Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

These firms have gained the notice of those in high places.

2014 to date has been the best year since the 2007–09 recession for young biopharmas dreaming of securing big money as the financial markets have finally warmed to drug or diagnostic developers, whether they raised private money as on this list, went public or, as happened in several instances, they raised private financing and then went public.

That encouraging trend is reflected in many of the big numbers and big names that dot this year’s list. The number-one company this time around received $200 million, compared with $150 million for the top-ranked company on last year’s GEN List of Young Companies in the Money: 25 Firms to Watch.

And talk about big-name investors: This year’s 25 Young Companies in the Money include biopharmas that received funding from Google, J. Craig Venter, Ph.D., the founder of Watson Pharmaceuticals, and two managing partners in the Boston Celtics.

Again as with last year, this GEN List does not include companies that secured private funding and later went public, or filed registration statements to do so. By our count, no fewer than seven companies that received private funding and otherwise would have appeared here went public (Auspex, Avalanche Biotechnologies, Civitas Therapeutics, Kite Pharma, Otonomy, Proteon Therapeutics, and Versartis), while an additional two companies have IPOs pending but have not yet traded their first public shares (Coherus Biosciences, Kolltan Pharmaceuticals).

Below is a list of 25 privately held biotechnology and pharmaceutical companies, ranked by the amount of nonpublic financing they have raised so far in 2014. Each company is listed by total raised (with a breakdown of the amount for companies reporting two events this year), the type of financing, the date announced, the purpose of the financing as stated by companies, the financing leader, other firms involved in the financing with their status as new or existing investors noted where available, and footnotes offering details of the events.


#25. Anokion

Amount: $34.5 million (CHF 33 million)

Type: Series A financing

Date announced: May 5

Purpose: Develop clinical candidates in the areas of immune-masked protein therapeutics, autoimmune, and allergic diseases. First clinical trials are planned for 2017.

Financing leaders: Novartis Venture Fund, Novo Ventures, and Versant Ventures

Additional financing partners: “Additional participation by private investors.”


#24. JHL Biotech

Amount: $35 million

Type: Series B financing

Date announced: June 19

Purposes: Develop biosimilar monoclonal antibody pipeline, accelerate filing of investigational new drug applications, support ongoing facility expansion in Taiwan, acquire additional equipment for commercial manufacturing facility in Wuhan, China, which is expected to be completed by year’s end.

Financing leaders: Milestone Capital and a consortium of Taiwan venture capitalists and individuals, including President International Development Corp., Fubon Financial Holdings, Taishin Financial Holdings, TaiAn Technologies, Hotung Group, and Allen Chao, Ph.D., founder of Watson Pharmaceuticals and its CEO from 1994–2008 (the company is now Actavis).

Additional financing partners: Existing Series A shareholders China Development Industrial Bank, Biomark Capital, and Kleiner Perkins Caufield & Byers.


#23. Flex Pharma

Amount: $40 million

Type: Private funding from syndicate of “blue chip public and private investors,” including “top-tier venture capital and leading crossover funds”

Date announced: September 8

Purpose: Further advance the science and its clinical candidates addressing neuromuscular disorders.

Financing leader: No leader denoted

Financial partners: Longwood Fund; Bessemer Venture Partners; EcoR1 Capital; Jennison Associates (on behalf of clients); Lightstone Ventures; Alexandria Equities; CD-Venture GmbH; Bindley Capital Partners; City Hill Ventures; Richard Mark Beleson, retired SVP, Capital Research Company; Todd Dagres, Spark Capital general partner; Richard Gibbs, Gibbs Capital Management; Daniel Gold, managing partner, founder, and CEO, QVT Financial; Wyc Grousbeck, Boston Celtics managing partner and CEO; Robert Steven Kaplan, Ph.D., Harvard Business School; The Kraft Group; Peter S. Lynch, trustee, The Lynch Foundation; John Maraganore, Ph.D., Alnylam CEO; Judy Pagliuca, managing partner, Pags Growth Capital; Steve Pagliuca, Bain Capital managing director, Boston Celtics managing partner; William Sahlman, Ph.D., Harvard Business School; Diane and John Sculley, former Apple CEO & Sculley Family Office; Jonathan Seelig, co-founder, Akamai Technologies.


#22. Alios BioPharma1

Amount: $41 million

Type: Series B financing

Date announced: April 7

Purpose: Primarily support continued clinical development of Alios' respiratory portfolio, including AL-8176, an anti-RSV nucleoside analog now in Phase II clinical development, and multiple late-stage preclinical development programs targeting influenza and rhinovirus.

Financing leader: Undisclosed new investor

Additional financial partners: Existing investors Novo Ventures, SR One (the corporate venture capital arm of GlaxoSmithKline), Roche Venture Fund, and Novartis Ventures.


