When BIO 2019 closed its doors in Philadelphia last June, none of the delegates of the industry’s largest event would have predicted that the next meeting, scheduled for San Diego, would be canceled and the event would be transformed into a virtual version. In just a few months, the COVID-19 pandemic has decimated our normal way of life and, until effective therapeutics and vaccines become available, how we conduct the business of biotechnology will remain radically different. That was one of the many themes explored during BIO Digital Week last week. While face-to-face meetings and social events were off the agenda this time around, the BIO Digital virtual event still helped companies interact via its one-on-one partnering platform. BIO said that the more than 6,500 registrants had the opportunity to “meet” hundreds of companies focused in 31 therapeutic areas and hear them make their pitches online and describe their pipelines, growth objectives and capital and partnering needs. In fact, according to BIO, more than 5,500 partnering meetings were booked on the platform prior to the meeting. Also, there were plenty of live and “on demand” sessions that could be be accessed during the week.

Clinical trials

Soleno Therapeutics Inc.’s phase III DESTINY PWS (C601) trial evaluating once-daily diazoxide choline controlled-release tablets for treating patients with Prader-Willi syndrome (PWS) missed its primary endpoint of change from baseline in hyperphagia, or insatiable hunger, which is the disease’s predominant syndrome. The top-line results sent Soleno’s stock into a downward spiral as shares closed 35% lower June 9 at $2.10.

COVID-19 developments

The European Commission is laying plans for joint procurement of COVID-19 vaccines on behalf of all 27 EU member states and is in discussions to agree on a harmonized approach to access and who gets vaccinated first.

A2A Pharmaceuticals Inc. inked an agreement to co-develop SARS-CoV-2 main proteases inhibitors for the treatment of COVID-19 with Laxai Life Sciences Pvt. Ltd. Under the collaboration, A2A designed the molecules using its computational AI-enabled drug discovery platform Sculpt, which are currently being synthesized and will be evaluated by Laxai. Both parties will collaborate in preclinical optimization and selection of lead candidates to enter the clinic. Adaptive Biotechnologies Corp. launched Immunecode with Microsoft Corp., which provides detailed views of the immune response to COVID-19 in real time based on de-identified data generated from thousands of COVID-19 blood samples from patients around the globe. CSL Ltd. and the University of Queensland (UQ) have entered a development agreement with the Coalition for Epidemic Preparedness Innovations (CEPI) to accelerate development, manufacture and distribution of a COVID-19 vaccine candidate developed by the University of Queensland. The agreement formalizes support provided by CSL to UQ and CEPI from the outset of the pandemic earlier this year. Emergent Biosolutions Inc. will deploy its molecule-to-market contract development and manufacturing services to support the manufacturing of Astrazeneca plc’s vaccine candidate for COVID-19, AZD-1222, a viral vector-based, weakened version of adenovirus containing the genetic material of SARS-CoV-2 spike protein, developed by Oxford University’s Jenner institute working with the Oxford Vaccine Group. IGY Immune Technologies & Life Sciences Inc. said it will collaborate with MMS Holdings to further the development of IgY-110, an anti-CoV-2 therapeutic antibody with a focus on a nasal spray application. The approach of an IgY antibody in a nasal spray will be complementary to any vaccine developed, the company said. Johnson & Johnson said its Janssen Pharmaceutical Cos. division is accelerating initiation of a phase I/IIa first-in-human trial of SARS-CoV-2 vaccine Ad26.COV2-S, recombinant. The study is now expected to start in the second half of July rather than in September as previously disclosed. Kalytera Therapeutics Inc. said preclinical proof-of-concept data show its R-107 liquid nitric oxide donor candidate for pulmonary arterial hypertension was effective in a classic rodent model designed to mimic life-threatening COVID-19 lung disease. Sinovac Biotech Ltd. and Instituto Butantan, of Sao Paolo, will collaborate to advance clinical trials of Coronavac, Sinovac’s inactivated vaccine candidate against COVID-19. Sinovac said it received approval from China’s National Medical Products Administration to conduct phase I/II trials in China to determine the vaccine candidate’s safety, tolerance, dosage and immunization schedule. Twist Bioscience Corp. is collaborating with Serimmune Inc. to develop an antibody targeting SARS-CoV-2. The collaboration will use Serimmune's Serum Epitope Repertoire Analysis (SERA) platform to map the antigenic targets of antibody candidates from Twist's libraries. Those epitopes will be used to rescreen Twist's libraries. Twist will be responsible for advancing all antibodies resulting from the collaboration. Vault Pharma Inc. is working with the University of California, Los Angeles and other universities to incorporate its bioparticle-based delivery system in potential COVID-19 vaccination.

Deals of the week

CSL Ltd. said its subsidiary, CSL Behring, has agreed to exercise an option to acquire Canadian biotech Vitaeris Inc. for its lead phase III asset for rejection in solid organ kidney transplant patients. The two companies entered a partnership in 2017 to expedite the development of clazakizumab, a humanized anti-interleukin 6 monoclonal antibody, and CSL had the option to acquire Vitaeris in full.

