On August 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported second quarter results and business highlights for the quarter ended June 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, AUG 14, 2024, View Source [SID1234645891]).

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"We are seeing clear evidence that our commercial launch is accelerating, with over 20 cell collections completed in sickle cell disease and beta-thalassemia to date in 2024, and more than 40 additional patients already scheduled to initiate the treatment journey for a bluebird gene therapy by the end of this year," said Andrew Obenshain, chief executive officer, bluebird bio. "We are further encouraged by the commitment to provide patient access across both commercial and government payers, most recently conveyed through multiple positive Medicaid decisions and the growing number of published coverage policies for LYFGENIA, and we expect approximately 85 patient starts across our portfolio this year."

COMMERCIAL LAUNCH UPDATES

LYFGENIA launch building (lovotibeglogene autotemcel); continued commercial momentum for ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

27 patient starts completed across portfolio to date in 2024 (19 ZYNTEGLO, 4 LYFGENIA, 4 SKYSONA).
Launch momentum building with more than 40 additional patients scheduled for cell collection across commercial portfolio through year end.
Successfully completed manufacturing and release testing for first commercial LYFGENIA patient and the first infusion is being scheduled.
Rapid acceleration projected for ZYNTEGLO following the recent approval of additional manufacturing capacity.
Validated access and reimbursement strategy is driving favorable coverage landscape

Significant progress with formalized paths to market access. To date, more than half of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 20% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.
Multiple outcomes-based agreements are published and in place for LYFGENIA with national commercial payer organizations, representing more than 200 million U.S. lives.
Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials to date for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established

bluebird has activated more than 70 total QTCs for LYFGENIA and ZYNTEGLO (defined as a signed MSA).
Six centers are activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
RECENT COMPANY UPDATES

James Sterling appointed as Chief Financial Officer

Effective June 10, 2024, James Sterling became Chief Financial Officer of bluebird bio. Mr. Sterling most recently served as chief financial officer of Renalytix plc, a diagnostics company focused on clinical management of kidney disease. Mr. Sterling was previously managing partner at Renwick Capital LLC, and managing director at investment banks Brock Capital Group LLC and Aleutian Capital Group. He also serves as a board director for a fund managed by Star Mountain Capital. Mr. Sterling has experience as a management consultant at Booz Allen Hamilton. He received his B.A. from Boston University and an M.B.A. from Columbia Business School.
Michael Cloonan appointed to bluebird bio’s Board of Directors

On June 20, 2024, Michael Cloonan was appointed to bluebird bio’s Board of Directors. Mr. Cloonan is currently President and Chief Executive Officer of Sionna Therapeutics. He was previously Chief Operating Officer at Sage Therapeutics leading all business and G&A functions. During his four years with Sage, he helped lead the growth of the organization through multiple capital raises, the launch of the company’s first product, and execution of a transformational, multi-billion-dollar collaboration. An experienced and respected global biotech leader, Mr. Cloonan has over 20 years of biopharma experience across global organizations in various business and commercial roles.
2024 GUIDANCE

The Company anticipates approximately 85 patient starts (cell collections) combined across its portfolio of three FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts by indication for each of its therapies.
The Company continues to anticipate gross-to-net discounts across all three products to be in the range of 20% to 25% of gross revenue in 2024 and expects they will fluctuate based on product and payer mix, as well as utilization of outcomes-based agreements for LYFGENIA and ZYNTEGLO.
Based on projected timelines from cell collection to infusion, the Company anticipates recognizing revenue from its first infusion of LYFGENIA in the third or fourth quarter of 2024.
SECOND QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $193 million, including restricted cash of approximately $49 million, as of June 30, 2024.

Based on current operating assumptions, bluebird expects its cash and cash equivalents as of June 30, 2024 will be sufficient to fund its operations into the second quarter of 2025i, not accounting for the cash minimums required under the Company’s loan agreement with Hercules Capital, Inc. and excluding receipt of any future tranches under the agreement.