#20 (tie). PanOptica

Amount: $45 million

Type: Series B financing

Date announced: April 15

Purpose: Fund clinical program for lead compound, PAN-90806, a small molecule selective inhibitor of vascular endothelial growth factor-signaling being investigated as a topical (eye drop) treatment for neovascular age-related macular degeneration.

Financing leaders: New investor Novo Ventures and existing investor Third Rock Ventures

Additional financing partners: Founding investor SV Life Sciences.


#20 (tie). Lumena Pharmaceuticals

Amount: $45 million

Type: Series B financing

Date announced: March 11

Purpose: Advance clinical development of lead product candidate LUM001 for rare cholestatic liver disease in pediatric and adult patients, as well as planned Phase II clinical trial of LUM002 for nonalcoholic steatohepatitis (NASH).

Financing leader: New Enterprise Associates (NEA)

Additional financial partners: New investors Adage Capital Management and RA Capital Management; Existing stakeholders Pappas Ventures, RiverVest Venture Partners, and Alta Partners.


#19. Integrated Diagnostics (Indi)

Amount: $47.25 million

Type: $30.25 million in Series B financing; $17 million nondilutive debt financing

Date announced: April 29

Purpose: Further commercialize Xpresys™ Lung, a molecular diagnostic blood test launched last year to help doctors manage indeterminate lung nodules; develop additional proteomic blood tests using Indi’s technology platform.

Financing leaders: Series B financing led by Baird Capital; debt financing provided by Life Sciences Alternative Funding

Additional financing partners: Series B financing joined by new investor Alexandria Venture Investments, the strategic venture arm of Alexandria Real Estate Equities, and by existing investors InterWest Partners and the Wellcome Trust.


#18. Thesan Pharmaceuticals

Amount: $49 million

Type: Series B financing

Date announced: February 24

Purpose: Support development of novel approaches for the treatment of dermatological disorders, including acne and atopic dermatitis.

Financing leader: Novo Ventures, an existing investor

Additional financial partners: SV Life Sciences, Lundbeckfond Ventures.


#17. Principia Biopharma

Amount: $50 million

Type: Series B financing

Date announced: April 22

Purpose: Further advance the company's pipeline, including an oral BTK inhibitor for potential use in autoimmune disease and oncology, an oral, selective, irreversible FGFR inhibitor program for the treatment of solid tumors, an oral IL-17 pathway inhibitor program with utility across a range of autoimmune and inflammatory diseases; as well as helping Principia expand into a clinical stage company.

Financing leader: Sofinnova Ventures

Additional financing partners: All of Principia's existing institutional investors: Morgenthaler Ventures, New Leaf Venture Partners, OrbiMed, SR One, Mission Bay Capital, and additional undisclosed investors.


#16. Advanced Accelerator Applications

Amount: $51.7 million (€41 million)

Type: Unspecified capital increase

Date announced: February 17

Purpose: Help fund expansion plans and finance clinical trials of its portfolio of MNM diagnostic and therapeutic products.

Financing leader: HBM Healthcare Investments, which invested $25.2 million (€20 million)

Additional financial partners: Existing and new shareholders, including undisclosed private investors and funds that include a company of the Tamburi Investment Partners Group.


#15. Aduro BioTech

Amount: $55 million

Type: Series C financing

Date announced: June 11

Purposes: Advance lead program in metastatic pancreatic cancer through its ongoing Phase IIb ECLIPSE clinical trial, continue clinical development in mesothelioma and high-grade glioma, expand into additional indications, and advance small molecule program targeting the immunomodulatory STING receptor.

Financing leader: None denoted

Additional financing partners: Johnson & Johnson Development Corporation (JJDC), J&J’s venture capital subsidiary; Morningside; and other undisclosed “new and existing investors”.


#14. Oxford Nanopore

Amount: $56.8 million (£35 million)

Type: Private placement of ordinary shares

Date announced: August 12

Purpose: Accelerate and scale the adoption of MinION™ sequencers, which use nanopore technology; further develop commercial and manufacturing infrastructure serving early customers through the company's MinION Access Programme (MAP), launched last year—including scaling-up of production and logistics, and expansion of customer support and application development functions. In addition, the funds will support the company's R&D operation including its pipeline of scalable nanopore-sensing products.

Financing leader: None specified

Additional financing partners: New investors, including the CF Woodford Equity Income Fund managed by Woodford Investment Management; existing investors in the U.K., U.S., and mainland Europe.


#13. NuCana

Amount: $57 million

Type: Series B financing

Date announced: April 10

Purpose: Expand the clinical program for lead product Acelarin, which will be developed initially for patients with pancreatic, biliary, lung, and ovarian cancers; bring a second ProTide (NUC-3373) into clinical trials later this year, with two further ProTides scheduled for 2015.

