On June 29, 2020 CRISPR Therapeutics (Nasdaq:CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, reported that it is commencing an underwritten public offering of $325,000,000 of common shares (Press release, CRISPR Therapeutics, JUN 29, 2020, View Source [SID1234563988]). In addition, the underwriters will have a 30-day option to purchase up to an additional $48,750,000 of common shares at the public offering price less the underwriting discount.

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Goldman Sachs & Co. LLC, BofA Securities and Jefferies are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering.

The common shares will be offered and sold pursuant to the Company’s previously filed automatically effective shelf registration statement on Form S-3 (File No. 333-227427) filed with the U.S. Securities and Exchange Commission (the "SEC") on September 19, 2018. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering will be made only by means of a prospectus. A copy of the prospectus supplement relating to the offering will be filed with the SEC and may be obtained, when available, from Goldman Sachs & Co. LLC by mail at 200 West Street, New York, NY 10282, Attention: Prospectus Department, by telephone at (866) 471-2526, or by email at [email protected]; from BofA Securities by mail at NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at [email protected]; or from Jefferies, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 547-6340, or by email at [email protected].

On June 29, 2020 Cancer Prevention Pharmaceuticals, Inc. (CPP), a private biotech company developing novel therapeutics to prevent cancer and other diseases, reported that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for CPP-1X/sul for treatment of adults with familial adenomatous polyposis (FAP) (Press release, Cancer Prevention Pharmaceuticals, JUN 29, 2020, https://www.canprevent.com/wp-content/uploads/2020/06/NDA-Submission-Press-Release-FINAL-2020-06-29.pdf [SID1234563867]). FAP is a rare genetic disease that if left untreated progresses to colorectal cancer in nearly 100% of patients. The clinical development of CPP-1X/sul was designed to establish this fixed dose combination product as a potential pharmaco-preventive drug treatment specifically for FAP patients.

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"The NDA submission for our lead drug candidate, CPP-1X/sul, represents a significant milestone for FAP patients and their families," said CPP CEO Jeff Jacob. "For most FAP patients, current medical practice involves a lifetime of periodic monitoring as well as highly invasive surgical procedures. If approved, CPP-1X/sul could provide an alternative to surgery for many patients, significantly improving their quality of life."

The FDA’s accelerated approval process allows the agency to approve drugs which address a serious or life-threatening condition based on an intermediate or surrogate endpoint that is likely to predict a clinical long-term benefit. The FDA takes into account such factors as the severity, rarity, or the lack of effective current treatments. The designation has been frequently applied to cancer drugs.

In addition, CPP-1X/sul received an orphan drug designation from the FDA. Among the benefits are eligibility for seven years of market exclusivity upon approval of a drug and tax credits for various costs of clinical testing.

CPP also recently submitted a Marketing Authorization Application (MAA) with the European Union (EU) for CPP-1X/sul for the same indication. The drug received an orphan medicinal product designation for FAP following a favorable assessment provided by the European Medicines Agency’s Committee for Orphan Medicinal Products.

About CPP-1X/sul
CPP-1X/sul is a combination of CPP-1X (eflornithine) and sulindac (CPP-1X/sul). In a clinical trial in patients with large bowel polyps, the CPP-1X/sul combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Based on the close biologic similarities with FAP, a Phase 3 pivotal trial compared this same combination to each drug alone (CPP FAP-310).

"With up to four years of treatment, the combination appears to greatly delay the need for major surgeries in the colon, rectum or surgical pouch. We hope that this new drug regimen will soon be available as an adjunct in the management of FAP patients facing large bowel surgery," said Dr. Alfred Cohen, Chief Medical Officer of CPP.

The CPP FAP-310 trial enrolled 171 FAP patients at 17 research institutes in the U.S., Canada, and Europe. It was the largest ever prospective, controlled study performed in FAP and treated patients for up to 48 months, much longer than any other clinical trial in this population. The study was designed to determine if CPP-1X/sul is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of any FAP-related event, such as surgical removal of the colon, rectum, surgical pouch, duodenum and/or high-risk adenomas. The trial design included FAP patients with varying lower and upper GI disease. CPP-1X/sul did not demonstrate statistical significance in outperforming the single agents in the overall population; however, further analysis of the data showed key differential effects of the agents in the lower vs upper GI anatomy. Focusing on lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), the data showed statistically significant benefit for CPP-1X/sul vs both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. Also, the safety profile of the combination did not significantly differ from that already known for the single agents to support the overall safety assessment of the fixed combination product for long-term therapeutic use.