The Company has also renegotiated certain terms of its loan agreement with Hercules Capital, Inc. and is eligible to receive two future tranches totaling $50 million, contingent upon achievement of patient start and product delivery milestones and completion of additional financing. These tranches take the place of previously agreed tranches tied to patient starts and gross profit.
Revenue, net: Total revenue, net was $16.1 million for the three months ended June 30, 2024, compared to $6.9 million for the three months ended June 30, 2023. The increase of $9.2 million was due to increased ZYNTEGLO product revenue.
On March 26, 2024, bluebird announced that it will restate its consolidated financial statements as of and for the year ended December 31, 2022, and for each of the first three quarters of 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Form 10-K"). The restatements relate to the identification of leases and the treatment of non-lease components contained in lease agreements. The restatement is not expected to impact the Company’s cash position or revenue. As a result of the restatement, the Company is delayed in filing its 2023 Form 10-K and its Quarterly Reports on Form 10-Q for the quarters ended March 31, 2024 (the "Q1 2024 Form 10-Q") and June 30, 2024 (the "Q2 2024 Form 10-Q"). The Company is continuing to work expeditiously to complete these filings.

The financial results included in this press release represent the most current information available to the Company’s management. The Company expects that its actual results to be reported in its Q2 2024 Form 10-Q will not differ materially from the results included herein, however, these results are subject to change following the completion of the Company’s financial close procedures and the review of its consolidated financial statements for the quarter ended June 30, 2024.

CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its second quarter 2024 results and business updates today, Wednesday, August 14, 2024, at 8:00 am ET.

To participate in the conference call, please dial +1 (800) 715-9871 (U.S. and Canada) and ask to be joined into the bluebird call or provide the Conference ID 2776050.

The live webcast of the call may be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.

On August 14, 2024 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 2024 financial results and business updates (Press release, Precigen, AUG 14, 2024, View Source [SID1234645926]).

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"We are all in on PRGN-2012 given the immense unmet need for RRP patients and our groundbreaking pivotal data supporting the potential of what we hope to be the first-ever FDA approved therapy to treat RRP," said Helen Sabzevari, PhD, President and CEO of Precigen. "By strategically focusing our portfolio, streamlining resources and recent public offering, we have optimized the company to rapidly prepare for submission of a rolling biologics license application under an accelerated approval pathway. We are excited to have initiated enrollment in the confirmatory clinical trial and will continue to accelerate our commercial readiness campaign for a potential launch in 2025 under the leadership of our newly hired Chief Commercial Officer. Additionally, we plan to maximize portfolio value by focusing on strategic partnerships to further advance our highly promising UltraCAR-T programs."

"Our recent reprioritization and public offering is expected to fund our operations into early 2025 allowing us to focus on advancement of PRGN-2012 while continuing to explore potential non-dilutive financing opportunities for future liquidity," said Harry Thomasian Jr., CFO of Precigen.

Key Program Highlights

· PRGN-2012 AdenoVerse Gene Therapy in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA). PRGN-2012 also received Orphan Drug Designation from the FDA and Orphan Drug Designation from the European Commission.

o Results from the pivotal clinical study of PRGN-2012 for the treatment of RRP were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in a late-breaking oral presentation titled, "PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients."

§ Pivotal study met primary safety and efficacy endpoints.

§ 51% (18 out of 35) of patients achieved Complete Response, requiring no surgeries after treatment with PRGN-2012; Complete Responses have been durable beyond 12 months with median duration of follow up of 20 months as of the May 20, 2024 data cutoff.

§ 86% of patients (30 out of 35) had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment; RRP surgeries reduced from a median of 4 (range: 3-10) pre-treatment to 0 (range: 0-7) post-treatment.

§ PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.

§ PRGN-2012 treatment induced HPV 6/11-specific T cell responses in RRP patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders.

§ PRGN-2012 significantly (p < 0.0001) improved Derkay and quality of life scores in complete responders.

o A rolling Biologics License Application (BLA) submission under an accelerated approval pathway is anticipated in the second half of 2024.

o The Company has initiated enrollment in the confirmatory clinical trial, in accordance with the guidance from the FDA, prior to submission of the BLA.

o The Company and the Recurrent Respiratory Papillomatosis Foundation held the inaugural RRP Awareness Day on June 11, 2024. The multi-stakeholder event raised awareness by bringing together individuals living with RRP, caregivers, clinicians, and government officials.

