Hemab chairman John Maraganore (Ryan Emberley/Getty Images for Klick Inc.)

Rare blood dis­or­ders biotech Hemab rais­es $135M, be­gins trek in­to clin­ic

Hemab Ther­a­peu­tics, led by Al­ny­lam vet Ben­ny Sorensen and chaired by John Maraganore, has raised $135 mil­lion from a new crop of in­vestors as it be­gins push­ing its blood dis­or­der pro­grams in­to the clin­ic.

Ben­ny Sorensen

The raise gives Hemab a cash run­way in­to 2025, and the biotech has a slate of goals it wants to check off be­fore then, in­clud­ing hav­ing five clin­i­cal can­di­dates by 2025. Maraganore said in an in­ter­view with End­points News that the biotech wants to be in Phase III tri­als by then as well.

For now, how­ev­er, the com­pa­ny has just got­ten start­ed with hu­man tri­als.

Hemab just be­gan a Phase I/II tri­al of a bis­pe­cif­ic an­ti­body dubbed HMB-001, which it’s test­ing for Glanz­mann throm­bas­the­nia, a rare blood clot­ting dis­or­der. In Jan­u­ary, the biotech an­nounced it had dosed its first pa­tient with the drug. The tri­al is cur­rent­ly run­ning in the UK, though Hemab said it plans on ex­pand­ing that to the US and oth­er Eu­ro­pean coun­tries. Ear­ly da­ta from the tri­al are ex­pect­ed lat­er this year.

The biotech is al­so work­ing on a pre­clin­i­cal treat­ment for von Wille­brand dis­ease. Take­da’s en­zyme re­place­ment ther­a­py for von Wille­brand dis­ease, Von­ven­di, was ap­proved ear­ly last year. Ve­ga Ther­a­peu­tics, which like Hemab is backed by RA Cap­i­tal, is al­so work­ing on a treat­ment for the bleed­ing dis­or­der and it hopes to start clin­i­cal tri­als this year.

Im­por­tant­ly, Hemab is not work­ing on he­mo­phil­ia A or B, Sorensen em­pha­sized dur­ing the joint in­ter­view, and is in­stead de­vel­op­ing treat­ments for ne­glect­ed bleed­ing dis­or­ders that cur­rent­ly have few or no ap­proved op­tions.

Hemab said it hopes its Se­ries B will bankroll the cur­rent study on HMB-001 and the start of a piv­otal tri­al, as well as a Phase I/II tri­al for the von Wille­brand dis­ease ther­a­py.

Hemab’s Se­ries B was led by Ac­cess Biotech­nol­o­gy, fol­lowed by Deep Track Cap­i­tal, Avoro Cap­i­tal, In­vus, Rock Springs Cap­i­tal and Dan­ish fund Maj In­vest Eq­ui­ty — all new in­vestors. In­vestors from the biotech’s $55 mil­lion Se­ries A — No­vo Hold­ings, RA Cap­i­tal Man­age­ment, and Health­Cap — al­so re­turned for the round.

Hemab could have raised over $200 mil­lion, Maraganore said, but took the $135 mil­lion it need­ed to reach its 2025 goals.

So what hap­pens in 2025?

“At that point in time, we can choose to go pub­lic or we can choose to raise an­oth­er round pri­vate­ly, but at a high­er val­u­a­tion,” Maraganore said.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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As­pen looks to re­bound in pro­duc­tion and rev­enue af­ter Covid-19

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.