On June 29, 2020 OmniSeq, an innovator in next generation sequencing (NGS) in oncology, reported the benefits of its OmniSeq Advance comprehensive genomic and immune profiling assay to detect the presence of rare alterations in the RET gene in the tumors of some patients with non-small cell lung cancer (NSCLC) and thyroid cancer (Press release, OmniSeq, JUN 29, 2020, View Source [SID1234561571]). Those patients eligible for treatment with Eli Lilly and Company’s recently FDA-approved selective RET kinase inhibitor Retevmo* (selpercatinib).

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Retevmo is the first FDA-approved therapy specifically for cancer patients with fusions or mutations in the RET (rearranged during transfection) gene. OmniSeq Advance delivers the distinct advantage of using two technologies; RNA-sequencing for RET fusions and DNA-sequencing for RET mutations to provide optimal, comprehensive testing for Retevmo. OmniSeq Advance was one of the tests that could qualify a patient for enrollment to the LIBRETTO-001 Retevmo (selpercatinib) clinical trial.

The National Comprehensive Cancer Network (NCCN) recommends for NSCLC that when feasible, testing be performed via a broad, panel-based approach, most typically performed by next generation sequencing. For patients who, in broad panel testing don’t have identifiable driver oncogenes (especially in never smokers), consider RNA-based NGS if not already performed, to maximize detection of fusion events.

"OmniSeq Advance offers significant advantages for detecting RET fusions by RNA-sequencing as opposed to DNA-only sequencing," stated OmniSeq’s Chief Medical Officer, Roger Klein, MD, JD, FACP. "OmniSeq’s proprietary sample preparation methodologies also allow for minimal tumor tissue input, a critical factor for ensuring NSCLC patient testing can be completed with frequently limited biopsy material available."

OmniSeq’s NGS-based assays provide comprehensive genomic and immune profiling from RNA and DNA to enable oncologists to select the most appropriate therapies or clinical trials for each patient. OmniSeq’s suite of clinical diagnostic tests are offered exclusively by LabCorp to U.S.-based physicians through its Integrated Oncology specialty laboratory and to global biopharmaceutical customers through Covance, LabCorp’s drug development business. OmniSeq Advance is approved by New York State’s Clinical Laboratory Evaluation Program.

Retevmo is indicated for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer, of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trial(s). There is currently no FDA-approved companion diagnostic for Retevmo.

On June 29, 2020 ALX Oncology Holdings, a next-gen cancer biotech reported that that launched in 2015 seeking a $100 million IPO to add to its recent $105 million funding round (Press release, FierceBiotech, JUN 29, 2020, View Source [SID1234561570]).

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The Dublin- and California-based biotech has so far had a good year and, back in February, just after its series C round, grabbed an FDA speedy tag for its leading candidate ALX148 for the first-line treatment of patients with head and neck squamous cell carcinoma and for the second-line treatment of patients with HER2-positive gastric or gastroesophageal junction carcinoma.

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CD47 myeloid checkpoint inhibitor ALX148 is now in line for phase 2 trials in combination with other anti-cancer therapies.

Multiple drug developers have gone after CD47 since evidence of its role in the interactions between cancer cells and the immune system became clear. The evidence suggested tumors evade immune attacks by upregulating CD47, leading researchers to posit that blocking the signal will expose cancer cells to a full-force offensive.

However, efforts to realize the therapeutic potential of anti-CD47 antibodies have been undermined by hematological adverse events stemming from the fact the target is found on host cells such as red blood cells.

ALX, formerly known as Alexo Therapeutics, thinks fusion protein ALX148 has a better risk-benefit profile than its rivals and has persuaded investors to buy into that idea.

Some of its more recent data were from patients with relapsed or refractory non-Hodgkin lymphoma (NHL) who received ALX148 in combination with rituximab. Among the 21 evaluable patients, ALX saw an objective response rate of 43% and a median progression-free survival of 7.3 months. As importantly, the drug appeared to be relatively safe and well tolerated.

The phase 1 trial that generated the lymphoma data is continuing, with other arms of the study testing the CD47 prospect in combination with Herceptin and Keytruda in solid tumor patients. But, with ALX148 coming through its first test in NHL, ALX also has plans to take the candidate deeper into the clinic.