Evox Therapeutics Ltd. sealed a $1.2 billion collaboration with Eli Lilly and Co. to apply its exosome technology to the doubly difficult task of systemically delivering RNA interference and antisense oligonucleotide drugs, to reach central nervous system targets. It will be responsible for engineering exosomes to achieve brain and CNS delivery to five unspecified neurological disease targets. The terms include $20 million up front, research funding over three years and a $10 million investment from Lilly in exchange for a convertible bond. The rest of the $1.2 billion will be directed to milestone payments if all five products are approved and will be followed by additional royalties on net sales of products that make it to market.

Genmab A/S has established an oncology alliance with Abbvie Inc., receiving $750 million up front and up to $3.15 billion in development, regulatory and commercial milestones for up to seven next-generation antibody-based therapeutics.

Innovent Biologics Inc. is expanding its product portfolio and enhancing efforts in developing bispecific antibodies and cell therapies in a potential multibillion-dollar deal with Roche Holding AG with a collaboration to take aim at hematological and solid cancers. Innovent will pay undisclosed up-front development and commercial milestone payments and royalties to Roche for accessing some of its technologies nonexclusively. Those technologies will be used to enable the discovery and development of specific 2-to-1 T-cell bispecific antibodies and the universal CAR T platform. Roche retains an option right to license product candidates for development and commercialization outside of China.

Microbiotica Ltd. plans to collaborate with Cancer Research U.K. and Cambridge University Hospitals (CUH) NHS Foundation Trust to identify and develop microbiome co-therapeutics and biomarkers for cancer patients receiving immune checkpoint inhibitor therapy. The collaboration is based on clinical studies conducted by CUH that evaluate immune checkpoint inhibitor drug response in cancer patients. Microbiotica will conduct mass culturing of patient gut bacteria, microbiome sequencing and machine learning analysis with the goal of identifying gut bacterial signatures correlated with drug efficacy as well as side effects in individuals in treatment for melanoma, non-small-cell lung and renal cancer. Novo Nordisk A/S is buying Corvidia Therapeutics Inc. for $2.1 billion, which includes a $725 million up-front payment. Novo will receive all Corvidia’s outstanding shares. Payments to Corvidia’s shareholders could eventually hit $2.1 billion if Novo hits certain regulatory and sales milestones. Oxford Biomedica plc said Sanofi SA, following completion of a company-wide portfolio review, intends to return rights to ophthalmology gene therapy programs SAR-422459 for Stargardt disease and SAR-421869 for Usher’s syndrome type Ib. Sernova Corp. said it acquired cellular immune protection technology through an asset purchase agreement with Converge Biotech Inc. In the transaction, Sernova is acquiring the intellectual property associated with Converge's conformal coating cell encapsulation technology. Selecta Biosciences Inc. and Swedish Orphan Biovitrum AB (Sobi) signed a licensing agreement for SEL-212, which is designed to control serum uric acid, reduce immunogenicity and allow for repeated monthly dosing when treating chronic refractory gout. Sobi will make initial payments to Selecta of $100 million, including a $75 million up-front license fee and a $25 million private placement of shares of Selecta common stock. Selecta could receive milestone payments of up to $630 million if certain regulatory and development targets and sales thresholds are met. Selecta also could receive tiered double-digit royalties on net sales. Scopus Biopharma Inc. has signed an exclusive, worldwide license to a potentially first-in-class, targeted immuno-oncology gene therapy drug developed at City of Hope, of Los Angeles. The licensed gene therapy drug, CpG-STAT3siRNA, is a highly selective and targeted therapy that silences activity of the STAT3 gene by way of RNA interference while stimulating the TLR9 receptors to activate the body's immune defense to recognize and kill cancer cells. Teneobio Inc. inked a research collaboration and licensing agreement with Intellia Therapeutics Inc. covering the use of Teneobio’s heavy chain antibodies, or Uniabs, for Intellia’s engineered cell therapies. Teneobio also will use its platforms to assess Uniabs against targets that can be used in engineered cell therapies to treat life-threatening diseases.

Financial transactions

Public offerings: Applied Molecular Transport Inc. closed its IPO of 12.65 million shares, which includes the exercise in full by the underwriters of their option to purchase up to 1.65 million additional shares, at a public offering price of $14 each. The total gross proceeds were $177.1 million. Calliditas Therapeutics AB said it raised $90 million in its U.S. IPO, listing its American depositary shares (ADS) on Nasdaq as CALT. Chemocentryx Inc. priced an underwritten public offering for expected gross proceeds of $301.6 million. Fate Therapeutics Inc. priced a public offering for aggregate gross proceeds of about $175 million. Fusion Pharmaceuticals Inc. filed to raise up to $100 million in an IPO. Lantern Pharma Inc. priced its IPO of 1.75 million common shares at $15 apiece, raising gross proceeds of $26.25 million. Legend Biotech Corp. closed its IPO of about 21 million American depositary shares (ADSs), each representing two ordinary shares, at a public offering price of $23 per ADS, for total gross proceeds of about $487.3 million. Polypid Ltd. filed to raise up to $58 million in an IPO. Replimune Group Inc. priced its public offering for gross proceeds of about $100 million. Retrophin Inc. priced its public offering for gross proceeds of about $100.8 million. Vaxcyte Inc. filed to raise $210 million by offering 14 million shares in an IPO at a price range of between $14 to $16.

