%-66%, and up from prior guidance of $375 to $395 million;
GAAP Operating Income of $260 million to $280 million, representing year-over-year growth of 80%-94%;
GAAP Net Income of $235 million to $255 million, representing year-over-year growth of 82%-98% and Non-GAAP Net Income of $280 million to $300 million, representing year-over-year growth of 75%-87%;1
GAAP Diluted Earnings per Share of $1.55 to $1.70, representing year-over-year growth of 70%-86%, up from prior guidance of $1.25 to $1.40; and
Non-GAAP Diluted Earnings per Share of $1.85 to $2.00, representing year-over-year growth of 65%-78%, up from prior guidance of $1.55 to $1.70.1
Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the second quarter of 2021 today, Monday, August 9, 2021, at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To register for this conference call, please use this link: View Source After registering, you will receive an email confirmation that includes dial in details and unique conference call codes for entry. Registration is open through the live call. However, to ensure you are connected for the full call, please register a day in advance or at minimum 10 minutes before the start of the call.