· Strategic Prioritization: In August 2024, the Company announced a strategic prioritization of its clinical portfolio and associated streamlining of resources, including a reduction of over 20% of its workforce, to focus on potential commercialization of PRGN-2012.

o PRGN-2009 AdenoVerse Gene Therapy Clinical Trials

§ The Company plans to continue PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer.

§ PRGN-2009 cervical cancer clinical trial enrollment at non-NCI clinical sites will be paused.

o UltraCAR-T Clinical Programs

§ The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the FDA, and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps.

§ The Company will pause the PRGN-3005 and PRGN-3007 clinical trials.

§ The Company will minimize UltraCAR-T spend and focus on strategic partnerships to further advance UltraCAR-T programs.

o Preclinical Programs

§ The Company will pause all preclinical programs.

o ActoBio

§ The Company has initiated shutdown of its Belgium-based ActoBio subsidiary operations, including planned elimination of all ActoBio personnel.

§ In conjunction with this shutdown, ActoBio’s portfolio of intellectual property will be made available for prospective transactions.

Financial Highlights

· Strategic prioritization resulted in non-cash impairment charges of $32.9 million, net of tax, in the second quarter and severance charges of $3.0 million, of which $2.1 million was recorded in the second quarter and $0.9 million is expected to be recorded in the third quarter.

· The Company closed a public offering of its common stock in August 2024, resulting in net proceeds of approximately $31.4 million.

Second Quarter 2024 Financial Results Compared to Prior Year Period

Research and development expenses increased $3.8 million, or 32%, compared to the three months ended June 30, 2023. Salaries, benefits, and other personnel costs increased $2.1 million primarily due to severance charges related to the shutdown of the Company’s ActoBio subsidiary. Additionally, fees paid to contract research organizations related to the start of the PRGN-2012 confirmatory clinical trial and close out of the PRGN-2012 pivotal clinical trial activities and professional fees incurred related to our manufacturing facility readiness for anticipated BLA submission increased compared to the same period in 2023.

SG&A expenses increased by $1.0 million, or 11%, compared to the three months ended June 30, 2023. This increase was primarily driven by severance costs incurred related to the suspension of ActoBio operations of $0.4 million, increased costs associated with PRGN-2012 commercial readiness as well as increased professional fees incurred related to general corporate matters compared to the same period in 2023.

In conjunction with the suspension of ActoBio’s operations, the Company recorded $34.5 million of impairment charges related to goodwill and other noncurrent assets in the second quarter of 2024, as well as a related tax benefit of $1.7 million.

Total revenues decreased $1.1 million, or 59%, compared to the three months ended June 30, 2023. This decrease was related to reductions in product and service revenues at Exemplar.

Net loss was $58.8 million, or $(0.23) per basic and diluted share, compared to net loss of $20.3 million, or $(0.08) per basic and diluted share, in period ended June 30, 2023.

First Half 2024 Financial Results Compared to Prior Year Period

Research and development expenses increased $5.9 million, or 25%, compared to the six months ended June 30, 2023. Salaries, benefits, and other personnel costs increased by $3.3 million primarily due to $2.1 million of severance charges related to the shutdown of the Company’s ActoBio subsidiary and an increase in the hiring of employees related to the advancement of PRGN-2012 in 2023 at Precigen. Additionally, fees paid to contract research organizations related to the start of the PRGN-2012 confirmatory clinical trial and close out of the PRGN-2012 pivotal clinical trial activities and professional fees incurred related to our manufacturing facility readiness for anticipated BLA submission increased compared to the prior year period. These increases were offset by lower costs incurred at contract research organizations for other programs compared to the same period in 2023.

SG&A expenses decreased by $0.5 million, or 2%, compared to the six months ended June 30, 2023. This decrease was primarily due to lower stock compensation and insurance expenses in 2024 compared to the same period in 2023. These decreases were offset by severance costs incurred in the second quarter of 2024 related to the suspension of ActoBio’s operations, and increased costs related to PRGN-2012 commercial readiness compared to the same period in 2023.