Other public offerings: Albireo Pharma Inc. agreed to terms with Hercules Capital Inc. on a debt facility to provide up to $80 million of new capital. Amyris Inc. closed its private investment in public equity and received gross proceeds of $200 million. Antibe Therapeutics Inc. entered a bought deal agreement with a syndicate of underwriters who will purchase 62.5 million units at CA40 cents each for gross proceeds of CA$25 million (US$18.67 million). Cerecor Inc. signed an underwriting agreement with Oppenheimer & Co. Inc., which agreed to purchase 13.2 million shares of common stock of the company at $2.50 per share. Fulcrum Therapeutics Inc. closed a $68.5 million private placement with a group of institutional investors. Jazz Pharmaceuticals plc priced $850 million aggregate principal amount of 2% exchangeable senior notes due 2026 in a private offering by Jazz Investments I Ltd., its wholly owned subsidiary, to qualified institutional buyers.

Venture funding: Autobahn Therapeutics Inc. raised $76 million from a series B round to support advancing lead candidate ABX-002, a thyroid hormone receptor beta agonist therapy for multiple sclerosis and adrenomyeloneuropathy, a rare genetic disorder, plus a portfolio of central nervous system programs that leverage the company’s brain-targeting chemistry platform. Checkmate Pharmaceuticals Inc. completed an $85 million series C round to continue developing CMP-001, a differentiated Toll-like receptor 9 (TLR9) agonist, for treating anti-PD-1-refractory melanoma and to study additional indications that include front-line melanoma and head and neck squamous cell carcinoma. Glyscend Therapeutics Inc. closed a $20.5 million series A financing round led by Brandon Capital Partners, through its Medical Research Commercialization Fund, and Sante Ventures. JW Therapeutics (Shanghai) Co. Ltd. completed a $100 million series B round. Lycia Therapeutics Inc. raised $50 million in series A funding from founding investor Versant Ventures to take forward a novel concept in targeted protein degradation. The new company is building on the work of Carolyn Bertozzi, professor of chemistry at Stanford University and Howard Hughes Medical Institute investigator, who has invented bifunctional structures called Lytacs – lysosomal targeting chimeras – which target extracellular or circulating proteins for internalization and lysosomal degradation by tethering them to lysosome targeting receptors at the cell surface. Verve Therapeutics Inc. raised $63 million in a series A2 financing.

Regulatory news

Responding to the times, the FDA issued a final guidance Monday allowing patients to get drug samples delivered to their homes for the duration of the COVID-19 public health emergency. Some rules must be followed. For instance, a licensed practitioner must request the samples in writing and designate specific patients who are to receive them. Receipt of the drug samples must be properly documented and manufacturers or the authorized distributor of record must comply with the recordkeeping and other requirements of the Prescription Drug Marketing Act and FDA Part 203 regulations. While direct-to-the-patient delivery is a temporary measure, the FDA said a provision allowing samples to be delivered to a physician’s home likely will become a permanent measure.

The FDA and National Center for Advancing Translational Sciences (NCATS) at the NIH said they made updates to the CURE ID application to be a more effective tool during the COVID-19 public health emergency. CURE ID is an internet-based repository that allows clinicians to report their experiences treating difficult-to-treat infectious diseases with novel uses of existing drugs through a website, a smartphone or other mobile device. Clinicians can also access cases entered by other users and view them as individual case reports or an aggregated dataset. The FDA and NCATS/NIH are also collaborating with the National Institute of Allergy and Infectious Diseases at NIH, the CDC, the Critical Path Institute, the World Health Organization and the Infectious Diseases Society of America to assess the global utility of the CURE ID platform for COVID-19 case information.


Word on the Street: Industry responses to the issues of the week

"I think what we're witnessing right now is an unprecedented mobilization of companies and technology and capabilities on multiple levels, the likes of which we've never seen before.”
Gil Van Bokkelen, CEO, Athersys Inc., speaking at the BIO Digital meeting

“Our job, working together with member states and other international partners, is to provide the right framework. There are a number of dimensions to this and, inescapably, a political dimension – it can’t be allowed to degenerate into a race about who gets there first. For a challenge on this scale, procurement has to be done as jointly as possible. European member states won’t be able to do it better on [their] own.”
Martin Seychell, deputy director of the EU health directorate DG Santé, on EU plans for joint procurement of COVID-19 vaccines on behalf of all 27 EU member states

“Even with an event as significant as COVID-19, memories fade and attention moves quickly to the next crisis. That makes it imperative that Congress act on needed changes this year in order to better prepare for the next pandemic.”
Sen. Lamar Alexander (R-Tenn.), who identified areas that must be addressed for preparing for the next pandemic in a white paper – accelerating R&D for tests, treatments and vaccines; improving infectious disease surveillance; rebuilding stockpiles and improving the medical supply surge capacity and distribution; improving local and state public health capabilities; and improving coordination of federal agencies during a health emergency

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