Financing leader: Sofinnova Ventures, a new investor

Additional financial partners: Existing investors Sofinnova Partners (largest shareholder), Morningside Ventures, Alida Capital International, and the Scottish Investment Bank, the investment arm of Scottish Enterprise.


#12. ProNAi

Amount: $59.5 million

Type: Series D financing

Date announced: April 21

Purpose: Advance lead drug candidate PNT2258, a cancer drug targeting the BCL2 gene, in several Phase II clinical studies in patients with relapsed or treatment refractory non-Hodgkin's lymphoma including those with diffuse large B-cell lymphoma (DLBCL), Richter's transformation, and follicular lymphoma. Proceeds will also be used to support drug manufacturing, advance development of preclinical drug candidates, and build ProNAi's organization in Michigan.

Financing leader: Vivo Capital

Additional financing partners: New investors Frazier Healthcare Ventures, OrbiMed Advisors, Adams Street Partners, RA Capital Management, Caxton Alternative Management, Hopen Life Science Ventures, Sectoral Asset Management, and Janus Capital Management; also participating were existing investors, including Capital Midwest Fund, Apjohn Ventures Fund, Amherst Fund, and Grand Angels.


#11. KemPharm

Amount: $60 million2

Types: Senior secured term debt facility ($50 million); senior secured convertible note ($10 million)2

Date announced: June 3

Purpose: Fund clinical and regulatory operations and advance lead product candidate, KP201, through regulatory approval and initiate commercialization. KP201 is being developed as an abuse-deterrent opioid analgesic.

Financing leader: Deerfield Management

Additional financing partners: None.


#10. PaxVax

Amount: $62 million

Types: Secured debt financing ($50 million); Series B venture funding extension ($12 million)3

Date announced: July 28

Purpose: Support new European operations and clinical development, regulatory review, and anticipated global launch of cholera vaccine candidate PXVX0200, currently in Phase III clinical testing.

Financing leader: Secured debt financing from an investment fund managed by Pharmakon Advisors

Additional financial partners: Series B financing by “existing and new investors.” Company’s equity investors include Ignition Ventures, Ignition Growth Capital and the Blue Haven Initiative.


#9. Pernix Therapeutics

Amount: $65 million

Type: Convertible senior notes due 20194

Date announced: February 5

Purpose: Provide the company with expansion capital for acquisition of accretive specialty products to be added to the company's portfolio, as well as for working capital and general corporate purposes.

Financing leader: Athyrium Capital Management

Additional financial partners: Group of undisclosed institutional investors.


#8. Novimmune

Amount: $62.7 million (CHF $60 million)

Type: Series B financing

Date announced: February 18

Purpose: Pursue “three-pillar” strategy: Advance interferon-gamma inhibitor NI-0501, a fully human monoclonal antibody and clinical-stage drug candidate for the rare immune system disorder hemophagocytic lymphohistiocytosis, which company intends to develop and commercialize with its own resources into a “high-value orphan business”; advance NI-0101, a first-in-class clinical-stage anti-TLR4 antibody, with the goal of establishing “a major strategic partnership”; develop kappa/lambda body™ bispecific drug platform, particularly its application to selectively inhibit CD47 on cancer cells, designed to benefit a broad range of cancer patients.

Financing leader: Rosetta Capital

Additional financial partners: Undisclosed “new private investors as well as existing investors.”


#7. Bellicum Pharmaceuticals

Amount: $69.7 million5

Type: Series B financing ($14.7 million) and Series C financing ($55 million)

Dates announced: January 7 (Series B completion); August 27 (Series C financing)

Purpose: Expand the clinical evaluation of stem cell transplant, chimeric antigen receptor (CAR) T-cell and cancer vaccine product candidates in a variety of blood and solid tumor cancers, and in nonmalignant genetic diseases.

Financing leader: No leader denoted

Additional financial partners: 11 new investors, including funds managed by RA Capital Management; Perceptive Advisors; Jennison Associates (on behalf of certain clients); Sabby Capital; Ridgeback Capital Management; venBio Select; Redmile Group; AJU IB Investment; and other new investors that were not disclosed in announcement. Also, all of the company’s existing investors participated, including AVG Ventures and Remeditex Ventures.


#5 (tie). Melinta

Amount: $70 million

Type: Series C financing

Date announced: February 10

Purposes: Advance delafloxacin, a differentiated fluoroquinolone, through an ongoing Phase III study of a single oral dose in individuals with uncomplicated gonorrhea, to the submission of a New Drug Application (NDA); support delafloxacin's two ongoing Phase III trials for the treatment of acute bacterial skin and skin structure infections (ABSSSI) and the selection of lead candidates from Melinta’s RX-04 research program.