The biotech said in a Securities and Exchange Commission (SEC) filing that it now also plans to advance ALX148 into a phase 1b/2 trial in combination with Vidaza (azacitidine) for myelodysplastic syndromes by the end of 2020 and a phase 1b/2 trial in combination with standard-of-care agents for acute myeloid leukemia in 2021.

It plans to list on the Nasdaq under the symbol "ALXO."

The second IPO attempt is a $75 million effort from autoimmune biotech Pandion Therapeutics. Since it uncloaked two years ago, Pandion has teamed up with Astellas on bispecific drugs for Type 1 diabetes and has pushed its lead program into the clinic.

Back in April, it raised $80 million to test that treatment in patients with ulcerative colitis and move a second program into phase 1.

Cambridge, Massachusetts-based Pandion set up shop with its so-called TALON platform to create better treatments for autoimmune conditions, in which the immune system "forgets what the self is" and attacks its own tissues and organs. It aims to replace old-school systemic immunosuppression—which can lead to side effects like an increased risk of infection or cancer—as well as newer anti-cytokine antibodies, which have improved care for some patients but don’t work for all autoimmune diseases.

Its lead asset, PT101, is a combination of an interleukin-2 mutein effector module with a protein backbone and is designed to selectively expand regulatory T cells systemically without activating proinflammatory cells, such as conventional T cells and natural killer cells.

"We are initially developing PT101 for the treatment of patients with moderate-to-severe ulcerative colitis, or UC, and are currently conducting a Phase 1a clinical trial of PT101 in healthy volunteers, with final data expected in the first half of 2021," the biotech said in its SEC-1 filing.

Using its TALON platform, it added it would also "continue to develop and expand our library of effector and tether modules as part of our early stage research and discovery pipeline."

On June 29, 2020 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel immunotherapy treatments for cancer and autoimmune diseases, reported it has enrolled and safely dosed the last patient for stage 2 of Part A (1st line NSCLC) of its TACTI-002 Phase II study, completing recruitment for Part A (Press release, Immutep, JUN 29, 2020, View Source [SID1234561557]).

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TACTI-002 is being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada) and is evaluating the combination of Immutep’s lead product candidate, eftilagimod alpha ("efti" or "IMP321") with MSD’s KEYTRUDA (pembrolizumab).

Immutep recently reported new data from TACTI-002 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Annual Meeting 2020, including results from stage 1 of Part A which showed an improving Progression Free Survival ("PFS") estimate of more than 9 months in patients with 1st line NSCLC.

The Company expects to report more mature data from TACTI-002 in H2 CY20.

TACTI-002 Recruitment Update

In total 81 patients out of up to 109 (74%) are already enrolled in the trial at 12 clinical sites across Australia, Europe, the UK and US. Recruitment is ongoing for Part B (second line NSCLC) and for stage 2 of Part C (2nd line HNSCC). Current recruitment numbers for each Part are below.

About the TACT-002 Trial

TACTI-002 (Two ACTive Immunotherapies) is being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada). The study is evaluating the combination of efti with MSD’s KEYTRUDA (pembrolizumab) in up to 109 patients with second line head and neck squamous cell carcinoma or non-small cell lung cancer in first and second line.

The trial is a Phase II, Simon’s two-stage, non-comparative, open-label, single-arm, multicentre clinical study that is taking place in up to 12 study centres across the U.S., Europe, UK and Australia.

Patients participating in three parts:

Part A – First line Non-Small Cell Lung Cancer (NSCLC), PD-X naive

Part B – Second line NSCLC, PD-X refractory

Part C – Second line Head and Neck Squamous Cell Carcinoma (HNSCC), PD-X naive

TACTI-002 is an all comer study in terms of PD-L1 status, a well-known predictive marker for response to pembrolizumab monotherapy especially in NSCLC. PD-L1 expression is typically reported in three groups for NSCLC: < 1%, 1-49% and ³50% (Tumour Proportion Score or TPS). Patients with a high PD-L1 status are typically more responsive to anti-PD-1 monotherapy such as pembrolizumab, whereas those with low PD-L1 status are overall significantly less responsive. Pembrolizumab monotherapy is registered in the US and the EU for first-line NSCLC patients with a TPS score ³1% (US) and ³50% (EU), reflecting 65% and 30% of all first line NSCLC patients, respectively.