In conjunction with the suspension of ActoBio’s operations, the Company recorded $34.5 million of impairment charges related to goodwill and other noncurrent assets in the second quarter of 2024, as well as a related tax benefit of $1.7 million.

Total revenues decreased $1.9 million, or 51%, compared to the six months ended June 30, 2023. This decrease was related to reductions in product and service revenues at Exemplar.

Net loss was $82.5 million, or $(0.33) per basic and diluted share, compared to net loss of $43.1 million, or $(0.18) per basic and diluted share, in period ended June 30, 2023.

On August 14, 2024 Evotec reported its half-year interim report 2024 (Presentation, Evotec, AUG 14, 2024, View Source [SID1234647164]).

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On August 14, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported the ESMO (Free ESMO Whitepaper) Congress decided to move C4T’s previously accepted preliminary monotherapy Phase 1 abstract for CFT1946, a novel BiDAC degrader in mutant BRAF V600 solid tumors, to an oral presentation (Press release, C4 Therapeutics, AUG 14, 2024, View Source [SID1234645892]). This oral presentation session at the ESMO (Free ESMO Whitepaper) Congress 2024 is scheduled for Friday, September 13, 2024, at 4:00 pm to 5:30 pm CEST. Additionally, C4T announced it will host an investor webcast on Friday, September 13, 2024.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Updated Details for ESMO (Free ESMO Whitepaper) Congress 2024 Presentation
Title: Preliminary Results from a Phase 1 Study of CFT1946, a Novel BiDAC Degrader in Mutant BRAF V600 Solid Tumors
Presentation Date and Time: Friday, September 13, 2024, 4:00 – 5:30 pm CEST
Final Publication Number: 612O
Presenter: Maria Vieito, M.D., Msc (Barcelona, Spain)

C4T Webcast for Analysts and Investors
C4T will host an investor webcast on Friday, September 13, 2024 to discuss the CFT1946 monotherapy data from ongoing CFT1946 Phase 1 trial in BRAF V600 solid tumors. The time of the webcast and access information will be shared closer to the webcast event.

On August 14, 2024 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that allowance for the Company’s "proenzyme composition" patent was received from the Canadian Intellectual Property Office (CIPO) (Press release, Propanc, AUG 14, 2024, View Source [SID1234645927]). The patent broadly captures both high dose and high ratio claims for future clinical doses of the company’s lead asset, PRP. This is the second Canadian patent either allowed or granted in this important North American jurisdiction. Currently, the Company’s intellectual property portfolio consists of 93 patents filed in major jurisdictions relating to the use of PRP against solid tumors.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The proenzymes composition patent is an important part of the IP portfolio covering possible future clinical dosage ranges for PRP, as the Company advances to a Phase 1, First-In-Human (FIH) study in advanced cancer patients suffering from solid tumors. The patent has been granted in major jurisdictions such as Europe, Japan and South East Asia, and is currently under examination in the United States. PRP is targeting the global metastatic cancer treatment market, projected to be worth US$111.2 Billion by 2027, according to current analysis by Emergen Research.

"We continue to grow our intellectual property portfolio in key global jurisdictions," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer. "Our lead asset, PRP, is a novel method to prevent and treat metastatic cancer from solid tumors, but without the severe, or even serious side effects normally associated with standard therapies. The proenzymes composition patent will cover future PRP clinical doses as a welcome addition to the treatment process, such as when resistant tumors could be pretreated by PRP, as a chemo-sensitizing agent. This practical application of our patent portfolio provides a strong indication of our commercial embodiment for future licensing partners, which we believe can potentially revolutionize the way we treat metastatic cancer from solid tumors. Today, metastatic cancer remains the main cause of patient death for sufferers. We look forward to updating our shareholders as we progress."

About PRP:

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas, administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include pancreatic, ovarian, kidney, breast, brain, prostate, colorectal, lung, liver, uterine, and skin cancers. Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.

To view the Company’s "Mechanism of Action" video on the Company’s lead asset, PRP, please click on the following link: View Source