Financing leader: Vatera Healthcare Partners, an existing investor

Additional financial partners: New investor Falcon Flight, an investment vehicle for the Santo Domingo Group; Undisclosed “existing investors as well as new investors.”


#5 (tie). Human Longevity (HLI)

Amount: $70 million

Type: Series A financing

Date announced: March 4

Purpose: Support company’s operations through its first 18 months.

Financing leader: Tan Sri Lim Kok Thay, chairman, president, and CEO of the Genting Group in Malaysia

Additional financial partners: Undisclosed investors termed “mostly very high net-worth individuals” by company co-founder J. Craig Venter, Ph.D.


#4. Adaptimmune

Amount: $104 million

Type: Series A financing

Date announced: September 25

Purpose: Advance internal programs into the clinic in multiple cancer indications.

Financing leader: New Enterprise Associates (NEA)

Additional financial partners: Participating new investors included OrbiMed Advisors, Wellington Management Company, Fidelity Biosciences, Foresite Capital Management, Ridgeback Capital Management, Novo A/S, QVT, Rock Springs Capital, venBio Select, and Merlin Nexus. Participating existing investors included the University of Oxford and other undisclosed partners.


#3. Adaptive Biotechnologies

Amount: $105 million

Type: Completion of Series C financing ($5 million) and Series D financing ($100 million)

Date announced: April 7

Purpose: Expand the company's research platform immunoSEQ™ via the launch of a Research Use Only kit in the third quarter, designed to enable customers to perform immunosequencing in-house; expand the company's first diagnostic product clonoSEQ™ for monitoring relapse in patients with certain blood cancers; validate and launch an assay called quanTILfy™ that interrogates tumor-infiltrating lymphocytes in solid tumors for prognosis and as a potential predictive biomarker of response to treatment with cancer immunotherapies.

Financing leader: Viking Global Investors

Additional financial partners: Three undisclosed investors (launch of Series C);6 none (Series D).


#2. Flatiron Health

Amount: $130 million

Type: Series B financing

Date announced: May 8

Purpose: Acquire Altos Solutions, developer of the OncoEMR™ cloud-based electronic medical record system, and integrate OncoEMR with Flatiron Health’s OncologyCloud™ platform, enabling Flatiron to combine point-of-care data capture and patient engagement with advanced analytics. Altos continues to operate as a wholly owned subsidiary of Flatiron Health.

Financing leader: Google Ventures

Additional financing partners: Existing investors First Round Capital and Laboratory Corporation of America Holdings (LabCorp®).


#1. Intarcia Therapeutics

Amount: $200 million

Type: Private round of equity financing

Date announced: April 1

Purpose: Complete Phase III FREEDOM clinical trial program for wholly owned lead product ITCA 650, positioned as the first and only once-yearly, injection-free GLP-1 therapy for the treatment of type 2 diabetes;7 prepare for future product launch; build pipeline of additional programs.

Financing leader: RA Capital

Additional financial partners: New and existing investors that include Farallon Capital Management, Foresite Capital, Franklin Templeton, Fred Alger Management, New Leaf Venture Partners, Quilvest, and three additional “large top-tier” institutional investors that participated in an earlier $210 million round of equity and debt financing in November 2012.

















Notes:
1 Company has agreed to be acquired by Johnson & Johnson for $1.75 billion cash, in a deal announced September 30, and expected to close in the fourth quarter.
2 At the initial closing of the transaction, KemPharm said, Deerfield funded $15 million of the term loan and $10 million of the convertible notes. KemPharm said it may access the remaining $35 million of the term debt upon completion of undisclosed milestones.
3 Financing secured as part of the company's acquisition of the FDA-approved oral typhoid vaccine Vivotif® from Johnson & Johnson entity Crucell Switzerland AG.
4 The notes carry a coupon of 8% and are convertible into shares of Pernix common stock at an initial conversion price of $3.60 per share, representing a conversion premium of more than 70% over the last reported sale price of Pernix' common stock on the NASDAQ Global Market on February 4, 2014.
5 Includes $14.7 million secured in the second and final closing of its Series B financing, though not the initial roughly $20 million in Series B capital that was announced as having been raised and closed in 2012. The total value of the Series B financing was $34.4 million.
6 According to a Form D dated July 7, 2013 and filed by the company with the U.S. Securities and Exchange Commission, $5,024,792 in equity was sold to the three investors, toward an equity offering amount of $19,999,997 whose date of first sale was July 1, 2013.
7 Company said it expects final clinical results needed to support global regulatory filings by early 2016.

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